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https://euclinicaltrials.eu/search-for-clinical-trials/trial-map/?lang=fr
| Maladies rares concernées par l'étude | Medical conditions | Nom de l'étude en anglais |
|---|---|---|
| Vascularite associée aux anticorps anticytoplasme des polynucléaires neutrophiles | ANCA-associated vasculitis | A multicenter randomized trial to evaluate the efficacy of pioglitazone to promote renal tolerance in ANCA-associated vasculitis - RENATO |
| Traitement de la granulomatose chronique liée au chromosome X | Treatment of X-linked Chronic Granulomatous Disease | A phase I/II, non randomized, monocentric open-label study of autologous CD34+ cells transduced with the G1XCGD lentiviral vector in patients with X-linked chronic granulomatous disease |
| Syndrome néphrotique à changements minimes | Minimal Change Nephrotic Syndrom | RIFIREINS. Rituximab from the first episode of Minimal Change Nephrotic Syndrome for preventing relapse risk in adult patients |
| Démence fronto-temporale avec mutations du gène de la progranuline (FTD-GRN) | Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) | A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of LY3884963 in Patients with Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) (PROCLAIM) |
| Polycythémie vraie (PV) | Polycythaemia Vera (PV) | MITHRIDATE: A phase III, randomised, open-label, Multicenter International Trial comparing ruxolitinib with either HydRoxycarbamIDe or interferon Alpha as first line ThErapy for high risk polycythemia vera |
| Sarcoïdose extrapulmonaire | Extra pulmonary sarcoidosis | HydroxyChloroquine as steroid-sparing Agent in Extra-pulmonary SARcoidosis. A multicenter, prospective, placebo-controlled, randomized trial. CAESAR |
| Phénomène de Raynaud | Raynaud phenomenon | Phase II/III double-blind randomized placebo-controlled trial assessing the preventive effect of Clopidogrel on the systemic sclerosis development risk in subjects with specific dysimmunity and Raynaud phenomenon - PSSIT |
| Polyneuropathie inflammatoire démyélinisante chronique | Chronic Inflammatory Demyelinating Polyneuropathy | Phase 2/3, Multistage, Multicenter, Randomized, Double-Blind, Placebo-Controlled Parallel Group Withdrawal Study to Evaluate the Efficacy and Safety of Nipocalimab Administered to Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) |
| Hyperplasie des cellules neuroendocrines de l'enfance | NEUROENDOCRINE CELLS HYPERPLASIA OF INFANCY | Efficacy of methylprednisolone pulses in neuroendocrine cells hyperplasia of infancy: an early phase study (CORTICONEHI). |
| Maladie pulmonaire fibrotique | Fibrotic Lung Disease | An Open-label Extension Study of Inhaled Treprostinil in Subjects with Fibrotic Lung Disease |
| Mucoviscidose | Cystic fibrosis | A Phase 2a, Randomized, Placebo-Controlled, Double Blind Multiple Ascending Dose Study in Patients with Cystic Fibrosis Carrying the 3849 +10 Kb C->T Mutation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84? |
| Mucoviscidose | Cystic fibrosis | A clinical trial to evaluate the long-term safety and durability of efficacy of BI 3720931, an inhaled lentiviral vector gene therapy, after single dose administration in a previous clinical trial in people with cystic fibrosis rolled-over from a previous clinical trial with BI 3720931 (LenticlairTM-ON). |
| Sclérose systémique cutanée diffuse (dcSSc) | Diffuse cutaneous systemic sclerosis (dcSSc) | A Phase II, multi-part, five-year, randomized, open-label, assessor-blinded, active-controlled, multicenter study to evaluate the efficacy and safety of rapcabtagene autoleucel versus rituximab treatment in participants with severe refractory diffuse cutaneous systemic sclerosis |
| Myélofibrose naïve de traitement par inhibiteur JAK, thrombocytopénie modérée | JAK inhibitor-naïve myelofibrosis, moderate thrombocytopenia | A Phase 2 study to evaluate the efficacy and safety of selinexor monotherapy in subjects with JAK inhibitor-naïve myelofibrosis and moderate thrombocytopenia. |
| Lipodystrophie généralisée | Generalised lipodystrophy | Open-label, Phase 3b study to evaluate effectiveness, safety and pharmacokinetic parameters of metreleptin in patients under 6 years of age with generalised lipodystrophy and associated diabetes mellitus and/or hypertriglyceridaemia |
| Neuropathie sensorielle paranéoplasique avec anticorps anti-Hu | paraneoplastic sensory neuropathy with anti-Hu antibodies | Early immunotherapy with intravenous immunoglobulin, cyclophosphamide and methylprednisolone in patients with anti-Hu-associated paraneoplastic sensory neuropathy (NESPA). |
| Syndrome myélodysplasique (SMD) | Myelodysplastic Syndrome (MDS) | A Phase 3, Open-label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE 536) vs Epoetin Alfa for the Treatment of Anemia Due to Revised International Prognostic Scoring System (IPSS-R) Very Low, Low, or Intermediate Risk Myelodysplastic Syndrome (MDS) in Erythropoiesis-stimulating Agent (ESA)-naive Participants who are Non-transfusion Dependent (NTD): The “ELEMENT-MDS” Trial |
| Gliomes du tronc cérébral chez l'adulte, adultes présentant des gliomes diffus du tronc cérébral sans rehaussement contrasté avec progression clinique et/ou radiologique infiltrative non menaçante | Adult brain stem gliomas, Adults with non-contrast-enhancing diffuse brainstem gliomas with non-threatening infiltrative clinical and/or radiological progression | First-line Chemotherapy With Temozolomide Alone for Non-enhancing Adult Brainstem Gliomas, With a Diffuse Subtype and Showing Clinical and/or Radiological Infiltrative Pattern of Progression (TEMOTRAD01) |
| Maladie rénale protéinurique médiée par l'APOL1 (AMKD) | APOL1-mediated Proteinuric Kidney Disease (AMKD) | A Phase 2/3 Adaptive, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Adult and Pediatric Subjects With APOL1-mediated Proteinuric Kidney Disease |
| Troubles métaboliques - Phénylcétonurie | Metabolic Disorders - Phenylketonuria | A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria |
| Drépanocytose | Sickle-cell disease | Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector |
| Cryoglobulinémie vasculaire | Cryoglobulinemia vasculitis | TRIBECA - Multicenter randomized double-blind study comparing the efficacy and safety of belimumab in the treatment of non-infectious active cryoglobulinemia vasculitis compared to placebo TRIBECA STUDY (Treatment after RItuximab with BElimumab in mixed Cryoglobulinemia Associated vasculitis) |
| Sarcome d'Ewing récurrent et primaire réfractaire | Recurrent and Primary Refractory Ewing Sarcoma | rEECur: International Randomised Controlled Trial of Chemotherapy for the Treatment of Recurrent and Primary Refractory Ewing Sarcoma |
| Acidémie méthylmalonique isolée due à un déficit en méthylmalonyl-CoA mutase | Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency | A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants with Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency |
| Hypertension artérielle pulmonaire (HAP) | Pulmonary arterial hypertension (PAH) | A Study Evaluating the Long-term Efficacy and Safety of Ralinepag in Subjects with PAH via an Open-label Extension (ADVANCE-Extension) |
| Télangiectasie hémorragique héréditaire | Hereditary haemorrhagic telangiectasia | Phase II randomized study on efficacy of nintedanib for treatment of epistaxis in hereditary haemorrhagic telangiectasia (HHT) patients - EPISTOP |
| Myasthénie grave généralisée (MGg) | Generalized Myasthenia Gravis (gMG) | A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to < 18 years of age) with Generalized Myasthenia Gravis (gMG) |
| Syndromes myélodysplasiques à haut risque non traités et non éligibles pour une greffe allogénique | Higher-risk myelodysplastic syndromes untreated and ineligible for allogenic transplantation | GFM-ONUVEN-MDS : A phase I/II, open-label, single arm, multicenter dose-finding study to assess the safety and preliminary efficacy of Oral Azacitidine CC-486 (ONUREG®) in combination with Venetoclax (VENCLYXTO®) in previously untreated higher-risk myelodysplastic syndromes ineligible for allogenic transplantation |
| Prurit cholestatique | Cholestatic Pruritus | Randomized Double-Blind Placebo-Controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Participants with Cholestatic Pruritus |
| Glomérulopathie du complément 3 | complement 3 glomerulopathy | A multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in complement 3glomerulopathy |
| Participants atteints d'acidémie méthylmalonique isolée (MMA) due à un déficit en méthylmalonyl-coenzyme A mutase (MUT) | participants with isolated methylmalonic acidemia (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency | A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705. |
| Syndrome de Turner, déficit en gène contenant un homéobox et syndrome de Noonan | Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome | A Phase 2, Randomized, Human Growth Hormone-Controlled, Multicenter, Basket Study of Vosoritide in Children with Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome with an Inadequate Response to Human Growth Hormone |
| Épidermolyse bulleuse simple | Epidermolysis bullosa simplex | EBUL0. A 20-Week Multicentre, Open Study Assessing the Efficacy and Safety of Apremilast in Patients > 6 years of age with Epidermolysis bullosa simplex generalized |
| Syndrome hémolytique et urémique atypique (SHUa) | Atypical Hemolytic Uremic Syndrome (aHUS) | A multicenter, single arm, open-label study to evaluate efficacy and safety of switching from anti-C5 antibody therapy to iptacopan therapy in study participants with aHUS |
| Polyglobulie familiale associée à une mutation du gène EPO | familial polycythemia associated with an EPO gene mutation | Evaluation of the efficacy and safety of drug treatment in familial polycythemia associated with an EPO gene mutation: a multicentre, open-label, phase I trial |
| Mastocytose systémique non avancée | Nonadvanced Systemic Mastocytosis | (Summit) A Multi-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Clinical Study of the Safety and Efficacy of CGT9486 in Subjects with NonAdvanced Systemic Mastocytosis |
| Hypoglycémie post-bariatrique | Post-Bariatric Hypoglycaemia | A double-blind, randomized, placebo-controlled, dose-finding phase II study to assess the efficacy and safety of pasireotide s.c. in patients with Post-Bariatric Hypoglycaemia |
| Infection chronique par le virus de l'hépatite D (VHD) | Chronic Hepatitis D Virus (HDV) Infection | A Phase 3 Randomized, Open Label Study to Evaluate the Efficacy and Safety of Tobevibart+Elebsiran Combination Therapy in Participants with Chronic HDV Infection Not Virologically Suppressed with Bulevirtide (ECLIPSE 2) |
| Polyneuropathie amyloïde héréditaire à transthyrétine | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | An Open-label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy |
| Dystrophie myotonique de type 1 (DM1) | Myotonic dystrophy type 1 (DM1) | A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple-Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Clinical Efficacy of Intravenous Administration of ATX-01 In Male and Female Participants aged 18 to 64 with Classic Myotonic Dystrophy Type 1 (DM1) |
| Fibrose pulmonaire progressive | Progressive Pulmonary Fibrosis | A Randomized, Double-Blind, Placebo-Controlled, Phase 2b Study Evaluating the Safety and Efficacy of Pirfenidone Solution for Inhalation (AP01) in Subjects with Progressive Pulmonary Fibrosis (PPF) |
| Hypochondroplasie | Hypochondroplasia | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children with Hypochondroplasia |
| Variante TBR1 pathogène avérée ou probablement pathogène | Proven pathogenic or probably pathogenic TBR1 variant | A pilot, multicentre, controlled, open-label study evaluating 24 months of lithium carbonate treatment in patients with TBR1-related neurocognitive disorder. ESALIT (Efficacy- Safety-Lithium-TBR1) |
| Hémophilie A | Haemophilia A | A multinational, prospective, open-label, roll-over study (LIBERTY) to provide post-trial access to treatment for patients with severe haemophilia A who have completed a previous trial with efanesoctocog alfa |
| Hypertension artérielle pulmonaire (groupe I) | Pulmonary arterial hypertension (group I) | Randomized trial comparing efficacy and safety of initial triple therapy including parenteral treprostinil to initial double oral therapy in PAH group I patients – TripleTRE |
| Ataxie spinocérébelleuse de type 1, 3 et maladie de Huntington | Spinocerebellar ataxia types 1, 3 and Huntington's disease | A phase 1/2a, open-label trial to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending doses of intrathecally administered VO659 in participants with spinocerebellar ataxia types 1, 3 and Huntington’s disease |
| Angio-œdème héréditaire | Hereditary angioedema | A Phase II/III, Extension Study of Orally Administered PHA-022121 for Acute Treatment of Angioedema Attacks in Patients with Hereditary Angioedema |
| Polyneuropathie inflammatoire démyélinisante chronique (PIDC) | Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) | A Phase 2, Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Intravenous NVG-2089 in Participants with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) |
| Mélanome uvéal | Uveal Melanoma | EORTC 2022-MG: Adjuvant tebentafusp (IMCgp100) versus observation in HLA-A*02:01 positive patients following definitive treatment of high-risk uveal melanoma: an EORTC randomized phase III study (ATOM Trial) |
| Prurit cholestatique chez les patients atteints de cholangite biliaire primitive (CBP). | Cholestatic Pruritus in Patients with Primary Biliary Cholangitis (PBC). | A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients with Primary Biliary Cholangitis (VANTAGE) |
| Défini par le protocole parent. L'étude recrutera tous les sujets adultes et pédiatriques ayant reçu au moins une perfusion de cellules T génétiquement modifiées dans le cadre d'une précédente étude sponsorisée par Celgene. | Defined by parent protocol. The study will enroll all adult and paediatric subjects who received at least one genetically modified T cells infusion in a previous Celgene sponsored study. | LONG-TERM FOLLOW-UP PROTOCOL FOR SUBJECTS TREATED WITH GENE-MODIFIED T CELLS |
| Sarcomes des tissus mous localisés et opérables | Localised and operable soft tissue sarcomas | RT-IMMUNE - A European, multicenter, randomized, open-label, Phase II trial, aiming to assess the clinical and biological activity of an anti-PD-L1 (atezolizumab) in operable localised soft tissue sarcomas patients to be treated with radiotherapy |
| Ichtyose congénitale | Congenital Ichthyosis | Phase 2 study evaluating the efficacy of injectable gentamicin in hereditary ichthyosis - GENTIC |
| Néoplasmes myéloprolifératifs | Myeloproliferative Neoplasms | A study to evaluate the effects and safety of INCA033989 in participants with myelofibrosis or essential thrombocythemia |
| Léiomyosarcome utérin localisé, réséqué, stade FIGO I (classification 2018) | Localized, resected, FIGO stage I (2018 classification), uterine leiomyosarcoma | L-UteCIN : Interest of adjuvant chemotherapy in patients with CINSARC high-risk localized resected uterine leiomyosarcoma |
| Anémie due à des syndromes myélodysplasiques à faible risque | Anemia Due to Lower-Risk Myelodysplastic Syndromes | A Phase 2a/2b, Open-label, Proof of Concept (Phase 2a) and Open-label (Phase 2b), Multicenter, Efficacy, and Safety Study of AG-946in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes |
| Patients atteints d'hémopathies malignes et subissant une greffe allogénique de cellules hématopoïétiques | Patients with hematologic malignancies and undergoing allogenic hematopoietic cell transplantation | A multi-center randomized, double blinded phase IIb trial evaluating oral pooled fecal microbiotherapy MaaT033 to prevent allogeneic hematopoietic cell transplantation complications |
| Mégalencéphalie-malformation capillaire Syndrome de polymicrogyrie | Megalencephaly-CApillary malformation Polymicrogyria syndrome | A Phase II double-blind multi-center, placebo-controlled trial, to assess the efficacy and safety of alpelisib (BYL719) in pediatric and adult patients with Megalencephaly-CApillary malformation Polymicrogyria syndrome (MCAP) |
| Amylose à chaînes légères | Light Chain Amyloidosis | Study evaluating the Safety and Efficacy of Etentamig (ABBV-383) in Subjects with Light Chain Amyloidosis |
| Atrophie multisystémique | Multiple System Atrophy | A Multi-centered, Double-blind, Randomized, Placebo-controlled, Parallel Group Phase 2 Study of TEV-56286 for the Treatment of Patients with Multiple System Atrophy |
| Syndrome de Crigler-Najjar sévère chez les patients nécessitant une photothérapie | severe Crigler-Najjar syndrome in patients requiring phototherapy | CareCN: A phase I/II, open-label, study to evaluate the safety and efficacy of an intravenous injection of GNT0003 (Adeno-associated Viral Vector expressing the UGT1A1 transgene) in patients with severe Crigler-Najjar syndrome requiring phototherapy |
| Malignités à cellules B récidivantes/réfractaires | Relapsed/Refractory B-cell Malignancies | A Study of NX-5948 in Adults With Relapsed/Refractory B-cell Malignancies |
| Myopathies inflammatoires idiopathiques (IIM) sévères réfractaires | Severe refractory idiopathic inflammatory myopathies (IIM) | A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucelversus comparator in participants with severe refractory idiopathic inflammatory myopathies |
| Myasthénie grave généralisée résistante au traitement | Treatment-resistant generalized Myasthenia Gravis | An open-label, multi-center, phase I/II study to assess safety, efficacy, and cellular kinetics of YTB323 in participants with treatment-resistant generalized myasthenia gravis |
| Néphropathie à IgA | IgA nephropathy | Therapeutic effect of Hydroxychloroquine on IgA Nephropathy course QUIgAN Study |
| Ulcères digitaux dans la sclérose systémique | Digital ulcers in systemic sclerosis | Local and systemic safety and tolerability of ascending doses of TOP-N53, a nitric oxide (NO)-releasing phosphodiesterase-5 (PDE5) inhibitor, administered topically, on wound in patients with digital ulcers (DU) in systemic sclerosis (SSc) in an open-label, vehicle-controlled, phase 2a, multi-center clinical trial |
| Fibrodysplasie ossifiante progressive | Fibrodysplasia Ossificans Progressiva | A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva (PROGRESS) |
| Maladie du greffon contre l'hôte aiguë à haut risque | High-Risk Acute Graft Versus Host Disease | A Multicenter, Randomized, Double-blind, Placebo-Controlled Phase II Study to Investigate the Efficacy and Safety of CYP-001 in Combination with Corticosteroids vs Corticosteroids Alone for the Treatment of High-Risk Acute Graft Versus Host Disease |
| Polyneuropathie inflammatoire démyélinisante chronique | Chronic Inflammatory Demyelinating Polyneuropathy | Open-label Extension of the ARGX-113-1802 Trial to Investigate the Longterm Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) |
| Amylose à transthyrétine | Transthyretin Amyloidosis | A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial) |
| WM non traitée auparavant, macroglobulinémie de Waldenström récidivante/réfractaire | previously untreated WM, Relapsed/Refractory Waldenström’s Macroglobulinemia | An Open Label, Multicenter Phase 2 Study to Evaluate the Efficacy and Safety of the BCL2 Inhibitor Sonrotoclax (BGB 11417) as Monotherapy and in Combination with Zanubrutinib (BGB-3111) in Patients With Waldenström Macroglobulinemia |
| Myélofibrose primaire ou secondaire à risque intermédiaire ou élevé | Intermediate or High-risk Primary or Secondary Myelofibrosis | A Phase 1/2, Open-label, Dose-escalation, Safety, Pharmacokinetic, and Pharmacodynamic Study of Oral Nuvisertib (TP-3654) in Patients with Intermediate or High-risk Primary or Secondary Myelofibrosis |
| Fibrose pulmonaire progressive | Progressive Pulmonary Fibrosis | A Randomized, Double-blind, Placebo-controlled, Multinational, Phase 3 Study of the Efficacy and Safety of Inhaled Treprostinil in Subjects with Progressive Pulmonary Fibrosis (TETON-PPF) |
| Myosite à inclusions | Inclusion body Myositis | BIGTIM: Blocking Interferon-? by ruxolitinib for Treating Inclusion body Myositis: a phase IIb trial. |
| Interferonopathies de type 1 | Type 1 interferonopathies | "A study to test how well BI 3000202 is tolerated by people with type 1 interferonopathies" |
| Dystrophie musculaire de Duchenne (DMD) | Duchenne muscular dystrophy (DMD) | Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy, safety and tolerability of givinostat in non-ambulant patients with Duchenne Muscular Dystrophy |
| Myopathie nécrosante à médiation immunitaire, syndrome des antiphospholipides, syndrome de Behçet, dermatomyosite, pemphigoïde bulleuse, thrombocytopénie immunitaire | Immune-mediated necrotizing myopathy, Antiphospholipid syndrome, Behçet’s Syndrome, Dermatomyositis, Bullous pemphigoid, Immune Thrombocytopenia | Phase Ib Open Label Basket Trial of RAY121 to Inhibit Classical Complement Pathway in Immunological Diseases (RAINBOW Trial) |
| Syndrome de Dravet | Dravet Syndrome | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Investigate the Efficacy, Safety, and Tolerability of LP352 in the Treatment of Seizures in Children and Adults with Dravet Syndrome |
| Atrésie biliaire | Biliary Atresia | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia (BOLD-EXT) |
| Maladie de Von Willebrand de type 3 | Type 3 Von Willebrand Disease | A Phase III, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Prophylaxis in Patients with Type 3 Von Willebrand Disease |
| Hémophilie A | Haemophilia A | Nuwiq for Perioperative management Of patients With haemophilia A on Emicizumab Regular prophylaxis study (NuPOWER) |
| Gliome pédiatrique de bas grade présentant une altération activatrice du gène RAF nécessitant un traitement systémique de première ligne | Pediatric low-grade glioma harboring an activating RAF alteration requiring first-line systemic therapy | LOGGIC/FIREFLY-2: A Phase 3, Randomized, International Multicenter Trial of DAY101 Monotherapy Versus Standard of Care Chemotherapy in Patients with Pediatric Low-Grade Glioma Harboring an Activating RAF Alteration Requiring First-Line Systemic Therapy |
| Myasthénie grave généralisée | Generalized Myasthenia Gravis | A randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy, safety, and tolerability of remibrutinib in patients with generalized myasthenia gravis, followed by an open-label extension phase. |
| Mastocytose systémique indolente et SSM | Indolent Systemic Mastocytosis and SSM | A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic Mastocytosis |
| Drépanocytose et thalassémie | Sickle Cell Disease and Thalassaemia | An open-label, multi-centre, rollover study to characterise long-term safety and efficacy of etavopivat in adults, adolescents and children who have sickle cell disease or thalassaemia and have completed a treatment period in an etavopivat study |
| Traitement du syndrome de Wiskott-Aldrich | Treatment of Wiskott-Aldrich Syndrome | Long term safety follow up of patients enrolled in the phase I/II clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome (GTG002-07 and GTG003-08) |
| Cholangite biliaire primitive | Primary Biliary Cholangitis | A Phase III Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Efficacy and Safety of Elafibranor 80 mg on Long-Term Clinical Outcomes in Adult Participants With Primary Biliary Cholangitis (PBC) |
| Maladie hépatique associée à un déficit en alpha-1-antitrypsine | Alpha-1 Antitrypsin Deficiency-Associated Liver Disease | A Randomized, Double-blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency–Associated Liver Disease With METAVIR Stage F2 to F4 Fibrosis |
| Syndrome de Prader-Willi | Prader-Willi syndrome | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients with Prader-Willi Syndrome |
| Adultes atteints de cardiomyopathie amyloïde à transthyrétine (ATTR-CM) | Adults with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) | A Phase 2, Single Arm, Multicenter Study to Evaluate the Pharmacodynamics and Safety of Re-Treatment with ALXN2220 in Patients with Transthyretin Amyloid Cardiomyopathy |
| Syndrome de Tourette | Tourette disorder | Involvement of the serotonergic system in the control of impulsivity in Tourette disorder : ARITEP |
| Nouvellement diagnostiqué avec un épendymome intracrânien ou spinal (tous grades OMS), y compris les variantes d'épendymome : cellulaire, papillaire, myxopapillaire, à cellules claires et tanycytique ou épendymome anaplasique. | Newly diagnosed with an intracranial or spinal ependymoma (all WHO grades) including ependymoma variants: cellular, papillary, myxopapillary, clear-cell and tanycytic or anaplastic ependymoma. | SIOP Ependymoma Program II: An International Clinical Program for the diagnosis and treatment of children, adolescents and young adults with ependymoma |
| Patients pédiatriques hautement sensibilisés présentant un test de compatibilité croisée positif avec un rein provenant d'un donneur vivant ou décédé. | Highly sensitised paediatric patients with a positive crossmatch against a living or deceased donor kidney. | A single-arm, multi-centre trial to evaluate efficacy and safety of imlifidase in highly sensitised children (1-17 years) receiving a kidney transplant with positive crossmatch against a living or deceased donor converted to negative after imlifidase treatment |
| Hyperoxalurie primaire | Primary Hyperoxaluria | An Open-Label Roll-Over Study to Evaluate the Long-Term Safety and Efficacy of DCR-PHXC Solution for Injection (subcutaneous use) in Patients with Primary Hyperoxaluria |
| Cardiomyopathie amyloïde à transthyrétine (ATTR CM) | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) |
| Adultes diagnostiqués avec un SJS-TEN touchant au moins 10 % de la surface corporelle | Adults diagnosed with SJS-TEN with at least 10% of body surface area involved | Mesenchymal stromal cells treatment in Lyell syndrome: A pilot phase 1-2 open trial (LYSYME) |
| Traitement dans le cadre d'une greffe de cellules souches hématopoïétiques | Treatment in haematopoietic stem cell transplantation | An open-label, randomized, non-comparative multi-center phase I/II study to assess the safety and the efficacy of SMART101 after HLA-mismatched peripheral blood stem cell transplantation with post-transplant cyclophosphamide in patients with hematological malignancies |
| Patients atteints de CMML n'ayant jamais reçu d'HMA et présentant un risque élevé (HR, défini comme un risque CPSS-mol intermédiaire-2 ou élevé) | HMA-naïve, higher-risk (HR, defined as CPSS-mol risk intermediate-2 or high) CMML patients | AVENHIR trial: Phase II study with safety run-in of Azacitidine (AZA) combined with Venetoclax (VEN) in patients with higher-risk Chronic Myelomonocytic Leukemia (CMML) |
| Dystrophie musculaire de Duchenne (DMD) | Duchenne Muscular Dystrophy (DMD) | Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies. |
| Hémophilie | Haemophilia | B7841008 - AN OPEN-LABEL STUDY IN PEDIATRIC (<18 YEARS OF AGE), SEVERE HEMOPHILIA A PARTICIPANTS (COAGULATION FACTOR ACTIVITY <1%) WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY ?2%) WITH OR WITHOUT INHIBITORS COMPARING 12 MONTHS OF HISTORICAL STANDARD TREATMENT TO MARSTACIMAB PROPHYLAXIS |
| Achondroplasie chez les enfants et les adolescents | Achondroplasia in Children and Adolescents | A Phase 2, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial, evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Infants (0 to <2 years of age) with Achondroplasia followed by an Open Label Extension (OLE) period. |
| Amylose à transthyrétine avec cardiomyopathie (amylose ATTR avec cardiomyopathie) | Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy) | An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy) |
| Granulomatose éosinophile avec polyangéite (EGPA) | Eosinophilic granulomatosis with polyangiitis (EGPA) | A Phase 2, Double-blind, Randomized, Placebo-controlled Study to Investigate the Efficacy and Safety of NS-229 in the Treatment of Eosinophilic Granulomatosis With Polyangiitis |
| Tumeurs solides avec mutation ou surexpression/amplification du gène HER2 | HER2-mutated or overexpressed/amplified solid tumours | Beamion PANTUMOR-1: A Phase II, multicentre, multicohort, open-label trial to evaluate the efficacy and safety of oral zongertinib (BI 1810631) for the treatment of selected HER2-mutated or overexpressed/amplified solid tumours |
| Trouble du spectre de la neuromyélite optique (NMOSD) | Neuromyelitis Optica Spectrum Disorder (NMOSD) | A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD) |
| Syndrome de Crigler-Najjar | Crigler-Najjar syndrome | An open-label, phase 2 trial to evaluate the efficacy and safety of a single intravenous administration of GNT0003 (an adeno-associated viral vector expressing the UGT1A1 transgene) following imlifidase pre-treatment in adult participants with severe Crigler-Najjar syndrome requiring daily phototherapy and presenting pre-existing anti-AAV8 antibodies. |
| Arthrite juvénile idiopathique systémique | Systemic Juvenile Idiopathic Arthritis | I4V-MC-JAHU: Open-Label, Randomized Study with a Tocilizumab Reference Arm to Evaluate Safety, Efficacy and Pharmacokinetics of Baricitinib in Children from 1 to Less Than 18 Years of Age with Systemic Juvenile Idiopathic Arthritis |
| Patients après une greffe de cellules souches hématopoïétiques (GCSH) souffrant d'une infection nouvelle ou réactivée par le CMV, l'EBV ou l'AdV, réfractaire au traitement antiviral standard | Patients after HSCT suffering from new or reactivated CMV or EBV or AdV infection, refractory to standard antiviral treatment | Treatment of chemo-refractory viral infections after allogeneic stem cell transplantation with multispecific T cells against CMV, EBV and AdV: A phase III, prospective, multicentre clinical trial (TRACE) |
| Lupus érythémateux disséminé, myopathies inflammatoires idiopathiques | Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathies | A Study to Investigate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AZD5492 in Adult Participants with Systemic Lupus Erythematosus or Idiopathic Inflammatory Myopathies. |
| Gliome | Glioma | I3Y-MC-JPEH: A Randomized, Open-Label, Phase 2 Study Evaluating Abemaciclib in Combination With Temozolomide Compared to Temozolomide Monotherapy in Children and Young Adults With Newly Diagnosed High-Grade Glioma Following Radiotherapy. |
| Dysplasie ectodermique hypohidrotique liée au chromosome X (XLHED) | X-linked hypohidrotic ectodermal dysplasia (XLHED) | A prospective, open-label, genotype-match controlled, multicenter clinical trial to investigate the efficacy and safety of intra-amniotic ER004 as a prenatal treatment for male subjects with X-linked hypohidrotic ectodermal dysplasia (XLHED) |
| Néphropathie à immunoglobulines A (IgAN) | Immunoglobulin A Nephropathy (IgAN) | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants with Immunoglobulin A Nephropathy (IgAN) |
| Dermatomyosite, syndrome de Behçet, thrombocytopénie immunitaire, pemphigoïde bulleuse, syndrome des antiphospholipides, myopathie nécrosante à médiation immunitaire | Dermatomyositis, Behçet’s Syndrome, Immune Thrombocytopenia, Bullous pemphigoid, Antiphospholipid syndrome, Immune-mediated necrotizing myopathy | Phase IB open label, long-term, extension basket trial of RAY121 to inhibit classical complement pathway in immunological diseases (RAINBOW-LTE trial) |
| Hémophilie A | Hemophilia A | A Long-Term Follow-Up Study in Subjects with Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial |
| Mucopolysaccharidose de type II [MPS II] | Mucopolysaccharidosis Type II [MPS II] | A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II |
| Hyperoxalurie primaire de type 1 (PH1) | Primary Hyperoxaluria Type 1 (PH1) | A Phase 1/2 Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of ABO-101 in Participants with Primary Hyperoxaluria Type 1 (PH1) |
| Acromégalie | Acromegaly | A Phase 3 randomized 3-arm trial (double-blind Debio 4126, placebo control, and open-label Debio 4126), to assess the efficacy and safety of Debio 4126, a 12-week octreotide formulation, in patients with acromegaly previously treated with somatostatin analogs. |
| Syndrome hémolytique et urémique | Hemolytic Uremic Syndrome | A Phase III study to evaluate the efficacy of INM004 (Shiga antitoxin) in pediatric patients with Hemolytic Uremic Syndrome associated to infection by Shiga toxin-producing Escherichia coli |
| Syndrome myélodysplasique | Myelodysplastic Syndrome | A Phase 2, Randomized, Open-label, Study of Momelotinib in Participants with Anemia due to Low-risk Myelodysplastic Syndrome. |
| Fibrose pulmonaire idiopathique | idiopathic pulmonary fibrosis | A Phase 2, Multi-Center, Randomized, Double-Blind, Controlled Trial Evaluating the Safety and Efficacy of ENV-101 in Patients with Lung Fibrosis (WHISTLE-PF Trial) |
| Neuropathie motrice multifocale | Multifocal Motor Neuropathy | A Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Study to Evaluate Safety, Tolerability, Pharmacometrics, and Efficacy of DNTH103 in Adults with Multifocal Motor Neuropathy (MOMENTUM) |
| Néphropathie membraneuse | Membranous Nephropathy | Impact of dapagliflozin for the regulation of immunological activity in Membranous Nephropathy |
| Maladie rénale | Kidney Disease | A study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of GSK4771261 in healthy participants and participants with autosomal dominant polycystic kidney disease. |
| Maladies du système immunitaire. | Immune system diseases. | Long-term extension study to evaluate the safety and efficacy of riliprubart (SAR445088) in participants with chronic inflammatory demyelinating polyneuropathy (CIDP) |
| Patients pédiatriques atteints d'asthme éosinophile sévère. L'asthme est une affection pulmonaire chronique qui provoque une inflammation des voies respiratoires (les tubes qui transportent l'air vers et hors des poumons). Les patients atteints d'asthme sévère sont susceptibles d'avoir des taux élevés d'éosinophiles dans le sang. Les éosinophiles sont un type de globules blancs qui peuvent provoquer une inflammation, augmenter le nombre de crises d'asthme et contribuer au déclin de la fonction pulmonaire. | Paediatric Patients with Severe Eosinophilic Asthma Asthma is a long-term lung disorder that causes airways (the tubes that carry air in and out of the lungs) to become inflamed. Patients with severe asthma are likely to have high levels of eosinophils in their blood. Eosinophils are a type of white blood cell that can cause inflammation and can increase the number of asthma attacks and contribute to the decline of lung function. | A Multicentre, Randomised, Double-blind, Parallel Group, Placebocontrolled, Time-to-first Asthma Exacerbation Phase III Efficacy and Safety Study of Benralizumab in Paediatric Patients with Severe Eosinophilic Asthma (DOMINICA) |
| Patient atteint du syndrome de l'intestin court traité par antibiotiques pour une infection bactérienne | Patient with short bowel syndrome treated with antibiotics for a bacterial infection | Short bowel syndrome and study of the absorption of antibiotics with good oral bioavailability |
| HYPERSOMNIE IDIOPATHIQUE | IDIOPATHIC HYPERSOMNIA | A CHRONOBIOLOGICAL TREATMENT combining evening melatonin and morning light therapy IN IDIOPATHIC HYPERSOMNIA: a prospective, double bind, randomized, placebo-controlled -trial |
| Patients atteints de granulomatose avec polyangéite (GPA) et présentant une réponse insuffisante au traitement standard, définie comme une réponse insuffisante à une association de glucocorticoïdes et de cyclophosphamide ET/OU à une association de glucocorticoïdes et de rituximab. | Patients with GPA and inadequate response to standard of care therapy defined as an inadequate response to either a combination of glucocorticoids plus cyclophosphamide AND/OR a combination of glucocorticoids plus rituximab. | SATELITE - Salvage Therapy for Patients with Inadequate Response to Standard of Care Therapy in Granulomatosis with Polyangiitis |
| Patients adultes atteints de thrombocytopénie immunitaire (PTI) avec manifestations hémorragiques légères à sévères | Adult patients with immune thrombocytopenia (ITP) with mild to severe bleeding manifestations | Intravenous Immunoglobulin plus oral prednisone or high-dose dexamethasone, for adults with immune thrombocytopenia (ITP) with moderate and severe bleeding: a randomized, multicentre trial (IVIORDEX) |
| Maladie du greffon contre l'hôte aiguë réfractaire aux stéroïdes | Steroid refractory Acute Graft versus host Disease | A Randomised, Open label, Controlled, Multicentre, Phase 2 Trial of First line Treatment with Mesenchymal Stromal Cells MC0518 Versus Best Available Therapy in Paediatric Participants with Steroid refractory Acute Graft versus host Disease After Allogeneic Stem Cell Transplantation (BALDER Trial) |
| Maladie associée aux anticorps anti-glycoprotéine oligodendrocytaire de la myéline (MOGAD) | Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD) | A PHASE III, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SATRALIZUMAB AS MONOTHERAPY OR IN ADDITION TO BASELINE THERAPY IN PATIENTS WITH MYELIN OLIGODENDROCYTE GLYCOPROTEIN ANTIBODY-ASSOCIATED DISEASE (MOGAD) |
| Syndrome myélodysplasique à faible risque sans RS ayant échoué ou n'étant pas éligible à un agent stimulant les érythrocytes | Low risk myelodysplastic syndrom without RS having failed or being ineligible to Erythroid Stimulating Agent | COMBOLA Trial: A randomized phase I/ II multicenter study evaluating combination of luspatercept in LR-MDS without RS having failed or being ineligible to ESA |
| Fistule de Crohn. | Crohn’s fistula | Dose escalation of allogeneic Adipose derived Stroma/stem Cells for the treatment of Crohn’s fistula |
| Polyneuropathie inflammatoire démyélinisante chronique. | Polyneuropathy, Inflammatory Demyelinating, Chronic | A Phase 3, double-blind, placebo-controlled study evaluating efficacy and safety of riliprubart in participants with refractory chronic inflammatory demyelinating polyneuropathy |
| Sclérose latérale amyotrophique (SLA) | Amyotrophic Lateral Sclerosis (ALS) | A double-blind, randomized, placebo-controlled, multicentric, phase II study in adult patients with amyotrophic lateral sclerosis (ALS) to assess efficacy, safety, tolerability and pharmacokinetics of multiple intravenous infusions of NX210c. |
| Maladie hépatique associée à un déficit en alpha-1-antitrypsine | Alpha-1 Antitrypsin Deficiency-Associated Liver Disease | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Safety and Efficacy of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease With METAVIR Stage F1 Fibrosis |
| Maladie de stockage du glycogène de type 1a (GSD1a). | Glycogen storage disease type 1a (GSD1a) | A Phase 1/2, adaptive, open-label, single ascending dose to multiple ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of mRNA-3745 in participants with glycogen storage disease type 1a (GSD1a), followed by an open-label extension |
| Maladie pulmonaire interstitielle secondaire à une sclérose systémique (SSc-ILD) | interstitial lung disease secondary to systemic sclerosis (SSc-ILD) | CONQUEST Platform Clinical Study for Conquering Scleroderma: A Multicenter, Double-Blind, Randomized, Placebo-Controlled, Phase 2b Platform Clinical Study to Evaluate the Safety and Efficacy of Investigational Products in Participants with Interstitial Lung Disease Secondary to Systemic Sclerosis |
| Glomérulonéphrite membranoproliférative à complexes immuns idiopathique (IC-MPGN) | Idiopathic Immune Complex Mediated Membranoproliferative Glomerulonephritis (IC-MPGN) | A multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in idiopathic immune complex mediated membranoproliferative glomerulonephritis (IC-MPGN) |
| Lipodystrophie partielle | Partial Lipodystrophy | A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients with Partial Lipodystrophy |
| Déficit en ectonucléotide | Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency and ATP-binding cassette sub-family C member 6 (ABCC6) Deficiency, Generalized Arterial Calcification of Infancy (GACI), Pseudoxanthoma elasticum (PXE) | The ADAPT Study: An Open-Label, Long-term Safety Study of INZ-701 in Patients with ENPP1 Deficiency and ABCC6 Deficiency |
| Hémoglobinurie paroxystique nocturne | Paroxysmal Nocturnal Hemoglobinuria | A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis |
| Hémophilie | Haemophilia | B7841007 - An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Marstacimab Prophylaxis in Severe (Coagulation Factor Activity <1%) Hemophilia A Participants With or Without Inhibitors or Moderately Severe to Severe Hemophilia B Participants (Coagulation Factor Activity ?2%) With or Without Inhibitors |
| Amylose héréditaire à transthyrétine avec polyneuropathie (ATTRv-PN), cardiomyopathie liée à l'amylose à transthyrétine (ATTR-CM) | Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN)Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM) | Long-Term Follow-Up of Subjects Treated With NTLA-2001 |
| Folliculite induite par un inhibiteur de l'EGFR | EGFR inhibitor-induced folliculitis | A phase 2a multicentric study of TAR-0520 gel in EGFR inhibitor-induced folliculitis |
| Les patients hautement sensibilisés qui ont subi une transplantation rénale après l'administration d'Idefirix (imlifidase) et les patients moins sensibilisés ayant subi une transplantation rénale compatible, servant de cohorte de référence concomitante. | Highly sensitised patients who have undergone kidney transplantation after Idefirix (imlifidase) administration and less sensitized patients transplanted with compatible kidney transplant, serving as concurrent reference cohort | A prospective, post-authorisation long-term follow up trial of patients previously treated with imlifidase prior to kidney transplantation, including a non-comparative concurrent reference cohort |
| Sclérose latérale amyotrophique (SLA) | Amyotrophic Lateral Sclerosis (ALS) | A Phase 2a, Double-Blinded, Randomized, Placebo-Controlled, and Active-Treatment Extension Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of ARGX-119 in Participants With Amyotrophic Lateral Sclerosis |
| Arthrite juvénile idiopathique systémique (AJIS) | Systemic Juvenile Idiopathic Arthritis (sJIA) | An Open-label, Sequential, Ascending, Repeated Dose-finding Study of Sarilumab, Administered with Subcutaneous (SC) Injection, in Children and Adolescents, Aged 1 to 17 Years, with Systemic Juvenile Idiopathic Arthritis (sJIA), Followed by an Extension Phase. Name: SKYPS |
| Glomérulopathie C3 ou glomérulonéphrite membranoproliférative idiopathique à complexes immuns | C3 Glomerulopathy or idiopathic immune-complex membranoproliferative glomerulonephritis | An open-label, non-randomized extension study to evaluate the long-term efficacy, safety and tolerability of LNP023 in subjects with C3 glomerulopathy or idiopathic immune-complex-membranoproliferative glomerulonephritis |
| Dystrophie musculaire de Duchenne | Duchenne Muscular Dystrophy | Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boys |
| Amylose à transthyrétine avec cardiomyopathie | Transthyretin Amyloidosis with Cardiomyopathy | MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double?Blind, Placebo?Controlled Study to Evaluate the Efficacy and Safety of NTLA?2001 in Participants with Transthyretin Amyloidosis with Cardiomyopathy (ATTR?CM). |
| Patient présentant : une ossification ectopique secondaire à un iPPSD2 ou une calcification ectopique secondaire à une dermatomyosite ou une calcification ectopique secondaire à une sclérose systémique | Patient presenting with: - ectopic ossification secondary to iPPSD2 or - ectopic calcification secondary to dermatomyositis or - ectopic calcification secondary to systemic sclerosis | Subcutaneous Injections of Sodium Thiosulfate for ectopic calcifications or ossifications. A pilot study. |
| Lésions primaires indéterminées ou petit mélanome choroïdien | Primary Indeterminate Lesions or Small Choroidal Melanoma | A Phase 3 Randomized, Masked, Controlled Trial to Evaluate Efficacy and Safety of Belzupacap Sarotalocan (AU-011) Treatment Compared to Sham Control in Subjects with Primary Indeterminate Lesions or Small Choroidal Melanoma |
| Démence frontotemporale (FTD) | Frontotemporal Dementia (FTD) | A Phase 1/2, Multicenter, Randomized, Placebo-Controlled, Double-Blind Single Dose and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in Healthy Participants and Participants With Frontotemporal Dementia Followed by an Open-Label Extension |
| Traitement de l'IgAN | Treatment of IgAN | A Phase 2/3, Multicenter, Open?label Trial to Evaluate the Long?term Safety, Tolerability, and Efficacy of Sibeprenlimab Administered Subcutaneously in Subjects with Immunoglobulin A Nephropathy |
| Encéphalopathies développementales et épileptiques (DEE) | Developmental and Epileptic Encephalopathies (DEE) | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Investigate the Efficacy, Safety, and Tolerability of LP352 in the Treatment of Seizures in Children and Adults with Developmental and Epileptic Encephalopathies |
| CML-CP ou CML-AP patients ave une mutation T315I | CML-CP or CML-AP patients with T315I mutation | A Phase II, multi-center, prospective, open-label study of Asciminib in patients with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) or accelerated Phase (CML-AP) with T315I mutation who are resistant, intolerant or ineligible to ponatinib (ASC4TARGET) |
| Anémie hémolytique auto-immune | autoimmune hemolytic anemia | API-AHAI - Efficacy of Prolonged Anticoagulation for Primary Prevention of Venous Thromboembolic Disease in Autoimmune Hemolytic Anemia: a Prospective, Phase II, Randomized, Multicenter Study |
| Patients nouvellement diagnostiqués avec un sarcome d'Ewing métastatique (autre que des métastases pulmonaires/pleurales uniquement) | Newly diagnosed patients with metastatic (other than lung/pleura metastases only) Ewing sarcoma | Phase Ib study of the combination of regorafenib with conventional chemotherapy for the treatment of newly diagnosed patients with multimetastatic Ewing sarcoma - REGO-INTER-EWING1 |
| Sujets atteints de CBP avec une réponse non optimale à l'UDCA. | Subjects with PBC with a non-optimal response to UDCA. | Efficacy and safety of bezafibrate 400 mg and bezafibrate 200 mg as adjunctive treatments in patients with primary biliary cholangitis and non-optimal biochemical response to ursodeoxycholic acid therapy: a 12-month, double-blind, randomized, placebo-controlled trial with a 12-month, double-blind, placebo-free extension phase - BEZURSO 2 |
| Patients atteints de rétinite pigmentaire due à une mutation monogénique | Patients with retinitis pigmentosa due to monogenic mutation | A phase 1/2, open-label, safety, tolerability and preliminary efficacy study of implantation into one eye of hESC-derived RPE (Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE)) in patients with retinitis pigmentosa due to monogenic mutation |
| Génodermatoses épidermiques inflammatoires | Inflammatory EPidermal GENodermatoses | GENEPID: A 44-weeks monocentric open study assessing the efficacy and safety of Deucravacitinib in adults with Inflammatory EPidermal GENodermatoses (epidermolysis bullosa simplex and inflammatory congenital ichthyoses) |
| Néphropathie membraneuse | Membranous nephropathy | Study of Artificial Intelligence-based Personalized Rituximab Treatment Protocol in Membranous nephropathy |
| Sarcome synovial et liposarcome myxoïde/à cellules rondes (MRCLS) | Synovial sarcoma and Myxoid/Round Cell Liposarcoma (MRCLS) | Master Protocol to Assess the Safety and Antitumor Activity of Genetically Engineered NY-ESO-1-Specific (c259) T Cells, alone or in combination with other agents, in HLA-A2+ Participants with NY-ESO-1 and/or LAGE-1a Positive Solid Tumors (IGNYTE-ESO) |
| Tous les patients ayant été traités par Novartis ou Penn CAR-T pour toute indication | All patients who have been treated with Novartis or Penn CAR-T for any indication | Long Term Follow-Up of Patients Exposed to Lentiviral-Based CAR T-CellTherapy |
| Myosite à inclusions | inclusion body myositis | A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis |
| Brûlures profondes partielles et de pleine épaisseur | Deep partial and full-thickness burns | A multicentre, intra-patient randomised controlled Phase III study to confirm the efficacy and safety of denovoSkin™, a bilayer engineered collagen-based skin graft composed of autologous fibroblasts and keratinocytes, for the treatment of patients with deep partial and full-thickness burns |
| Maladie de von Willebrand sévère | severe von Willebrand Disease | A Phase 3, Prospective, Open-label, Uncontrolled, Multicenter Study on Efficacy and Safety of Prophylaxis with Vonicog Alfa (rVWF) in Children Diagnosed With Severe von Willebrand disease. |
| Maladie pulmonaire interstitielle associée à une sclérose systémique | systemic sclerosis associated interstitial lung disease | A Phase 2/3, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of belimumab administered subcutaneously in adults with systemic sclerosis associated interstitial lung disease (SSc-ILD) |
| Syndromes myélodysplasiques à faible risque ne répondant pas aux agents stimulant l'érythropoïèse et au luspatercept (ou non éligibles pour ce dernier) | low-risk Myelodysplastic Syndromes failing Erythropoiesis Stimulating Agents and Luspatercept (or ineligible for the latter) | ATOMYELO: Phase I study with dose-escalation and expansion evaluating the safety and efficacy of oral Arsenic (ATO) in low-risk Myelodysplastic Syndromes failing Erythropoiesis Stimulating Agents and Luspatercept (or ineligible for the latter) |
| Myasthénie grave | Myasthenia Gravis | A Phase 2b, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of 3 Dose Levels of NMD670 over 21 Days in Adult Patients with AChR/MuSK-Ab+ Myasthenia Gravis |
| Maladie de Charcot-Marie-Tooth de type 1 et de type 2 | Charcot-Marie-Tooth Disease Type 1 and Type 2 | A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 over 21 days in Ambulatory Adult Patients with Charcot-Marie-Tooth Disease Type 1 and Type 2 |
| Pyoderma gangrenosum | Pyoderma gangrenosum | A multi-centre, randomised, placebo-controlled, double-blind, parallel-group trial to evaluate safety and efficacy of spesolimab (BI 655130) in adult patients with ulcerative pyoderma gangrenosum (PG) who require systemic therapy |
| Hypercholestérolémie familiale hétérozygote | Heterozygous Familial Hypercholesterolemia | Two part (double-blind inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2]]) randomized multicenter study to evaluate safety, tolerability and efficacy of inclisiran in children (6 to less than 12 years) with heterozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-20) |
| Mésothéliome pleural inopérable | Unresectable Pleural Mesothelioma | A Phase III, Randomized, Open-Label, Multicenter, Global Study of Volrustomig (MEDI5752) in Combination with Carboplatin plus Pemetrexed Versus Platinum plus Pemetrexed or Nivolumab plus Ipilimumab in Participants with Unresectable Pleural Mesothelioma (eVOLVE-Meso) |
| Dystrophies rétiniennes héréditaires | Inherited retinal dystrophies | Clinical study to evaluate the safety and tolerability of SPVN06 (novel gene therapy) in a subset of patients with rod cone dystrophy (RCD). |
| Lymphome de la zone marginale (LZM) récidivant/réfractaire, lymphome folliculaire récidivant/réfractaire | Relapsed/Refractory Marginal Zone Lymphoma (MZL), Relapsed/Refractory Follicular Lymphoma | A Phase 3, Open Label, Randomized Study to Compare the Efficacy and Safety of Odronextamab (REGN1979), an Anti-CD20 x Anti-CD3 Bispecific Antibody, in Combination with Lenalidomide Versus Rituximab in Combination with Lenalidomide in Relapsed/Refractory Participants with Follicular Lymphoma and Marginal Zone Lymphoma (OLYMPIA-5) |
| Syndrome VEXAS | VEXAS Syndrome | PAXIS: A randomized, double-blind, placebo-controlled dose-finding phase 2 study (Part 1) followed by an open-label period (Part 2) to assess the efficacy and safety of pacritinib in patients with VEXAS syndrome |
| Narcolepsie de type 1, de type 2 et hypersomnie idiopathique | Narcolepsy Type 1, Type 2 and Idiopathic Hypersomnia | An Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Durability of Treatment Effect of ALKS 2680 in Subjects With Narcolepsy Type 1 and Type 2 and Idiopathic Hypersomnia |
| Syndromes myélodysplasiques naïfs d'agents hypométhylants (HMA), syndrome myélodysplasique (SMD), syndrome myélodysplasique (SMD) | Hypomethylating agent (HMA) naive myelodysplastic syndromes Myelodysplastic Symdrome (MDS), Myelodysplastic Symdrome (MDS) | A phase 3, multicenter, open label, randomized, non-comparative two-arm study of ivosidenib (IVO) monotherapy and azacitidine (AZA) monotherapy in adult patients with hypomethylating agent (HMA) naive myelodysplastic syndromes (MDS) with an isocitrate dehydrogenase-1 (IDH1) mutation (PyramIDH study). |
| Maladie du greffon contre l'hôte aiguë réfractaire aux stéroïdes | Steroid refractory Acute Graft versus host Disease | A Randomised, Open label, Multicentre, Phase 3 Trial of First line Treatment with Mesenchymal Stromal Cells MC0518 Versus Best Available Therapy in Adult and Adolescent Subjects with Steroid refractory Acute Graft versus host Disease After Allogeneic Haematopoietic Stem Cell Transplantation (IDUNN Trial) |
| Hémophilie A | Haemophilia A | Nuwiq Dosing and outcomes In the ManagEment of women/girls with haemophilia A Needing FVIII treatment for Surgery – an International, Open-label, Non-controlled study (NuDIMENSION) |
| Sarcoïdose pulmonaire active | Active Pulmonary Sarcoidosis | A randomized, double-blind, placebo-controlled, multicenter study to assess the efficacy and safety of a 12-week administration of OATD-01, an oral inhibitor of chitinase-1 (CHIT1), for the treatment of active pulmonary sarcoidosis (the KITE study) |
| Malignités à cellules B | B-Cell Malignancies | A Modular Phase I/II Open-label Dose Escalation and Dose Expansion Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of AZD5492, a T cell-engaging Antibody Targeting CD20 in Subjects with Relapsed or Refractory B-Cell Malignancies (TITANium) |
| Thrombocytopénie immunitaire | Immune Thrombocytopenia | A Phase 1/2, Dose-finding Study Investigating the Safety and Efficacy of Pirtobrutinib in Adults with Immune Thrombocytopenia |
| Ostéogenèse imparfaite | Osteogenesis Imperfecta | A Phase 3, Open-Label, Multicenter, Randomized Study to evaluate the Efficacy and Safety of Romosozumab Compared with Bisphophonates in Children and Adolecents with Osteogenesis Imperfecta |
| Malignités solides et hématologiques | Solid and Hematological Malignancies | Long-term Follow-up Study for Participants of Kite-Sponsored Interventional Studies Treated With Gene-Modified Cells |
| Recipients d'une greffe rénale présentant un rejet tardif à médiation immunitaire | Kidney Transplant Recipients with Late Antibody-Mediated Rejection | A Double-Blind, Placebo-Controlled, Multicenter, Randomized Phase 3 Trial Evaluating the Efficacy and Safety of Felzartamab in Kidney Transplant Recipients with Late Antibody-Mediated Rejection (AMR) (TRANSCEND / 299AR301) |
| Myasthénie grave généralisée | Generalized Myasthenia Gravis | ARGX-113-2207: An Open-label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod PH20 SC in Participants From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis |
| Thrombocytopénie immunitaire primaire (ITP) | Primary Immune thrombocytopenia (ITP) | A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Arm Study Followed by an Open-Label Arm to Evaluate the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia |
| Infection chronique par le virus de l'hépatite D (VHD) | Chronic Hepatitis D Virus (HDV) Infection | A Phase 2b Randomized, Open-Label Study to Evaluate the Efficacy and Safety of Tobevibart+Elebsiran Combination Therapy versus Bulevirtide in Participants with Chronic HDV Infection (ECLIPSE 3) |
| Hypophosphatémie liée au chromosome X. | X-linked hypophosphatemia. | A Multicenter, Open-label, Phase 1/2, Dose-escalation and Subsequent Safety Extension Study of Subcutaneous KK8123 in Adult Patients with X-linked Hypophosphatemia |
| Dystrophie myotonique de type 1 | Myotonic Dystrophy Type 1 | A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1 |
| PMM2-CDG | PMM2-CDG | A Phase 2b, Multicenter, Double-blind, Randomized, Placebo controlled Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 Administered Intravenously to Participants with PMM2-CDG |
| Syndrome de Hunter et troubles cognitifs | Hunter Syndrome and Cognitive Impairment | An Open-Label Extension for Subjects in Studies HGT-HIT-046 and SHP609-302 Evaluating Long-Term Safety of Intrathecal Idursulfase-IT Administered in Conjunction with Intravenous Elaprase® in Subjects with Hunter Syndrome and Cognitive Impairment |
| Adénocarcinome de l'intestin grêle | Small bowel adenocarcinoma | PRODIGE 86-FOLFIRINOX SBA- Phase II randomized trial evaluating modified FOLFIRINOX and FOLFOX in the treatment of locally advanced or metastatic small bowel adenocarcinoma |
| Hémiplégie alternante de l'enfance | Alternating Hemiplegia of Childhood | OXYTAHANE - Oxygen therapy: Acute Treatment for paroxysmal dystonic or plegic attacks in alternating hemiplegia of childhood |
| Dysplasie ventriculaire droite arythmogène | Arrhythmogenic right ventricular dysplasia | BLOCKADE OF THE RENIN-ANGIOTENSIN-ALDOSTERONE SYSTEM IN PATIENTS WITH ARVD: A DOUBLE-BLIND MULTICENTRE PROSPECTIVE RANDOMIZED STUDY BRAVE |
| Néphropathie à IgA | IgA Nephropathy | A multicenter rollover extension program (REP) to evaluate the long-term safety and tolerability of open label iptacopan in adult participants with primary IgA nephropathy who have completed a Novartis-sponsored iptacopan parent study in IgAN |
| Mastocytose | mastocytosis | Hydroxychloroquine in isolated cutaneous mastocytosis patients or indolent systemic mastocytosis with associated skin involvement patients: proof of concept study (HCQMa) |
| Achondroplasie chez les enfants et les adolescents | Achondroplasia in Children and Adolescents | teACH: A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo controlled Trial evaluating Efficacy and Safety of Subcutaneous Doses of Navepegritide Administered Once Weekly for 52 Weeks in Adolescents (12-<18 years of age) with Achondroplasia |
| Polycythémie vraie à haut risque JAK2V617F-positive | JAK2V617F-positive high-risk Polycythemia Vera | Randomized, open-label, multicenter phase 3 study to assess the efficacy and safety of GIVinostat versus hydroxyurea IN JAK2V617F-positive high-risk Polycythemia Vera patients: the GIV-IN PV TRIAL |
| Glomérulonéphrite à progression rapide | Rapidly progressive glomerulonephritis | A Randomized, Double-Blind, Placebo-Controlled Study of Intravenously Administered ALE.F02 to Evaluate the Safety, Tolerability, Pharmacokinetics, and Renal Sparing in Antineutrophil Cytoplasmic Antibody Associated Vasculitis With Rapidly Progressive Glomerulonephritis |
| Polykystose rénale autosomique dominante (ADPKD) | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | HYDROchlorothiazide to PROTECT polycystic kidney disease patients and improve their quality of life (HYDRO-PROTECT) |
| Hypertension artérielle pulmonaire | Pulmonary Arterial Hypertension | An Open-label Extension Study Evaluating the Long-term Safety and Efficacy of Seralutinib Orally Inhaled for the Treatment of Pulmonary Arterial Hypertension (PAH) |
| Léiomyosarcome ou liposarcome primaire à haut risque de l'espace rétropéritonéal ou des espaces infra-péritonéaux du bassin | Primary high risk leiomyosarcoma or liposarcoma of retroperitoneal space or infra-peritoneal spaces of pelvis | EORTC-1809-STBSG: A randomized phase III study of neoadjuvant chemotherapy followed by surgery versus surgery alone for patients with High Risk RetroPeritoneal Sarcoma (STRASS 2) |
| Syndrome de Lyell, syndrome de Stevens Johnson | Lyell syndrome, Stevens Johnson syndrome | Evaluating the Therapeutic Efficacy of Filgrastim in Severe Bullous Drug Eruptions (Lyell and Stevens-Johnson Syndromes) |
| Dystrophie myotonique de type 1 (DM1) | Myotonic dystrophy type 1 (DM1) | EVALUATION OF THE EFFICACY AND SAFETY OF METFORMIN IN THE MYOTONIC DYSTROPHY TYPE I (STEINERT’S DISEASE). PHASE III PROSPECTIVE, MULTICENTRE, RANDOMISED, DOUBLE-BLIND CONTROLLED STUDY. |
| Hypersomnie idiopathique (IH) | Idiopathic Hypersomnia (IH) | A Dose-Finding, Adaptive, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Safety, Tolerability, and Efficacy of TAK-360 in Participants with Idiopathic Hypersomnia (IH) |
| Malformations lymphatiques associées à la mutation PIK3CA | lymphatic malformations associated with PIK3CA mutation | A two-stage double-blind, randomized, placebo-controlled study to assess the efficacy, safety and pharmacokinetics of alpelisib in pediatric and adult patients with lymphatic malformations associated with a PIK3CA mutation |
| Myélofibrose | Myelofibrosis | A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat (GRN163L) Versus Best Available Therapy (BAT) in Patients with Intermediate-2 or High-risk Myelofibrosis (MF) Relapsed / Refractory (R/R) to Janus Kinase (JAK)-Inhibitor |
| Atrophie multisystémique | Multiple System Atrophy | Interventional, randomized, double-blind, placebo-controlled, optional open-label extension trial of Lu AF82422 in participants with Multiple System Atrophy |
| Trouble du spectre de la neuromyélite optique (NMOSD ; également connu sous le nom de syndrome de Devic et anciennement sous le nom de neuromyélite optique [NMO]) | Neuromyelitis optica spectrum disorder (NMOSD; also known as Devic's syndrome and previously known as neuromyelitis optica [NMO]) | An Open-label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects with Neuromyelitis Optica Spectrum Disorder |
| Sujets atteints d'une maladie hépatique associée à une insuffisance intestinale (IFALD) | Subjects with Intestinal Failure-Associated Liver Disease (IFALD) | A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Orziloben (NST-6179) in Subjects with Intestinal Failure-Associated Liver Disease (IFALD) |
| Glomérulosclérose segmentaire focale | Focal segmental glomerulosclerosis | A pivotal Phase 3, multicenter, randomized, double-blind, placebo-controlled study of the efficacy and safety of DMX-200 in patients with focal segmental glomerulosclerosis (FSGS) who are receiving an angiotensin II receptor blocker (ARB) |
| Aspergillose pulmonaire invasive réfractaire | Refractory Invasive Pulmonary Aspergillosis | A double-blind, randomized, placebo-controlled study to assess the safety and efficacy of nebulized PC945 when added to systemic antifungal therapy for the treatment of refractory invasive pulmonary aspergillosis (OPERA-T study) |
| Fibrose pulmonaire idiopathique | Idiopathic pulmonary fibrosis | A 26-Week, Randomized, Double-Blind, Placebo-Controlled, Multi-center Study to Evaluate the Efficacy, Safety, and Tolerability of Axatilimab in Subjects with Idiopathic Pulmonary Fibrosis (IPF) |
| Maladie de Pelizaeus-Merzbacher | Pelizaeus-Merzbacher Disease | ION356-CS1: A Phase 1b Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Patients with Pelizaeus-Merzbacher Disease |
| Polymyosite ou dermatomyosite selon les critères de classification EULAR / ACR 2017 | Polymyositis or dermatomyositis according to 2017 EULAR / ACR classification criteria | A Multicenter, Parallel-group, Double-blind, 2-Arm, Phase III Study to Investigate the Efficacy and Safety of Anifrolumab Administered as Subcutaneous Injection and Added to Standard of Care Compared with Placebo Added to Standard of Care in Adult Participants with Idiopathic Inflammatory Myopathies (Polymyositis and Dermatomyositis) |
| Patients atteints de la maladie de Huntington symptomatique et présymptomatique | Patients with symptomatic and pre-symptomatic Huntington's disease | I2BIO-HD. Innovative Imaging and cognitive BIOmarkers to predict Huntington’s Disease progression |
| Maladie chronique du greffon contre l'hôte | chronic graft-versus-host disease | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Axatilimab (INCA034176) and Corticosteroids as Initial Treatment for Chronic Graft-Versus Host Disease |
| Angio-œdème héréditaire | Hereditary Angioedema | A Phase?3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Orally Administered Deucrictibant Extended-release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults with Hereditary Angioedema |
| Polyneuropathie inflammatoire démyélinisante chronique | Chronic Inflammatory Demyelinating Polyneuropathy | A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of DNTH103 in Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CAPTIVATE) |
| Déficit en ectonucléotide pyrophosphatase/phosphodiestérase 1 (ENPP1) | Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency | The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants with Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency |
| Mucopolysaccharidose de type II, syndrome de Hunter | Mucopolysaccharidosis type II, Hunter Syndrome | An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects |
| Mucoviscidose | Cystic Fibrosis | A Phase 3, Open-label Study Evaluating the Long?term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Year of Age and Older |
| Maladie de Fabry | Fabry's disease | Multi-centre, Open-label Trial to Assess the SaFety, Pharmacodynamics, Efficacy and Pharmacokinetics of PegunigaLsidase Alfa in Patients from 2 Years to Less Than 18 Years of Age with Confirmed FabrY Disease (FLY) |
| Néoplasmes rectaux. | Neoplasms, Rectal | A Phase 2, Single-Arm, Open-Label Study with Dostarlimab Monotherapy in Participants with Untreated Stage II/III dMMR/MSI-H Locally Advanced Rectal Cancer |
| Abcès cérébral | Brain abscess | Partial oral antibiotic treatment for bacterial brain abscess: An open-label randomised non-inferiority trial (ORAL) |
| Thrombasténie de Glanzmann | Glanzmann Thrombasthenia | A Phase 1/2, First-in-Human, Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants With Glanzmann Thrombasthenia |
| Maladie de von Willebrand héréditaire sévère chez les enfants | Hereditary severe von Willebrand Disease in children | A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF with or without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed with Severe von Willebrand Disease |
| Myélofibrose post-polycythémie vraie. Myélofibrose primaire. Myélofibrose post-thrombocytémie essentielle. | Post-Polycythaemia Vera Myelofibrosis, Primary Myelofibrosis, Post-Essential Thrombocythaemia Myelofibrosis | PACIFICA Phase 3: A Randomized, Controlled Phase 3 Study of Pacritinib Versus Physician’s Choice in Patients with Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post Essential Thrombocythemia Myelofibrosis with Severe Thrombocytopenia (Platelet Counts <50,000/?L) |
| Syndrome d'Angelman | Angelman Syndrome | A Long-Term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients with Angelman Syndrome |
| Insuffisance intestinale associée au syndrome de l'intestin court (SBS-IF) | Short Bowel Syndrome-associated Intestinal Failure (SBS-IF) | A Multicenter, Proof-of-concept, Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of HM15912 in Adult Subjects with Short Bowel Syndrome-associated Intestinal Failure (SBS-IF) (DOLPHINS-2) |
| Myopathies inflammatoires idiopathiques actives (y compris la dermatomyosite ou la polymyosite) | Active Idiopathic Inflammatory Myopathies (Including Dermatomyositis or Polymyositis) | C0251006 - A PHASE 3, MULTICENTER, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF PF-06823859 IN PARTICIPANTS WITH ACTIVE IDIOPATHIC INFLAMMATORY MYOPATHIES (INCLUDING PARTICIPANTS WITH ACTIVE DERMATOMYOSITIS OR POLYMYOSITIS) |
| Maladies associées aux anticorps anti-glycoprotéine oligodendrocytaire de la myéline (MOGAD) | myelin oligodendrocytes glycoprotein antibody associated diseases (MOGAD) | IDAR.Immediate versus delayed treatment with azathioprine or rituximab in anti-myelin oligodendrocytes glycoprotein (anti-MOG) antibodies associated acute demyelinating syndromes in children: a randomized controlled clinical trial. |
| Télangiectasie maculaire de type 1 | macular telangiectasia type 1 | TELeMAC - Comparative multicenter randomized study of aflibercept versus placebo in macular telangiectasia type 1 |
| Valve postérieure urétrale | urethral posterior valve | PROSPECTIVE RANDOMIZED CLINICAL TRIAL OF EARLY OXYBUTININ TREATMENT FOR BOYS WITH POSTERIOR URETHRAL VALVES - PRETIPUV - |
| Mucoviscidose | Cystic Fibrosis | A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX 121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age (VX21-121-105) |
| Indications multiples | Multiple indications | An open label, multi-center roll-over study to assess long-term safety in patients who are ongoing or have completed a prior global Novartis or GSK sponsored Tafinlar (dabrafenib) and/or Mekinist (trametinib) study and are judged by the investigator to benefit from continued treatment. |
| Spectre de croissance excessive lié à PIK3CA (PROS) | PIK3CA-Related Overgrowth Spectrum (PROS) | EPIK-P2: A Phase II double-blind study with an upfront, 16-week randomized, placebo-controlled period, to assess the efficacy, safety and pharmacokinetics of alpelisib (BYL719) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS) |
| Sclérose systémique (SSc) se manifestant par des sous-ensembles cutanés limités ou diffus, maladies du système immunitaire, pneumopathie interstitielle (ILD), maladies auto-immunes | Systemic Sclerosis (SSc) presenting with Limited or Diffuse Cutaneous Subsets, Immune system diseases, Interstitial lung disease (ILD), Autoimmune diseases | A Multicenter, Randomized, Parallel-group, Double-blind, Two-arm, Phase III Study to Evaluate the Safety and Efficacy of Anifrolumab Compared with Placebo in Male and Female Participants 18 to 70 Years of Age Inclusive with Systemic Sclerosis |
| Myasthénie grave généralisée | Generalized Myasthenia Gravis | Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Nipocalimab Administered to Adults with Generalized Myasthenia Gravis |
| Néoplasmes/syndromes myélodysplasiques (MDS) | myelodysplastic neoplasms/syndromes (MDS) | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elritercept (KER-050) for the Treatment of Transfusion-Dependent Anemia in Adult Participants with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) (RENEW) |
| Épidermolyse bulleuse simple généralisée (EBS) | Generalized Epidermolysis Bullosa Simplex (EBS) | An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study with Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1% Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS) [EBShield Study] |
| Myasthénie grave généralisée | Generalized Myasthenia Gravis | ARGX-113-2008: A Long-term, Single-Arm, Open-label, Multicenter Trial to Evaluate Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis |
| Myasthénie grave généralisée | Generalized Myasthenia Gravis | Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis |
| Maladie associée aux anticorps anti-glycoprotéine oligodendrocytaire de la myéline (MOG-AD) | Myelin oligodendrocyte glycoprotein antibody-associated disease (MOG-AD) | A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Phase 3, Pivotal Study With an Open-Label Extension Period to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Myelin Oligodendrocyte Glycoprotein (MOG) Antibody-Associated Disease (MOG-AD) |
| Comportement lié à l'hyperphagie associé au syndrome de Prader-Willi (PWS) | Hyperphagia related behaviour associated with Prader-Willi Syndrome (PWS) | A Long-term, Open-label Extension Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome |
| Cholangite sclérosante primitive (PSC) | Primary Sclerosing Cholangitis (PSC) | An Open-label study of patients with primary sclerosing cholangitis (PSC) treated with norucholic acid tablets |
| Hémophilie B | Hemophilia B | A Two-Part Open-Label Study of REGV131-LNP1265, A CRISPR/CAS9-Based Coagulation Factor IX Gene Insertion Therapy in Participants with Hemophilia B |
| Rétinite pigmentaire | Retinitis Pigmentosa | A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects with Retinitis Pigmentosa. |
| Infections fongiques invasives dues à Aspergillus spp. | Invasive fungal infections due to Aspergillus spp. | A Phase III, adjudicator-blinded, randomised study to evaluate the efficacy and safety of treatment with olorofim versus treatment with AmBisome® followed by standard of care (SOC) in patients with invasive fungal disease (IFD) caused by Aspergillus species |
| Mucopolysaccharidose de type II [MPS II] | Mucopolysaccharidosis Type II [MPS II] | An Open-Label Extension to Investigate the Long-Term Safety, Tolerability, and Efficacy of DNL310 in Patients With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 |
| Sclérose systémique | Systemic Sclerosis | A Randomized, Double-Blinded, Placebo-Controlled, Phase 2, Parallel-Group Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Adult Participants With Systemic Sclerosis |
| Mucoviscidose | Cystic fibrosis | A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate Nebulized Bacteriophage Treatment in Outpatient Adult Cystic Fibrosis (CF) Subjects with Chronic Pseudomonas aeruginosa (PsA) Pulmonary Infection |
| Perte auditive bilatérale sévère à profonde due à un déficit en otoferline. | Bilateral severe to profound hearing loss due to Otoferlin deficiency. | A Phase I/II, open-ended, adaptative, open label dose escalation and expansion clinical trial to evaluate the efficacy and safety of unilateral intracochlear injection of SENS-501 using an injection system in children with severe to profound hearing loss due to Otoferlin gene mutations |
| Dermatomyosite ; baricitinib chez les patients atteints de dermatomyosite récidivante ou naïve. | Dermatomyositis; Baricitinib in patients with relapsing or naïve dermatomyositis. | BIRD - Baricitinib in patients with relapsing or naive Dermatomyositis |
| Encéphalomyopathie mitochondriale, acidose lactique et épisodes de type accident vasculaire cérébral (MELAS) | Mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS) | A Multi-Center, Randomized, Single-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral TTI-0102 for Treatment of Patients with Mitochondrial encephalomyopathy, lactic acidosis and strokelike episodes (MELAS). |
| Vascularite associée aux anticorps anti-cytoplasme des neutrophiles (ANCA) actifs | active antineutrophilic cytoplasmic antibody (ANCA) associated vasculitis | A randomized, controlled study to evaluate LNP023 (iptacopan) in patients with active ANCA-associated vasculitis. |
| Patients immunodéprimés atteints de LEMP (avec une cause sous-jacente d'immunosuppression difficilement réversible, c'est-à-dire ni les patients atteints du VIH ni ceux recevant des produits biologiques pour des maladies inflammatoires chroniques). | Immunocompromised patients with PML (with an underlying cause of immunosuppression hardly reversible, i.e. not the patients with HIV nor those receiving biologics for chronic inflammatory diseases). | Pembrolizumab in progressive multifocal leukoencephalopathy (PML) in immunocompromised patients without HIV infection (PENALTY) |
| Achondroplasie | Achondroplasia | Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL OLE |
| Polyneuropathie inflammatoire démyélinisante chronique | Chronic Inflammatory Demyelinating Polyneuropathy | A Phase 3, randomized, double-blind study evaluating efficacy and safety of riliprubart versus intravenous immunoglobulin (IVIg) in participants with chronic inflammatory demyelinating polyneuropathy |