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22 Ergebnis(se)
Rekrutierende Studie = 
; Fortlaufende Studie = 
; Förderung durch ein IRDiRC-Mitglied = 
; ERN-Mitglied = 
Nationale klinische Studie(n)
FRANKREICH
ILE-DE-FRANCE
EVRY
Treatment of dysphagia due to Oculo-Pharyngeal Muscular Dystrophy (OPMD) by autologous transplantation of myoblasts (Phase I-II) - FR
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
Clinical trial on the effect of bezafibrate in the muscular form of Carnitine Palmitoyl Transférase 2 (CPT2) deficiency (Phase III) - Terminated -
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
Study of autologous cellular therapy tolerance and feasibility in facioscapulohumeral muscular dystrophy (Phase I-II) - FR
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid (Phase II)
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
Gene therapy development in X-linked Adrenoleukodystrophy for clinical trials
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
MYOMET: A randomized, double blind, placebo-controlled phase II study of metformin in myotonic dystrophy type 1 patients - FR
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth disease type 1A treated 15 months (PLEO-CMT) - FR
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
A phase I/II, open label study of intracerebral administration of adeno-associated viral vector containing the human alpha-N-acetylglucosaminidase (NAGLU) cDNA in children with Sanfilippo type B syndrome - FR
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
NEBIDYS : A Randomized, Double-Blind, Placebo-Controlled, Multi-center Study to Examine the Effect of Nebivolol, a Beta-Blockade Drug, for the Prevention of Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy (Phase III)
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
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
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
RAPAMI : Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis (Phase II-III)
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
WAS FUP : 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) - FR
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene (Phase I-II)
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - FR
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
MSC-LES: Treatment of severe refractory systemic lupus erythematosus by injection of allogeneic mesenchymal stem cells derived from the umbilical cord
AFM Téléthon - Association Française contre les Myopathies
Multinationale klinische Studie(n)
FRANKREICH
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Rim4DMD: A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy
Institution: Information not provided - FR
FRANKREICH
ILE-DE-FRANCE
EVRY
A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
Rim4DMD: A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
CareCN: A Phase I/II, Open Label, Escalating Dose Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy.
AFM Téléthon - Association Française contre les Myopathies
FRANKREICH
ILE-DE-FRANCE
EVRY
CareCN: A Phase I/II, Open Label, Escalating Dose Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy.
Généthon
Centre de Recherche Généthon
SCHWEIZ
Suisse Romande
GENÈVE
Rim4DMD: A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy
Campus Biotech Innovation park
EspeRare Foundation
VEREINIGTE STAATEN
Massachusetts
CAMBRIDGE