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22 Resultaat/Resultaten
Aanwerving proef
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Nationale klinische studie(s)
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
ILE-DE-FRANCE
EVRY
Gene therapy development in X-linked Adrenoleukodystrophy for clinical trials
AFM Téléthon - Association Française contre les Myopathies
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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(s)
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
FRANKRIJK
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
USA
Massachusetts
CAMBRIDGE
ZWITSERLAND
Suisse Romande
GENÈVE