Ongoing trials

The Institute of Myology leads cutting-edge clinical trials for neuromuscular diseases, in partnership with industry laboratories and academic researchers.

Below you will find the list of ongoing trials at the Institute of Myology

5 clinical trials in progress

GNT-016-MDYF

A clinical trial of gene therapy with a microdystrophin (GNT0004) in DMD: a gene therapy study using a microdystrophin for the treatment of Duchenne muscular dystrophy.

Duchenne muscular dystrophy I-Motion Pediatric Pending

A clinical trial of gene therapy with a microdystrophin (GNT0004) in DMD: a gene therapy study using a microdystrophin for the treatment of Duchenne muscular dystrophy.

Code: GNT-016-MDYF
Disease: Duchenne muscular dystrophy
Principal investigator: Silvana De Lucia
Sponsor: Genethon
Status: Pending
Public: Paediatric
Trial site: I-Motion Pediatric

MIS51ON

A randomised, double-blind, dose-finding and dose-comparison study of eteplirsen in DMD, evaluating the safety and efficacy of high-dose eteplirsen in patients with DMD with deletion mutations amenable to exon 51 skipping.

Duchenne muscular dystrophy I-Motion Pediatric Active

Code: MIS51ON
Disease: Duchenne muscular dystrophy
Principal investigator: Andreea Seferian
Sponsor: SAREPTA
Status: Active
Public: Paediatric
Trial site: I-Motion Pediatric

View on ClinicalTrials.gov

Italfarmaco 51

An open-label study of the long-term safety, tolerability and efficacy of givinostat in DMD: a study evaluating the long-term safety, tolerability and efficacy of givinostat in all previously treated patients with Duchenne muscular dystrophy.

Duchenne muscular dystrophy I-Motion Pediatric Active

Code: Italfarmaco-51
Disease: Duchenne muscular dystrophy
Principal investigator: Odile Boespflug-Tanguy
Sponsor: Italfarmaco
Status: Active
Public: Paediatric
Trial site: I-Motion Pediatric

View on ClinicalTrials.gov

GNT-014-MDYF – Natural history of DMD

A prospective, interventional, reference study examining the natural history of DMD in young male patients aged 4 to 6 years.

Duchenne muscular dystrophy I-Motion Pediatric Active

A prospective, interventional, reference study examining the natural history of DMD in young male patients aged 4 to 6 years.

Code: GNT-014-MDYF
Disease: Duchenne muscular dystrophy
Principal investigator: Silvana De Lucia
Sponsor: Genethon
Status: Active
Public: Paediatric
Trial site: I-Motion Pediatric

View on ClinicalTrials.gov

UMD-DMD – Dystrophinopathy database

Dystrophinopathy database bringing together the French molecular diagnostic laboratories and all the neuromuscular reference and competence centres.

Duchenne muscular dystrophy Ongoing

Dystrophinopathy database bringing together the French molecular diagnostic laboratories and all the neuromuscular reference and competence centres.

Code: UMD-DMD
Disease: Duchenne muscular dystrophy
Principal investigator: Rabah Ben Yaou / France Leturcq / Sylvie Tuffery-Giraud
Sponsor: Laboratoires francais de diagnostic moleculaire des dystrophinopathies + tous les centres de reference et competence MNM
Status: Ongoing
Public: Adults, Paediatric