113. Muscular dystrophy Clinical trials / Disease details


Clinical trials : 622 Drugs : 485 - (DrugBank : 99) / Drug target genes : 59 - Drug target pathways : 168

  
1 trial found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2021-004276-33-DK
(EUCTR)
11/02/202201/10/2021GNT0006 gene therapy trial in patients with limb-girdle muscular dystrophyA phase 1-2 multicenter study (2 stages) to evaluate the safety and efficacy of intravenous GNT0006, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle muscular dystrophy (LGMD R9, formerly LGMD2I) FKRP-related limb-girdle muscular dystrophy (LGMD R9)
MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: rAAV9-hFKRPco_miR-208a
Product Code: GNT0006
INN or Proposed INN: not yet available
Other descriptive name: Adeno-associated virus serotype 9 carrying the human fukutin-related protein and target sequence of the miR-208a
Atamyo TherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
39Phase 1;Phase 2France;United States;Canada;Denmark;Norway;Germany;United Kingdom;Sweden