113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details
臨床試験数 : 622 / 薬物数 : 485 - (DrugBank : 99) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 168
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2021-004276-33-DK (EUCTR) | 11/02/2022 | 01/10/2021 | GNT0006 gene therapy trial in patients with limb-girdle muscular dystrophy | A 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 Therapeutics | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Phase 1;Phase 2 | France;United States;Canada;Denmark;Norway;Germany;United Kingdom;Sweden |