113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details
臨床試験数 : 646 / 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170
| 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 | EUCTR2019-001825-28-GB (EUCTR) | 24/09/2019 | 29/07/2019 | Mesoangioblast-based gene therapy for Duchenne Muscular Dystrophy: a phase I/IIa study / DMD04 | Mesoangioblast-mediated exon 51 skipping for genetic correction of dystrophin, based upon a single injection in individual skeletal muscles of five non ambulant patients affected by Duchenne Muscular Dystrophy: a non randomized, open label, phase I/IIa study. - Mesoangioblast-based gene therapy for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Lentiviral Vector Trans-Skip gene modified Autologous Mesoangioblasts | The University of Manchester | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 5 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom |