111. Congenital myopathy Clinical trials / Disease details
Clinical trials : 10 / Drugs : 17 - (DrugBank : 5) / Drug target gene : 1 - Drug target pathways : 9
| 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 | EUCTR2017-000876-27-DE (EUCTR) | 22/05/2018 | 22/09/2017 | First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) Patients | ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients - ASPIRO | X-linked Myotubular Myopathy (XLMTM) MedDRA version: 20.0;Level: HLGT;Classification code 10029317;Term: Neuromuscular disorders;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: rAAV8-Des-hMTM1 Product Code: AT132 | Audentes Therapeutics Inc. | NULL | Not Recruiting | Female: no Male: yes | 26 | Phase 1;Phase 2 | France;United States;Canada;Germany;United Kingdom | ||
| 2 | EUCTR2017-000876-27-FR (EUCTR) | 23/04/2018 | 25/05/2018 | First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) Patients | ASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients - ASPIRO | X-linked Myotubular Myopathy (XLMTM);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: rAAV8-Des-hMTM1 Product Code: AT132 | Audentes Therapeutics Inc. | NULL | Not Recruiting | Female: no Male: yes | 12 | Phase 1;Phase 2 | United States;France;Germany;United Kingdom | ||
| 3 | EUCTR2017-000876-27-GB (EUCTR) | 06/12/2017 | 31/07/2017 | First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) Patients | ASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients - ASPIRO | X-linked Myotubular Myopathy (XLMTM);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: rAAV8-Des-hMTM1 Product Code: AT132 | Audentes Therapeutics Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 20 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Germany;United Kingdom | ||
| 4 | NCT03199469 (ClinicalTrials.gov) | August 2, 2017 | 21/6/2017 | Gene Transfer Clinical Study in X-Linked Myotubular Myopathy | ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients | X-Linked Myotubular Myopathy | Genetic: AT132 | Astellas Gene Therapies | NULL | Active, not recruiting | N/A | 5 Years | Male | 26 | Phase 1/Phase 2 | United States;Canada;France;Germany |