32. Autophagic vacuolar myopathy
[
1 clinical trial,
1 drug（DrugBank:
0 drug),
0 target gene / 0 target pathway ]
Searched query = "Autophagic vacuolar myopathy", "Danon disease", "X linked myopathy with excessive autophagy", "XMEA"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03882437 | April 17, 2019 | 15 July 2019 | Gene Therapy for Male Patients With Danon Disease Using RP-A501; AAV9.LAMP2B | Gene Therapy for Danon Disease (DD): A Clinical Study Evaluating the Infusion of a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B) Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease | Danon Disease | Biological: RP-A501 | Rocket Pharmaceuticals Inc. | Recruiting | 8 Years | N/A | Male | 24 | Phase 1 | United States |