32. 自己貪食空胞性ミオパチー
［臨床試験数：1，薬物数：1（DrugBank：0），標的遺伝子数：0，標的パスウェイ数：0］
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. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | NCT03882437 (ClinicalTrials.gov) | April 17, 2019 | 12/3/2019 | 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. | NULL | Recruiting | 8 Years | N/A | Male | 24 | Phase 1 | United States |