32. 自己貪食空胞性ミオパチー Autophagic vacuolar myopathy Clinical trials / Disease details
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
| 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 (DD) Using RP-A501; AAV9.LAMP2B | A Clinical Study Evaluating 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 DD | Danon Disease | Biological: RP-A501 | Rocket Pharmaceuticals Inc. | NULL | Active, not recruiting | 8 Years | N/A | Male | 7 | Phase 1 | United States |