Rp-a501 (DrugBank: -)
1 disease| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 32 | 自己貪食空胞性ミオパチー | 1 |
| 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 |