Drugs / RP-A501
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT03882437 | Apr 2019 → Mar 2025 | Danon disease | Rocket Pharmaceuticals Inc. | Unknown | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT06092034 | Sep 2023 → Apr 2028 expected | Danon disease | Rocket Pharmaceuticals Inc. | Recruiting | No outcome recorded |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | RP-A501 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03882437 ↗ |
| Known as | AAV9.LAMP2B | “Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the...” NCT06092034 ↗1“A Clinical Study Evaluating a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B)...” NCT03882437 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “RP-A501 is a gene therapy product consisting of a rAAV9 capsid containing the human LAMP2B transgene” NCT03882437 ↗ |
| Route | Intravenous | “a single intravenous (IV) infusion” NCT03882437 ↗ |
| Target | LAMP2 | “containing the human LAMP2B transgene” NCT06092034 ↗ |