Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
← catalyst calendarSponsored by Rocket Pharmaceuticals Inc. (industry) · RCKT — their whole pipeline →.
Phase
Phase 1
Status
Unknown status
Study type
Interventional
Design
Na · None · Treatment
Enrollment
7actual
Sites
3
Country
United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2019-04-17 | actual | When the study began enrolling |
| Primary completion | 2025-03-24 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2025-03-24 | estimated | The whole study's end, after follow-up |
| First posted | 2019-03-20 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2023-03-03 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Danon Disease
Intervention
- Biological: RP-A501
Primary outcomes
what the primary-completion date above is the date OFNumber of participants with treatment-related adverse events as assessed by United States (US) National Cancer Institute Common Terminology Criteria (NCI CTCAE)
measured 3 years
Number of participants within each dose level cohort with treatment-related adverse events as assessed by United States (US) National Cancer Institute Common Terminology Criteria (NCI CTCAE)
measured 3 years
Evaluation of cardiomyocyte histologic correction following administration of RP-A501 via endomyocardial biopsy
measured 3 years
Preliminary evaluation of clinical stabilization of cardiomyopathy following administration of RP-A501 via cardiopulmonary testing
measured 3 years
Publications
- PMID 39556016 — linked by the registry
- PMID 39302117 — linked by the registry
- PMID 36288619 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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