A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
← catalyst calendarNCT06092034 · readout ≤ 620 d
Sponsored by Rocket Pharmaceuticals Inc. (industry) · RCKT — their whole pipeline →.
Phase
Phase 2
Status
Recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
14estimated
Sites
6
Countries
Germany, Italy, United Kingdom +1
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 | 2023-09-05 | actual | When the study began enrolling |
| Primary completion | Apr 2028 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Apr 2032 | estimated | The whole study's end, after follow-up |
| First posted | 2023-10-23 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-05-04 | 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
- Genetic: RP-A501
Primary outcome
what the primary-completion date above is the date OFEvaluation of efficacy via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index
measured 12 Months post-infusion
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