NCT06092034Phase 2Recruiting

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

Study sponsor: Rocket Pharmaceuticals Inc. · RCKT

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2028-04Estimated

Enrollment · participants14Estimated

Source updated2026-05-04Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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Posted 2025-10-07 · 5 fields changed

Primary outcomes · main study measures

Text edit · unverified significance

− Previous value

measure: Evaluation of efficacy associated with RP-A501 via co-primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index

+ Updated value

measure: Evaluation of efficacy via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index

The saved outcome text may be shortened. Check the source for full wording.

Participant count type

Recorded edit

− Previous value

Actual

+ Updated value

Estimated

Enrollment · participant count

Recorded edit

− Previous value

count: 12; type: ACTUAL

+ Updated value

count: 14; type: ESTIMATED

Primary completion date

Recorded edit

− Previous value

2025-09

+ Updated value

2028-04

Recruitment status

Recorded edit

− Previous value

Active not recruiting

+ Updated value

Recruiting

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