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NCT06092034 · ClinicalTrials.gov registry record · Phase 2
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
A Phase 2 study of Danon Disease, sponsored by Rocket Pharmaceuticals.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 14
- Enrollment target
- 6
- Study locations
NCT06092034: Recruiting Phase 2 study of Danon Disease, sponsored by Rocket Pharmaceuticals.
NCT06092034 is a Phase 2 study of Danon Disease that is actively recruiting participants, run by Rocket Pharmaceuticals. The registered enrollment target is 14 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (89% lower). The trial reports 6 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06092034, a Phase 2 study of Danon Disease, is actively recruiting participants, sponsored by Rocket Pharmaceuticals.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 14 participants
- Enrollment target
- 6
- Study locations
Study Summary
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.
Primary Outcome
Increase of myocardial tissue expression of LAMP2 protein and decrease in left ventricular mass index (LVMI).
Conditions Studied
Interventions
- GENETIC RP-A501
Study Locations (6)
Other
- German Heart Center Munich - Munich
- Meyer Childrens Hospital - Florence
- Great Ormund Street Hospital & UCL Institute of Cardiovascular Science - London
California
- University of California, San Diego - La Jolla
Massachusetts
- Boston Children's Hospital - Boston
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 14 participants |
| Start Date | 2023-09-05 |
| Est. Completion | 2032-04 |
| Phase | Phase 2 |
What NCT06092034 shows while recruiting
NCT06092034 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 14 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (89% lower).
The record links to 1 condition, with Danon Disease appearing as the primary indexed condition, and to 1 intervention - of which RP-A501 is the first listed.
NCT06092034 lists 6 locations in 4 states (Other, California, Massachusetts).
Frequently Asked Questions
What is clinical trial NCT06092034 about?
NCT06092034 is a clinical study titled "A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease". This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.
What is the current status of trial NCT06092034?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 14 participants. The study started on 2023-09-05. Estimated completion is 2032-04.
What conditions does trial NCT06092034 study?
This clinical trial studies the following conditions: Danon Disease.
What interventions are being tested in trial NCT06092034?
The interventions under investigation include: RP-A501 (GENETIC).
Who is sponsoring clinical trial NCT06092034?
This trial is sponsored by Rocket Pharmaceuticals, which has 10 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06092034 being conducted?
This trial has 6 study locations across California, Massachusetts, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Danon Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06092034's enrollment target sits among peer trials
14 1942nd of 2000 higher than 54 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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