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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. 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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06092034
The ClinicalTrials.gov registry entry for NCT06092034 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 14 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Rocket Pharmaceuticals, which has 10 total studies on file at ClinicalTrials.gov.
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 reports 6 study locations spanning 4 distinct geographic areas - top geographies include 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.
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Related
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