Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
Public ClinicalTrials.gov record NCT03882437. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Clinical Study Evaluating a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B) Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With DD
Study identification
- NCT ID
- NCT03882437
- Recruitment status
- Not listed
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Enrollment
- 7 participants
Conditions and interventions
Conditions
Interventions
- RP-A501 Biological
Biological
Eligibility (public fields only)
- Age range
- 8 Years and older
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 16, 2019
- Primary completion
- Mar 23, 2025
- Completion
- Mar 23, 2025
- Last update posted
- Mar 2, 2023
2019 – 2025
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California, San Diego | La Jolla | California | 92037 | — |
| University of Colorado | Aurora | Colorado | 80045 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03882437, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 2, 2023 · Synced Jul 21, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03882437 live on ClinicalTrials.gov.