NCT06092034RecruitingPhase 2
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the Human LAMP2 Isoform B Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease
Source: ClinicalTrials.gov · Last updated 6 Oct 2026
Brief summary (registry, in English)
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon disease.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 18 (estimated)
- First submitted
- 11 Oct 2023
- Start date
- 5 Sept 2023
- Primary completion date
- Jun 2028
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Diseases
- Hypertrophic cardiomyopathy
- NCT number
- NCT06092034
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 18
- From 5 Sept 2023 to the primary completion date, Jun 2028
- 6 sites in 4 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Anti-AAV9 neutralizing antibody titer \>1:40.
- Severe heart failure or requirement for advanced therapies (requirement for intravenous inotropic, vasodilator, or diuretic therapy within the 30 days prior to enrollment, presence or requirement for mechanical circulatory support (MCS), and presence or requirement for mechanical ventilation).
- Prior cardiac or other organ (lung, liver, other) transplantation.
Other illness
- History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.
Who the study is looking for
- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
- Male.
- Age ≥8 years.
- Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following:
- Abnormal thickening of left ventricular wall,
- Left ventricular ejection fraction (LVEF) ≥ 50%.
- New York Heart Association (NYHA) Class II to III.
- High sensitivity Troponin I (hsTnI) ≥20% above the upper limit of normal (ULN) ≤ 12 months prior to screening.
- Ability to comply with study procedures including investigational therapy and follow-up evaluations.
Age in the registry: 8 years to - · Sex: Male · Does not accept healthy volunteers
Sites (6)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| German Heart Center Munich Coordinator of this site? Confirm its status | Munich | Germany | Recruiting |
| Great Ormond Street Hospital & UCL Institute of Cardiovascular Science Coordinator of this site? Confirm its status | London | United Kingdom | Recruiting |
| Meyer Children's Hospital Coordinator of this site? Confirm its status | Florence | Italy | Recruiting |
| Boston Children's Hospital Coordinator of this site? Confirm its status | Boston | United States | Recruiting |
| University of California San Diego Coordinator of this site? Confirm its status | La Jolla | United States | Recruiting |
| Children's Hospital of Philadelphia Coordinator of this site? Confirm its status | Philadelphia | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.