Status:
RECRUITING
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Lead Sponsor:
Rocket Pharmaceuticals Inc.
Conditions:
Danon Disease
Eligibility:
MALE
8+ years
Phase:
PHASE2
Brief 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 is...
Detailed Description
The study is a single arm Phase 2 clinical trial to characterize the safety and efficacy of RP-A501, a recombinant adeno-associated serotype 9 (rAAV9 capsid containing the human lysosome-associated me...
Eligibility Criteria
Inclusion
- Key
- 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)
- Ability to comply with study procedures including investigational therapy and follow-up evaluations.
- Key
Exclusion
- Anti-AAV9 neutralizing antibody titer \>1:40.
- Severe heart failure or requirement for advanced therapies.
- History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.
- Prior cardiac or other organ (lung, liver, other) transplantation.
Key Trial Info
Start Date :
September 5 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 1 2032
Estimated Enrollment :
14 Patients enrolled
Trial Details
Trial ID
NCT06092034
Start Date
September 5 2023
End Date
April 1 2032
Last Update
October 7 2025
Active Locations (6)
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1
University of California, San Diego
La Jolla, California, United States, 92037
2
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
3
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
4
German Heart Center Munich
Munich, Germany