Actively Recruiting
Gene Therapy for Danon Disease: Evaluating RP-A501 (AAV9.LAMP2B) Safety and Efficacy in Males
Led by Rocket Pharmaceuticals Inc. · Updated on 2026-05-04
14
Participants Needed
6
Research Sites
208 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the safety and effectiveness of RP-A501, a gene therapy using a modified virus to deliver the LAMP2B gene, in male patients 8 years and older with Danon Disease. This single-arm Phase 2 trial focuses on males who have a genetic variant of the LAMP2 gene and heart changes related to the disease. The goal is to understand how well this gene therapy works and how safe it is for this group of patients. Participants will receive one intravenous infusion of RP-A501, a gene therapy product made of a modified adeno-associated virus containing the human LAMP2B gene. This single dose is given during the treatment period, and patients are monitored over time to assess the therapy's effects. The study does not include a comparison group and focuses solely on this treatment. During the study, participants will be evaluated at regular intervals up to 60 months after infusion. Assessments include measuring LAMP2 protein expression in heart tissue, heart size and function via left ventricular mass index, and biomarkers indicating heart injury such as high sensitivity Troponin I and NT-proBNP. The study also tracks event-free survival and safety outcomes. Patients must be able to comply with study procedures and attend follow-up visits throughout the trial.
CONDITIONS
Brief Title
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene
- Male gender
- Age 8 years or older
- Evidence of left ventricular hypertrophy with abnormal thickening of the left ventricular wall
- Left ventricular ejection fraction (LVEF) 50% or higher
- New York Heart Association (NYHA) Class II to III
- High sensitivity Troponin I (hsTnI) 20% above the upper limit of normal
- Ability to comply with study procedures including therapy and follow-up evaluations
You will not qualify if you...
- Anti-AAV9 neutralizing antibody titer greater than 1:40
- Severe heart failure or need for advanced therapies
- History of intracardiac thrombosis or arterial thromboembolic events such as stroke, transient ischemic attack, acute coronary syndrome, myocardial infarction, or unstable angina
- Prior cardiac or other organ transplantation (lung, liver, or other)
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single day
Participants receive a single intravenous infusion of the gene therapy RP-A501.
1 infusion visit (in-person)
Duration - Up to 60 months post-infusion
Participants are monitored for safety and efficacy through assessments including myocardial tissue expression, left ventricular mass, biomarkers, and event-free survival.
Multiple visits over 60 months for follow-up assessments
Trial Site Locations
Total: 6 locations
1
University of California, San Diego
La Jolla, California, United States, 92037
Actively Recruiting
2
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
3
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
4
German Heart Center Munich
Munich, Germany
Actively Recruiting
5
Meyer Childrens Hospital
Florence, Italy
Actively Recruiting
6
Great Ormund Street Hospital & UCL Institute of Cardiovascular Science
London, United Kingdom
Actively Recruiting
Research Team
C
Clinical Information
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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