Actively Recruiting
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Led by Rocket Pharmaceuticals Inc. · Updated on 2026-05-04
14
Participants Needed
6
Research Sites
447 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male patients
- Age 8 years or older
- Documented pathogenic or likely pathogenic variant of the LAMP2 gene
- Evidence of left ventricular hypertrophy with abnormal thickening of the left ventricular wall
- Left ventricular ejection fraction (LVEF) of 50% or higher
- New York Heart Association (NYHA) Class II to III
- High sensitivity Troponin I (hsTnI) level at least 20% above the upper limit of normal
- Ability to comply with study procedures including investigational 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 including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction, or unstable angina
- Prior cardiac or other organ transplantation such as lung or liver
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here