for males ages 8 years and up (full criteria)
study started
completion around
Principal Investigator
by Barry Greenberg, MD (ucsd)
Headshot of Barry Greenberg
Barry Greenberg



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.

Official Title

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


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 membrane protein 2 isoform B (LAMP2B) transgene) in male patients with Danon Disease.

Male subjects ≥8 years of age will receive a single intravenous infusion of RP-A501.


Danon Disease, Hypertrophic Cardiomyopathy, HCM, X-linked disease, LAMP2, Pediatric, Skeletal Myopathies, Cardiomyopathy, Lysosomal Storage Disorder, Left Ventricular Hypertrophy, Glycogen Storage Disease Type IIb


You can join if…

Open to males ages 8 years and up

  1. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
  2. Male.
  3. Age ≥8 years.
  4. Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following:
    1. Abnormal thickening of Left ventricular wall,
    2. Left ventricular ejection fraction (LVEF) ≥ 50%.
  5. New York Heart Association (NYHA) Class II to III.
  6. High sensitivity Troponin I (hsTnI) ≥20% above the upper limit of normal (ULN)
  7. Ability to comply with study procedures including investigational therapy and follow-up evaluations.

You CAN'T join if...

  1. Anti-AAV9 neutralizing antibody titer >1:40.
  2. Severe heart failure or requirement for advanced therapies.
  3. History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.
  4. Prior cardiac or other organ (lung, liver, other) transplantation.


  • University of California, San Diego accepting new patients
    La Jolla California 92037 United States
  • Children's Hospital of Philadelphia accepting new patients
    Philadelphia Pennsylvania 19104 United States
  • Great Ormund Street Hospital & UCL Institute of Cardiovascular Science not yet accepting patients
    London United Kingdom
  • German Heart Center Munich accepting new patients
    Munich Germany

Lead Scientist at University of California Health

  • Barry Greenberg, MD (ucsd)
    Professor, Medicine, Vc-health Sciences-schools. Authored (or co-authored) 19 research publications


accepting new patients
Start Date
Completion Date
Rocket Pharmaceuticals Inc.
Phase 2 Danon Disease Research Study
Study Type
Expecting 12 study participants
Last Updated