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Virus Therapy

Gene Therapy for Danon Disease

Boston, MA
Phase 2
Waitlist Available
Led By Joseph Rossano, MD
Research Sponsored by Rocket Pharmaceuticals Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age ≥8 years
New York Heart Association (NYHA) Class II to III
Must not have
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 60 months post infusion
Awards & highlights
No Placebo-Only Group

Summary

This trial tests a new treatment for Danon Disease in male patients to see if it is effective and safe.

Who is the study for?
This trial is for male patients with Danon Disease, a rare genetic condition. Participants must have a specific gene mutation, good heart pump function (LVEF ≥ 50%), elevated heart stress levels (hsTnI), be at least 8 years old, show signs of thickened heart muscle but normal pumping ability, and be able to follow the study's procedures. They should not need mechanical breathing support or have had certain serious cardiovascular events.
What is being tested?
The trial is testing RP-A501, a gene therapy delivered via a virus vector designed to introduce a healthy version of the LAMP2B gene into patients' cells. It aims to assess how effective and safe this treatment is in improving symptoms or halting progression of Danon Disease in males.
What are the potential side effects?
Potential side effects may include immune reactions to the viral vector like fever or fatigue, injection site reactions, changes in liver enzymes indicating liver inflammation, and other responses related to gene therapy which will be closely monitored.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 8 years old or older.
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My heart condition limits my physical activity but I can still perform light tasks.
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My tests show a genetic change in the LAMP2 gene.
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I am male.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have had a stroke, heart attack, or other serious heart-related issues.
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I have had a heart, lung, liver, or other organ transplant.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~60 months post infusion
This trial's timeline: 3 weeks for screening, Varies for treatment, and 60 months post infusion for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Evaluation of efficacy associated with RP-A501 via co-primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index
Secondary study objectives
Evaluation of efficacy associated with RP-A501 via assessment of event-free survival
Evaluation of efficacy associated with RP-A501 via biomarker evidence of myocardial injury - High Sensitivity Troponin I (hsTnI)
Evaluation of efficacy associated with RP-A501 via biomarker evidence of myocardial injury - N-Terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP)
+3 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: RP-A501Experimental Treatment1 Intervention
One planned dose of RP-A501 in cohorts of subjects with a confirmed diagnosis of Danon Disease.

Find a Location

Closest Location:Boston Children's Hospital· Boston, MA

Who is running the clinical trial?

Rocket Pharmaceuticals Inc.Lead Sponsor
16 Previous Clinical Trials
414 Total Patients Enrolled
Joseph Rossano, MDPrincipal InvestigatorChildren's Hospital of Philadelphia
1 Previous Clinical Trials
7 Total Patients Enrolled
Barry Greenberg, MDPrincipal InvestigatorUniversity of California, San Diego
6 Previous Clinical Trials
484 Total Patients Enrolled
~3 spots leftby Sep 2025