RecruitingPhase 2

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

United States, Germany, Italy14 participantsStarted 2023-09-05

Plain-language 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 isoform B (LAMP2B) transgene, in male patients with Danon Disease.

Who can participate

Age range

8 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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)

Exclusion criteria

. 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Evaluation of efficacy via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index

Timeframe: 12 Months post-infusion

Trial details

NCT IDNCT06092034

SponsorRocket Pharmaceuticals Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-04

Contact for this trial

Clinical Information

646-627-0033 clinicaltrials@rocketpharma.com

View on ClinicalTrials.gov More Danon Disease trials