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 range8 Years

SexMALE

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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.
✕. Prior cardiac or other organ (lung, liver, other) transplantation.

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