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

Plain-language summary

This study evaluates RP-A501, a gene therapy, in male patients with Danon disease caused by changes in the LAMP2 gene. Male patients aged 8 and older who carry a disease-causing LAMP2 variant and have certain heart findings may take part. The study is in Phase 2 and is currently enrolling participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Recruiting

Sponsor

Rocket Pharmaceuticals Inc.

Intervention

RP-A501

Start date

9/5/2023

Estimated completion

4/1/2032

Countries

United States, Germany, Italy, United Kingdom

Related genes

Locations

• La Jolla, United States
• Boston, United States
• Philadelphia, United States
• Munich, Germany
• Florence, Italy
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Key Inclusion Criteria:

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.

Key Exclusion Criteria:

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.