A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
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
Rocket Pharmaceuticals Inc.
14 participants
Sep 5, 2023
INTERVENTIONAL
Conditions
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.
Eligibility
Inclusion Criteria9
- 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)
- Ability to comply with study procedures including investigational therapy and follow-up evaluations.
Exclusion Criteria4
- 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.
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
RP-A501 is a gene therapy product consisting of a rAAV9 capsid containing the human LAMP2B transgene which will be administered as a single IV infusion.
Locations(6)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06092034