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.6 sites in 4 countries14 target enrollmentSeptember 5, 2023

ConditionsDanon Disease

Overview

Phase: Phase 2
Intervention: Not specified
Conditions: Danon Disease
Sponsor: Rocket Pharmaceuticals Inc.
Enrollment: 14
Locations: 6
Primary Endpoint: Evaluation of efficacy via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index
Status: Recruiting
Last Updated: 6 months ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials Related News

Overview

Brief 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.

Detailed Description

The study is a single arm Phase 2 clinical trial to characterize the safety and efficacy of RP-A501, a recombinant adeno-associated serotype 9 (rAAV9 capsid containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene) in male patients with Danon Disease. Male subjects ≥8 years of age will receive a single intravenous infusion of RP-A501.

Registry

clinicaltrials.gov

Start Date

September 5, 2023

End Date

April 1, 2032

Last Updated

6 months ago

Study Type

Interventional

Study Design

Single Group

Sex

Male

Investigators

Sponsor

Rocket Pharmaceuticals Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
•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 Criteria

•Anti-AAV9 neutralizing antibody titer \>1:
•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.

Outcomes

Primary Outcomes

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

Time Frame: 12 Months post-infusion

Increase of myocardial tissue expression of LAMP2 protein and decrease in left ventricular mass index (LVMI).

Secondary Outcomes

Evaluation of efficacy via components of the primary endpoint - LAMP2(12 months post infusion)
Evaluation of efficacy via components of the primary endpoint - Left Ventricular Mass Index (LVMI)(12 months post infusion)
Evaluation of efficacy via biomarker evidence of myocardial injury - High Sensitivity Troponin I (hsTnI)(12 months post infusion)
Evaluation of efficacy via biomarker evidence of myocardial injury - N-Terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP)(12 months post infusion)
Evaluation of efficacy via assessment of event-free survival(60 months post infusion)
Evaluation of safety(60 months post infusion)