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

Phase 2RecruitingNCT06092034

Rocket Pharmaceuticals Inc.14 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Danon Disease

Interventions

RP-A501GENETIC

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.

Eligibility

Sex: MALEMin age: 8 Years

Medical Language ↔ Plain English

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.

Locations (6)

University of California, San Diego

La Jolla, California, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Outcomes

Primary Outcomes

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

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

Time frame: 12 Months post-infusion

Secondary Outcomes

Evaluation of efficacy via components of the primary endpoint - LAMP2

Increase in LAMP2 protein expression

Time frame: 12 months post infusion

Evaluation of efficacy via components of the primary endpoint - Left Ventricular Mass Index (LVMI)

Decrease in Left Ventricular Mass Index (LVMI)

Time frame: 12 months post infusion

Evaluation of efficacy via biomarker evidence of myocardial injury - High Sensitivity Troponin I (hsTnI)

Decrease in high sensitivity Troponin I (hsTnI)

Time frame: 12 months post infusion

Evaluation of efficacy via biomarker evidence of myocardial injury - N-Terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP)

Decrease in N-terminal prohormone of brain natriuretic peptide (NT-proBNP)

Time frame: 12 months post infusion

Evaluation of efficacy via assessment of event-free survival

1. Event free survival with events defined as death, heart transplant, mechanical circulatory support (MCS) or heart failure hospitalization 2. Incidence of death, heart transplant, MCS, or heart failure hospitalization

Time frame: 60 months post infusion

Evaluation of safety

Central Contacts

Clinical Information

CONTACT

646-627-0033 clinicaltrials@rocketpharma.com

Data from ClinicalTrials.gov

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