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

Part of paid clinical trials in La Jolla, California.

Sponsor
Rocket Pharmaceuticals Inc.
Study ID
NCT06092034
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Danon Disease

Eligibility Criteria

Sex
MALE
Age
8 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • RP-A501 — GENETIC
    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.

Study Details

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.

Key Dates

Start date
Sep 5, 2023
Status verified
Apr 2026
Primary completion
Apr 30, 2028
Completion
Apr 30, 2032

Study Design

Enrollment
14 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: RP-A501
    One planned dose of RP-A501 in cohorts of subjects with a confirmed diagnosis of Danon Disease.

Primary Outcome Measure

Evaluation of efficacy via primary endpoint comprised of LAMP2 myocardial tissue expression and left ventricular mass index [ Time Frame: 12 Months post-infusion ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
University of California, San DiegoLa JollaCalifornia92037
Barry Greenberg, MD
[email protected]
858-246-2987
Boston Children's HospitalBostonMassachusetts02115
Emily Morris
[email protected]
Bridget Kerwin
[email protected]
Dominic Abrams, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Joseph Rossano, MD
[email protected]

Find similar trials in La Jolla, CA

By research site
University of California, San Diego· La Jolla, CABoston Children's Hospital· Boston, MAChildren's Hospital of Philadelphia· Philadelphia, PA

Related Studies