Gene Therapy for Danon Disease

Recruiting at 2 trial locations

Overseen By Clinical Information

Age: Any Age

Sex: Male

Trial Phase: Phase 1

Sponsor: Rocket Pharmaceuticals Inc.

Must not be taking: Positive inotropes, Vasodilators, Diuretics

Disqualifiers: Cardiac transplant, LVAD, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

7 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene (investigational product (IP), RP-A501) in male patients with Danon Disease (DD).

Research Team

Joseph W Rossano, M.D.

Principal Investigator

Children's Hospital of Philadelphia

Matthew Taylor, MD

Principal Investigator

University of Colorado, Anschutz Medical Ctr

Barry Greenberg, MD

Principal Investigator

University of California, San Diego

Eligibility Criteria

This trial is for male patients with Danon Disease, a genetic condition affecting the heart and muscles. Participants must be males aged 8 years or older with cardiac involvement, able to walk over 150 meters unassisted, have normal blood counts and liver function, and not have had recent heart surgery or other serious health conditions.

Inclusion Criteria

Patients and/or competent custodial parents must provide informed written consent

I can walk more than 150 meters by myself in a walking test.

Your blood levels of hemoglobin, neutrophils, and platelets are within the normal range.

Exclusion Criteria

Your body has a high level of antibodies that can neutralize AAV9.

I have an active blood or solid organ cancer.

I have had a heart attack or unstable chest pain before.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-treatment

Administration of rituximab and sirolimus prophylactically before infusion of the investigational product

1 week

Treatment

Single intravenous infusion of RP-A501 gene therapy product

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

36 months

Long-Term Follow-Up

Participants enter a long-term follow-up study for additional monitoring post-treatment

2 to 5 years

Treatment Details

Interventions

• RP-A501

Trial Overview The study tests RP-A501 gene therapy in males with Danon Disease. It's an open-label Phase 1 trial using AAV9.LAMP2B to deliver the LAMP2 gene. The goal is to assess safety and toxicity of this treatment in affected individuals without randomizing participants into different groups.

Participant Groups

Group I: RP-A501 Experimental Treatment 1 Intervention

RP-A501 is a gene therapy product consisting of a rAAV9 capsid containing the human LAMP2B transgene which will be administered as a single intravenous (IV) infusion. Subjects will receive one of three dose levels depending on the cohort.

Who Is Running the Clinical Trial?

Rocket Pharmaceuticals Inc.

Lead Sponsor

Trials

17

Recruited

430+