Gene Therapy for Danon Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
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.
Do I need to stop my current medications for the trial?
The trial protocol does not specify whether you need to stop taking your current medications.
Is gene therapy using adeno-associated virus (AAV) vectors generally safe in humans?
Research shows that gene therapy using adeno-associated virus (AAV) vectors is generally safe in humans and animals. In studies for different conditions, such as Leber's congenital amaurosis and phenylketonuria in mice, no significant adverse effects were observed, and any minor effects were transient and not harmful.12345
How does the treatment RP-A501 for Danon Disease differ from other treatments?
What data supports the effectiveness of the treatment RP-A501 for Danon Disease?
Research on similar gene therapies using adeno-associated virus (AAV) vectors for other conditions, like Pompe disease and phenylketonuria, shows promise. These studies indicate that AAV-mediated gene therapy can lead to long-term correction of metabolic issues and promote immune tolerance, suggesting potential effectiveness for RP-A501 in treating Danon Disease.2691011
Who Is on the Research Team?
Joseph W Rossano, M.D.
Principal Investigator
Children's Hospital of Philadelphia
Barry Greenberg, MD
Principal Investigator
University of California, San Diego
Are You a Good Fit for This Trial?
This trial is for male patients with Danon Disease, a rare genetic condition. Participants must have a specific gene mutation, good heart pump function (LVEF ≥ 50%), elevated heart stress levels (hsTnI), be at least 8 years old, show signs of thickened heart muscle but normal pumping ability, and be able to follow the study's procedures. They should not need mechanical breathing support or have had certain serious cardiovascular events.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single intravenous infusion of RP-A501
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term follow-up
Participants are monitored for long-term safety and efficacy outcomes
What Are the Treatments Tested in This Trial?
Interventions
- RP-A501
Find a Clinic Near You
Who Is Running the Clinical Trial?
Rocket Pharmaceuticals Inc.
Lead Sponsor