Danon Disease clinical trials at University of California Health
3 in progress, 1 open to eligible people
Danon Disease Natural History Study
open to eligible males ages 8 years and up
The goal of this international observational study is to learn about the natural history of Danon disease in male patients >8 years of age. The key objectives include assessing change over time in cardiac structure (left ventricular mass and wall thickness), cardiac biomarkers, symptoms, and quality of life and the incidence of clinical events such as HF hospitalization, cardiac transplantation, and death.
at UCSD
Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Sorry, in progress, not accepting new patients
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.
at UCSD
Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
Sorry, in progress, not accepting new patients
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).
at UCSD
Our lead scientists for Danon Disease research studies include Barry Greenberg, MD.
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