Danon Disease clinical trials at University of California Health

2 research studies open to eligible people

Showing trials for

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

    open to eligible males ages 8 years and up

    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

  • Danon Disease Natural History Study

    open to eligible people 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 and female patients <30 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

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