- Conditions
- Danon Disease
- Interventions
- Not listed
- Lead sponsor
- University of California, San Diego
- Other
- Eligibility
- 0 Years to 100 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2024
- U.S. locations
- 1
- States / cities
- San Diego, California
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- Conditions
- Glycogen Storage Disease, GSD Type 0A, GSD Type 0B, GSD VII, Tarui Disease, GSD X, GSD XII, GSD XIII, GSD XV, PGBM2, PRKAG2, Danon Disease, Polyglucosan Body Myopathy Type 1, Polyglucosan Body Myopathy Type 2, RBCK1 Deficiency
- Interventions
- No intervention
- Other
- Lead sponsor
- Duke University
- Other
- Eligibility
- 0 Years to 90 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2034
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Danon Disease
- Interventions
- RP-A501
- Genetic
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 8 Years and older · Male only
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2032
- U.S. locations
- 3
- States / cities
- La Jolla, California • Boston, Massachusetts • Philadelphia, Pennsylvania
- Conditions
- Danon Disease
- Interventions
- No intervention
- Other
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2030
- U.S. locations
- 8
- States / cities
- Phoenix, Arizona • La Jolla, California • Aurora, Colorado + 5 more
- Conditions
- Danon Disease
- Interventions
- RP-A501
- Biological
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 8 Years and older · Male only
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 3
- States / cities
- La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania