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Danon Disease Natural History Study

StatusRecruiting
PhaseNot specified
Started
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Record History

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  1. Site details revised at 1 of 15 sitesv13
  2. Study sites: 13 to 15v12
  3. Re-verified, no change to tracked fieldsv11
  4. Study sites revised (13)v10
  5. Primary completion date: Aug 31, 2026 to Dec 2029v9 Show 6 more changesShow less
    • Enrollment target: 46 to 60
    • Study completion date: Aug 31, 2026 to Mar 2030
    • Eligibility criteria shortened, about 12 words
    • Secondary endpoints: 15 to 16
    • Site contacts updated at 10 of 13 sites
    • Study description shortened, about 3 words
Show 7 earlier versionsHide 7 earlier versions
  1. Site details revised at 2 of 13 sitesv8
  2. Study sites: 12 to 13v7
  3. Site details revised at 1 of 12 sitesv6
  4. Study sites: 11 to 12
    • Eligibility criteria expanded, about 66 words
    v5
  5. Study sites: 9 to 11v4
  6. Study sites: 4 to 9
    • Secondary endpoints: 6 to 15
    • Study description expanded, about 17 words
    v3
  7. Study sites: 3 to 4v2
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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 (8 to 50 years of age).
Trial Details
NCT Number NCT06214507
Lead Sponsor Rocket Pharmaceuticals Inc.
Conditions
(from sponsor)		 Danon Disease
Related terms Cardiomyopathies, Glycogen Storage Disease Type IIb
Enrollment 60 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov