RecruitingNot Applicable

Danon Disease Natural History Study

United States60 participantsStarted 2023-12-20

Plain-language summary

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).

Who can participate

Age range8 Years

SexALL

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Exclusion criteria

✕. Concurrent enrollment in any other clinical investigation involving use of an investigational agent for any condition at time of enrollment to this study that could confound interpretation of this study
✕. Previous treatment with a gene therapy
✕. Prior mechanical circulatory support at time of enrollment to this study
✕. Prior cardiac transplantation at time of enrollment to this study
✕. Age \>51 years at enrollment

What they're measuring

Left Ventricular Mass Index (LVMI) by echocardiogram

Timeframe: Month 12, 24, 36

Trial details

NCT IDNCT06214507

SponsorRocket Pharmaceuticals Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2029-12

Contact for this trial

Clinical Information

646-627-0033 clinicaltrials@rocketpharma.com

View on ClinicalTrials.gov More Danon Disease trials