Trials / Recruiting
RecruitingNCT05112237
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
A Prospective and Retrospective Registry and Biomarker Study to Evaluate the Natural History of Pediatric Patients With Cardiomyopathy Due to MYBPC3 Mutations
- Status
- Recruiting
- Phase
- —
- Study type
- Observational
- Enrollment
- 200 (estimated)
- Sponsor
- Tenaya Therapeutics · Industry
- Sex
- All
- Age
- 0 Years – 18 Years
- Healthy volunteers
- Not accepted
Summary
The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality of life (QoL). This study will also collect information on treatments, procedures and outcome in infants and children up to 18 yrs who have this mutation.
Conditions
Timeline
- Start date
- 2021-11-01
- Primary completion
- 2028-06-01
- Completion
- 2028-06-01
- First posted
- 2021-11-08
- Last updated
- 2024-11-13
Locations
29 sites across 4 countries: United States, Canada, Spain, United Kingdom
Source: ClinicalTrials.gov record NCT05112237. Inclusion in this directory is not an endorsement.