ClinicaliQ Trial Snapshot
- Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy — Active Not Recruiting • NCT05112237.
- Study tracking heart disease progression in children with MYBPC3 gene mutations to understand symptoms, treatments, and quality of life.
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What This Trial Is Studying
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: Cardiomyopathy Lead Sponsor: Tenaya Therapeutics Planned Enrollment: 200 participants