Hypertrophic Cardiomyopathy Clinical Trial
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
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.
Data is available for patient <18 years of age. Patients must be <18 years of age at enrollment or at time of death.
Documented results of genotyping showing the presence of at least one pathogenic or likely pathogenic MYBPC3 mutation (heterozygous, homozygous, or compound heterozygous).
Patient received cardiac transplantation or died >10 years before study initiation. For homozygous or biallelic infants, data may be collected beyond this 10-year period.
Infants who are homozygous or compound heterozygous for the known pathogenic truncating MYBPC3 mutations are eligible.
For all other participants:
Age <18 at entry into the prospective study.
Documented results of genotyping identifying at least one pathogenic or likely pathogenic MYBPC3 mutation (heterozygous, homozygous, or compound heterozygous).
Diagnosis of Cardiomyopathy (CM): HCM, DCM, RCM, mixed CM, or LVNC.
Concurrent participation in an interventional clinical trial unless approved by the sponsor.
Severe noncardiac disease anticipated to significantly reduce life expectancy.
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There are 20 Locations for this study
Little Rock Arkansas, 72202, United States
Los Angeles California, 90027, United States
Sacramento California, 95817, United States
San Diego California, 92123, United States
Aurora Colorado, 80045, United States
Wilmington Delaware, 19803, United States
Hollywood Florida, 33021, United States
Kansas City Missouri, 64108, United States
Saint Louis Missouri, 63110, United States
Bronx New York, 10467, United States
Lake Success New York, 11042, United States
New York New York, 10029, United States
New York New York, 10032, United States
Cincinnati Ohio, 45229, United States
Middlefield Ohio, 44062, United States
Lancaster Pennsylvania, 17601, United States
Pittsburgh Pennsylvania, 15224, United States
Houston Texas, 77030, United States
Toronto Ontario, , Canada
Madrid , 28003, Spain
Vigo , 36312, Spain
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