Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
Tenaya Therapeutics
Study ID
NCT05112237
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
0 Years - 18 Years
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

Start date
Nov 1, 2021
Status verified
Nov 2024
Primary completion
Jun 30, 2028
Completion
Jun 30, 2028

Study Design

Enrollment
200 participants (estimated)

Arms

  • Arm: Retrospective
    All patients who meet the eligibility criteria will be eligible for retrospective chart review.
  • Arm: Prospective
    100 patients meeting the eligibility criteria will be followed for 5 years, in addition to a retrospective chart review. Assessments will be completed as part of a participant's regular schedule of physician visits, no additional visits will be required. Aside from a simple annual blood draw, assessments are non-invasive, including a Quality of Life questionnaire.

Primary Outcome Measure

To characterize the disease course and natural history in participants with pathogenic or likely pathogenic MYBPC3 mutations with a specific focus on cardiac events and measurement [ Time Frame: 5 years for prospective group, n/a for retrospective group ]

Central Contacts

Locations (21)

FacilityCityStateZIPSite coordinators
Arkansas Children's HospitalLittle RockArkansas72202-
Children's Hospital Los AngelesLos AngelesCalifornia90027-
University of California Davis HealthSacramentoCalifornia95817-
Rady Children's Hospital - San DiegoSan DiegoCalifornia92123-
University of Colorado Hospital - Anschutz Medical CampusAuroraColorado80045-
Nemours Alfred I. Dupont Hospital for ChildrenWilmingtonDelaware19803-
Joe DiMaggio Children's HospitalHollywoodFlorida33021-
Children's Mercy Hospital KansasKansas CityMissouri64108-
St. Louis Children's HospitalSt LouisMissouri63110-
Cohen Children's Medical CenterLake SuccessNew York11042-
Columbia University Irving Medical CenterNew YorkNew York10032-
Mount SinaiNew YorkNew York10029-
Montefiore Medical Center, Albert Einstein College of MedicineThe BronxNew York10467-
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229-
Cleveland Clinic Main CampusClevelandOhio44195-
DDC Clinic Center for Special Needs ChildrenMiddlefieldOhio44062-
Cardiology Care for ChildrenLancasterPennsylvania17601-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
University of Pittsburgh Medical Center - Children's Hospital of PittsburghPittsburghPennsylvania15224-
Children's Medical Center DallasDallasTexas75207-
Texas Children's HospitalHoustonTexas77030-

Find similar trials in Little Rock, AR

By research site
Arkansas Children's Hospital· Little Rock, ARChildren's Hospital Los Angeles· Los Angeles, CAUniversity of California Davis Health· Sacramento, CARady Children's Hospital - San Diego· San Diego, CAUniversity of Colorado Hospital - Anschutz Medical Campus· Aurora, CONemours Alfred I. Dupont Hospital for Children· Wilmington, DE

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