Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy (NCT05112237)
A Prospective and Retrospective Registry and Biomarker Study to Evaluate the Natural History of Pediatric Patients With Cardiomyopathy Due to MYBPC3 Mutations
π©Ί Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Arkansas Children's Hospital β Little Rock, Arkansas
Age range: 0 Years to 18 Years
π "Do I Qualify?" β 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria β
π All Participating Trial Locations (29 Sites)
Showing the first 25 of 29 sites.
- Arkansas Children's Hospital β Little Rock, Arkansas
- Children's Hospital Los Angeles β Los Angeles, California
- University of California Davis Health β Sacramento, California
- Rady Children's Hospital - San Diego β San Diego, California
- University of Colorado Hospital - Anschutz Medical Campus β Aurora, Colorado
- Nemours Alfred I. Dupont Hospital for Children β Wilmington, Delaware
- Joe DiMaggio Children's Hospital β Hollywood, Florida
- Children's Mercy Hospital Kansas β Kansas City, Missouri
- St. Louis Children's Hospital β St Louis, Missouri
- Cohen Children's Medical Center β Lake Success, New York
- Mount Sinai β New York, New York
- Columbia University Irving Medical Center β New York, New York
- Montefiore Medical Center, Albert Einstein College of Medicine β The Bronx, New York
- Cincinnati Children's Hospital Medical Center β Cincinnati, Ohio
- Cleveland Clinic Main Campus β Cleveland, Ohio
- DDC Clinic Center for Special Needs Children β Middlefield, Ohio
- Cardiology Care for Children β Lancaster, Pennsylvania
- Children's Hospital of Philadelphia β Philadelphia, Pennsylvania
- University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh β Pittsburgh, Pennsylvania
- Children's Medical Center Dallas β Dallas, Texas
- Texas Children's Hospital β Houston, Texas
- University of Alberta Hospital β Edmonton, Alberta
- The Hospital for Sick Children β Toronto, Ontario
- Hospital Sant Joan de DΓ©u Barcelona β Barcelona,
- Hospital General Universitario Gregorio MaraΓ±Γ³n β Madrid,
π Contact the Study Coordinator
Phone: (650) 209-8092
Email: mpollman@tenayathera.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
π° Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA Β§ 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
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π‘ How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.