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Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
Sponsor: Tenaya Therapeutics
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.
Official title: A Prospective and Retrospective Registry and Biomarker Study to Evaluate the Natural History of Pediatric Patients With Cardiomyopathy Due to MYBPC3 Mutations
Key Details
Gender
All
Age Range
0 Years - 18 Years
Study Type
OBSERVATIONAL
Enrollment
200
Start Date
2021-11-01
Completion Date
2028-06
Last Updated
2024-11-13
Healthy Volunteers
No
Conditions
Locations (29)
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
University of California Davis Health
Sacramento, California, United States
Rady Children's Hospital - San Diego
San Diego, California, United States
University of Colorado Hospital - Anschutz Medical Campus
Aurora, Colorado, United States
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, United States
Joe DiMaggio Children's Hospital
Hollywood, Florida, United States
Children's Mercy Hospital Kansas
Kansas City, Missouri, United States
St. Louis Children's Hospital
St Louis, Missouri, United States
Cohen Children's Medical Center
Lake Success, New York, United States
Mount Sinai
New York, New York, United States
Columbia University Irving Medical Center
New York, New York, United States
Montefiore Medical Center, Albert Einstein College of Medicine
The Bronx, New York, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Cleveland Clinic Main Campus
Cleveland, Ohio, United States
DDC Clinic Center for Special Needs Children
Middlefield, Ohio, United States
Cardiology Care for Children
Lancaster, Pennsylvania, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Children's Medical Center Dallas
Dallas, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
University of Alberta Hospital
Edmonton, Alberta, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada
Hospital Sant Joan de Déu Barcelona
Barcelona, Spain
Hospital General Universitario Gregorio Marañón
Madrid, Spain
Hospital Alvaro Cunqueiro - Clinico Universitario Vigo
Vigo, Spain
NHS Greater Glasgow and Clyde
Glasgow, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust
London, United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom