RECRUITING

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

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Conditions

CardiomyopathyGenetic CardiomyopathyPediatricMYBPC3Hypertrophic Cardiomyopathy (HCM)Dilated CardiomyopathyRestrictive CardiomyopathyNon-Compaction cardiomyopathy

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Quick Facts

Study Start:2021-11-01
Study Completion:2028-06
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05112237

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:0 Years to 18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * Data is available for patient \<18 years of age. Patients must be \<18 years of age at enrollment or at time of death.
  2. * Documented results of genotyping showing the presence of at least one pathogenic or likely pathogenic MYBPC3 mutation (heterozygous, homozygous, or compound heterozygous).
  1. * 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.

Contacts and Locations

Study Contact

Matthew Pollman, MD
CONTACT
650-209-8092
mpollman@tenayathera.com
LaTanya Tomlinson
CONTACT
clinical.trials@tenayathera.com

Study Locations (Sites)

Arkansas Children's Hospital
Little Rock, Arkansas, 72202
United States
Children's Hospital Los Angeles
Los Angeles, California, 90027
United States
University of California Davis Health
Sacramento, California, 95817
United States
Rady Children's Hospital - San Diego
San Diego, California, 92123
United States
University of Colorado Hospital - Anschutz Medical Campus
Aurora, Colorado, 80045
United States
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, 19803
United States
Joe DiMaggio Children's Hospital
Hollywood, Florida, 33021
United States
Children's Mercy Hospital Kansas
Kansas City, Missouri, 64108
United States
St. Louis Children's Hospital
Saint Louis, Missouri, 63110
United States
Montefiore Medical Center, Albert Einstein College of Medicine
Bronx, New York, 10467
United States
Cohen Children's Medical Center
Lake Success, New York, 11042
United States
Mount Sinai
New York, New York, 10029
United States
Columbia University Irving Medical Center
New York, New York, 10032
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
Cleveland Clinic Main Campus
Cleveland, Ohio, 44195
United States
DDC Clinic Center for Special Needs Children
Middlefield, Ohio, 44062
United States
Cardiology Care for Children
Lancaster, Pennsylvania, 17601
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
United States
Children's Medical Center Dallas
Dallas, Texas, 75207
United States
Texas Children's Hospital
Houston, Texas, 77030
United States

Collaborators and Investigators

Sponsor: Tenaya Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-11-01
Study Completion Date2028-06

Study Record Updates

Study Start Date2021-11-01
Study Completion Date2028-06

Terms related to this study

Keywords Provided by Researchers

  • Genetic Cardiomyopathy
  • Pediatric
  • MYBPC3
  • Hypertrophic Cardiomyopathy (HCM)
  • Dilated Cardiomyopathy
  • Restrictive Cardiomyopathy
  • Non-Compaction cardiomyopathy

Additional Relevant MeSH Terms

  • Cardiomyopathy