Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
NCT ID: NCT05112237
Last Updated: 2024-11-13
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
200 participants
OBSERVATIONAL
2021-11-01
2028-06-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Outcome of Clinical Phenotypes of Pediatric Myocarditis at Assiut University Children Hospital
NCT07175948
Multiparametric Echocardiography and Cardiac Biomarkers in Pediatric Inflammatory Heart Diseases
NCT05911386
Impact of Serious Pediatric Illness on Parent and Sibling Health
NCT03971344
Descriptive and Retrospective Analysis of Acute Myocarditis Associated With Pandemic COVID-19 in Children
NCT04420468
Role of Matrix Metalloproteinases (MMPs) and Tissue Inhibitors of Metalloproteinases (TIMPs) in Children With Myocarditis
NCT00693134
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
OTHER
OTHER
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Retrospective
All patients who meet the eligibility criteria will be eligible for retrospective chart review.
No interventions assigned to this group
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.
No interventions assigned to this group
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Documented results of genotyping showing the presence of at least one pathogenic or likely pathogenic MYBPC3 mutation (heterozygous, homozygous, or compound heterozygous).
For Infants:
* 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.
Exclusion Criteria
Prospective
* Concurrent participation in an interventional clinical trial unless approved by the sponsor.
* Severe noncardiac disease anticipated to significantly reduce life expectancy.
0 Years
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Tenaya Therapeutics
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
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
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
TN-201-0003
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.