Actively Recruiting
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
Led by Tenaya Therapeutics · Updated on 2024-11-13
200
Participants Needed
29
Research Sites
343 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient is under 18 years of age at enrollment or at time of death
- Documented genotyping results showing at least one pathogenic or likely pathogenic MYBPC3 mutation (heterozygous, homozygous, or compound heterozygous)
- Infants who are homozygous or compound heterozygous for known pathogenic truncating MYBPC3 mutations are eligible (prospective group)
- Diagnosis of cardiomyopathy including hypertrophic, dilated, restrictive, mixed, or left ventricular noncompaction cardiomyopathy (prospective group)
You will not qualify if you...
- Patient received cardiac transplantation or died more than 10 years before study start (except homozygous or biallelic infants, who may have data collected beyond 10 years)
- Concurrent participation in an interventional clinical trial without sponsor approval
- Severe noncardiac disease expected to significantly reduce life expectancy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 29 locations
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
2
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
3
University of California Davis Health
Sacramento, California, United States, 95817
Actively Recruiting
4
Rady Children's Hospital - San Diego
San Diego, California, United States, 92123
Actively Recruiting
5
University of Colorado Hospital - Anschutz Medical Campus
Aurora, Colorado, United States, 80045
Actively Recruiting
6
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, United States, 19803
Actively Recruiting
7
Joe DiMaggio Children's Hospital
Hollywood, Florida, United States, 33021
Actively Recruiting
8
Children's Mercy Hospital Kansas
Kansas City, Missouri, United States, 64108
Actively Recruiting
9
St. Louis Children's Hospital
St Louis, Missouri, United States, 63110
Actively Recruiting
10
Cohen Children's Medical Center
Lake Success, New York, United States, 11042
Actively Recruiting
11
Mount Sinai
New York, New York, United States, 10029
Actively Recruiting
12
Columbia University Irving Medical Center
New York, New York, United States, 10032
Actively Recruiting
13
Montefiore Medical Center, Albert Einstein College of Medicine
The Bronx, New York, United States, 10467
Actively Recruiting
14
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
15
Cleveland Clinic Main Campus
Cleveland, Ohio, United States, 44195
Actively Recruiting
16
DDC Clinic Center for Special Needs Children
Middlefield, Ohio, United States, 44062
Actively Recruiting
17
Cardiology Care for Children
Lancaster, Pennsylvania, United States, 17601
Actively Recruiting
18
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
19
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
20
Children's Medical Center Dallas
Dallas, Texas, United States, 75207
Actively Recruiting
21
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
22
University of Alberta Hospital
Edmonton, Alberta, Canada, T6G 2B7
Actively Recruiting
23
The Hospital for Sick Children
Toronto, Ontario, Canada
Actively Recruiting
24
Hospital Sant Joan de Déu Barcelona
Barcelona, Spain, 8950
Actively Recruiting
25
Hospital General Universitario Gregorio Marañón
Madrid, Spain, 28003
Actively Recruiting
26
Hospital Alvaro Cunqueiro - Clinico Universitario Vigo
Vigo, Spain, 36312
Actively Recruiting
27
NHS Greater Glasgow and Clyde
Glasgow, United Kingdom, G12 0XH
Actively Recruiting
28
Royal Brompton & Harefield NHS Foundation Trust
London, United Kingdom, SW3 6NP
Actively Recruiting
29
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom, WC1N 3JH
Actively Recruiting
Research Team
M
Matthew Pollman, MD
CONTACT
L
LaTanya Tomlinson
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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