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

Cardiomyopathy Clinical Trial

— MyCLIMB  

Official title:

A Prospective and Retrospective Registry and Biomarker Study to Evaluate the Natural History of Pediatric Patients With Cardiomyopathy Due to MYBPC3 Mutations

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05112237
• Other study ID #: TN-201-0003
• Status: Recruiting
• Start date: November 1, 2021
• Est. completion date: June 2028

Study information

• Verified date: November 2023
• Source: Tenaya Therapeutics
• Contact: Laura Robertson, MD
• Phone: 650-416-1186
• Email: lrobertson[@]tenayathera.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: June 2028
• Est. primary completion date: June 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 18 Years

Eligibility

Retrospective

Inclusion Criteria:

• Data is available for patient <18 years of age. Patients must be <18 years of age at enrollment or at time of death.

• Documented results of genotyping showing the presence of at least one pathogenic or likely pathogenic MYBPC3 mutation (heterozygous, homozygous, or compound heterozygous).

Exclusion Criteria:

• 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.

Prospective

Inclusion Criteria:

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:

• Concurrent participation in an interventional clinical trial unless approved by the sponsor.

• Severe noncardiac disease anticipated to significantly reduce life expectancy.

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy

Locations

Country	Name	City	State
Canada	University of Alberta Hospital	Edmonton	Alberta
Canada	The Hospital for Sick Children	Toronto	Ontario
Spain	Hospital Sant Joan de Déu Barcelona	Barcelona
Spain	Hospital General Universitario Gregorio Marañón	Madrid
Spain	Hospital Alvaro Cunqueiro - Clinico Universitario Vigo	Vigo
United Kingdom	NHS Greater Glasgow and Clyde	Glasgow
United Kingdom	Great Ormond Street Hospital for Children NHS Foundation Trust	London
United Kingdom	Royal Brompton & Harefield NHS Foundation Trust	London
United States	University of Colorado Hospital - Anschutz Medical Campus	Aurora	Colorado
United States	Montefiore Medical Center, Albert Einstein College of Medicine	Bronx	New York
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Cleveland Clinic Main Campus	Cleveland	Ohio
United States	Children's Medical Center Dallas	Dallas	Texas
United States	Joe DiMaggio Children's Hospital	Hollywood	Florida
United States	Texas Children's Hospital	Houston	Texas
United States	Children's Mercy Hospital Kansas	Kansas City	Missouri
United States	Cohen Children's Medical Center	Lake Success	New York
United States	Cardiology Care for Children	Lancaster	Pennsylvania
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Children's Hospital Los Angeles	Los Angeles	California
United States	DDC Clinic Center for Special Needs Children	Middlefield	Ohio
United States	Columbia University Irving Medical Center	New York	New York
United States	Mount Sinai	New York	New York
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	University of California Davis Health	Sacramento	California
United States	St. Louis Children's Hospital	Saint Louis	Missouri
United States	Rady Children's Hospital - San Diego	San Diego	California
United States	Nemours Alfred I. Dupont Hospital for Children	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
Tenaya Therapeutics

Countries where clinical trial is conducted

United States,  Canada,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To characterize the disease course and natural history in participants with pathogenic or likely pathogenic MYBPC3 mutations with a specific focus on cardiac events and measurement		5 years for prospective group, n/a for retrospective group