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

September 1, 2026 updated by: Tenaya Therapeutics

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Cardiomyopathy

Study Type

Observational

Enrollment (Estimated)

200

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Canada
- Alberta
  - Edmonton, Alberta, Canada, T6G 2B7
    - University of Alberta Hospital
- Ontario
  - Toronto, Ontario, Canada
    - The Hospital for Sick Children
Spain
- - Barcelona, Spain, 8950
    - Hospital Sant Joan de Déu Barcelona
  - Madrid, Spain, 28003
    - Hospital General Universitario Gregorio Maranon
  - Vigo, Spain, 36312
    - Hospital Alvaro Cunqueiro - Clinico Universitario Vigo
United Kingdom
- - Glasgow, United Kingdom, G12 0XH
    - NHS Greater Glasgow and Clyde
  - London, United Kingdom, WC1N 3JH
    - Great Ormond Street Hospital for Children NHS Foundation Trust
  - London, United Kingdom, SW3 6NP
    - Royal Brompton & Harefield NHS Foundation Trust
United States
- Arkansas
  - Little Rock, Arkansas, United States, 72202
    - Arkansas Children's Hospital
- California
  - Los Angeles, California, United States, 90027
    - Children's Hospital Los Angeles
  - Sacramento, California, United States, 95817
    - University of California Davis Health
  - San Diego, California, United States, 92123
    - Rady Children's Hospital - San Diego
- Colorado
  - Aurora, Colorado, United States, 80045
    - University of Colorado Hospital - Anschutz Medical Campus
- Delaware
  - Wilmington, Delaware, United States, 19803
    - Nemours Alfred I. Dupont Hospital for Children
- Florida
  - Hollywood, Florida, United States, 33021
    - Joe DiMaggio Children's Hospital
- Missouri
  - Kansas City, Missouri, United States, 64108
    - Children's Mercy Hospital Kansas
  - St Louis, Missouri, United States, 63110
    - St. Louis Children's Hospital
- New York
  - Lake Success, New York, United States, 11042
    - Cohen Children's Medical Center
  - New York, New York, United States, 10032
    - Columbia University Irving Medical Center
  - New York, New York, United States, 10029
    - Mount Sinai
  - The Bronx, New York, United States, 10467
    - Montefiore Medical Center, Albert Einstein College of Medicine
- Ohio
  - Cincinnati, Ohio, United States, 45229
    - Cincinnati Children's Hospital Medical Center
  - Cleveland, Ohio, United States, 44195
    - Cleveland Clinic Main Campus
  - Middlefield, Ohio, United States, 44062
    - DDC Clinic Center for Special Needs Children
- Pennsylvania
  - Lancaster, Pennsylvania, United States, 17601
    - Cardiology Care for Children
  - Philadelphia, Pennsylvania, United States, 19104
    - Children's Hospital of Philadelphia
  - Pittsburgh, Pennsylvania, United States, 15224
    - University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
- Texas
  - Dallas, Texas, United States, 75207
    - Children's Medical Center Dallas
  - Houston, Texas, United States, 77030
    - Texas Children's Hospital

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.

Eligibility Criteria

Ages Eligible for Study

1 second to 18 years (Child, Adult)

Accepts Healthy Volunteers

Sampling Method

Non-Probability Sample

Study Population

Patients under the age of 18 years (at enrollment) with cardiomyopathy (CM) and pathogenic or likely pathogenic MYBPC3 genetic mutation

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Number of groups / cohorts

Cohorts and Interventions

Group / Cohort
Retrospective All patients who meet the eligibility criteria will be eligible for retrospective chart review.
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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 Time Frame: 5 years for prospective group, n/a for retrospective group	5 years for prospective group, n/a for retrospective group

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

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 Major Dates

Study Start (Actual)

November 1, 2021

Primary Completion (Estimated)

June 1, 2028

Study Completion (Estimated)

June 1, 2028

Study Registration Dates

First Submitted

October 27, 2021

First Submitted That Met QC Criteria

October 27, 2021

First Posted (Actual)

November 8, 2021

Study Record Updates

Last Update Posted (Actual)

September 2, 2026

Last Update Submitted That Met QC Criteria

September 1, 2026

Last Verified