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Can a gene registry unlock the secrets of childhood heart failure?

NCT ID NCT05112237

Knowledge-focused Sponsor: Tenaya Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Sep 02, 2026 · Last updated Sep 03, 2026 · Updated 1 time

Summary

This study gathers health information from children under 18 who have cardiomyopathy linked to mutations in the MYBPC3 gene. Researchers aim to map the disease's natural course, identify risk factors, and measure how it affects quality of life. By reviewing past and future medical data, they hope to better understand the condition and inform care for affected children.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to
If successful, this study could clarify how MYBPC3-related cardiomyopathy progresses in children, guiding future treatments and care strategies.
What could go wrong
This is an observational registry, not a treatment trial, so it won't directly test a therapy. Data collection depends on available records and participant enrollment, which may limit conclusions.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 200 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2021

Expected to finish

Jun 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

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

Ages

0 to 18 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

cardiomyopathy dilated cardiomyopathy hypertrophic cardiomyopathy restrictive cardiomyopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Cardiomyopathy

Contacts and locations

Locations

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Cardiology Care for Children

    Lancaster, Pennsylvania, 17601, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Medical Center Dallas

    Dallas, Texas, 75207, United States

  • Children's Mercy Hospital Kansas

    Kansas City, Missouri, 64108, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Cleveland Clinic Main Campus

    Cleveland, Ohio, 44195, United States

  • Cohen Children's Medical Center

    Lake Success, New York, 11042, United States

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • DDC Clinic Center for Special Needs Children

    Middlefield, Ohio, 44062, United States

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, WC1N 3JH, United Kingdom

  • Hospital Alvaro Cunqueiro - Clinico Universitario Vigo

    Vigo, 36312, Spain

  • Hospital General Universitario Gregorio Marañón

    Madrid, 28003, Spain

  • Hospital Sant Joan de Déu Barcelona

    Barcelona, 8950, Spain

  • Joe DiMaggio Children's Hospital

    Hollywood, Florida, 33021, United States

  • Montefiore Medical Center, Albert Einstein College of Medicine

    The Bronx, New York, 10467, United States

  • Mount Sinai

    New York, New York, 10029, United States

  • NHS Greater Glasgow and Clyde

    Glasgow, G12 0XH, United Kingdom

  • Nemours Alfred I. Dupont Hospital for Children

    Wilmington, Delaware, 19803, United States

  • Rady Children's Hospital - San Diego

    San Diego, California, 92123, United States

  • Royal Brompton & Harefield NHS Foundation Trust

    London, SW3 6NP, United Kingdom

  • St. Louis Children's Hospital

    St Louis, Missouri, 63110, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • The Hospital for Sick Children

    Toronto, Ontario, Canada

  • University of Alberta Hospital

    Edmonton, Alberta, T6G 2B7, Canada

  • University of California Davis Health

    Sacramento, California, 95817, United States

  • University of Colorado Hospital - Anschutz Medical Campus

    Aurora, Colorado, 80045, United States

  • University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

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