CardiomyopathyMyCLIMBNCT05112237
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
An observational study of Cardiomyopathy, run by Tenaya Therapeutics at 29 sites in 4 countries.
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). Read more about this study
- Who is it for?
- Ages 0 to 18 years
- Conditions the study is for
- Cardiomyopathy
- How many people
- 200 participants
- Where
- 29 sitesin 4 countries
- Study dates
- 1 Nov 2021to Jun 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients under the age of 18 years (at enrollment) with cardiomyopathy (CM) and pathogenic or likely pathogenic MYBPC3 genetic mutation
Who may be able to join
- 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).
- 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.
Who cannot join
- 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
- Concurrent participation in an interventional clinical trial unless approved by the sponsor.
- Severe noncardiac disease anticipated to significantly reduce life expectancy.
Trial locations and contacts
29 sites in 4 countries.
29 sites in 4 countries
United States21 sites
University of Colorado Hospital - Anschutz Medical Campus
Aurora, Colorado, 80045
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Cleveland Clinic Main Campus
Cleveland, Ohio, 44195
Children's Medical Center Dallas
Dallas, Texas, 75207
Joe DiMaggio Children's Hospital
Hollywood, Florida, 33021
Texas Children's Hospital
Houston, Texas, 77030
Children's Mercy Hospital Kansas
Kansas City, Missouri, 64108
Cohen Children's Medical Center
Lake Success, New York, 11042
Cardiology Care for Children
Lancaster, Pennsylvania, 17601
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Children's Hospital Los Angeles
Los Angeles, California, 90027
DDC Clinic Center for Special Needs Children
Middlefield, Ohio, 44062
Mount Sinai
New York, New York, 10029
Columbia University Irving Medical Center
New York, New York, 10032
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
University of California Davis Health
Sacramento, California, 95817
Rady Children's Hospital - San Diego
San Diego, California, 92123
St. Louis Children's Hospital
St Louis, Missouri, 63110
Montefiore Medical Center, Albert Einstein College of Medicine
The Bronx, New York, 10467
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, 19803
Spain3 sites
Hospital Sant Joan de Déu Barcelona
Barcelona, 8950
Hospital General Universitario Gregorio Marañón
Madrid, 28003
Hospital Alvaro Cunqueiro - Clinico Universitario Vigo
Vigo, 36312
United Kingdom3 sites
NHS Greater Glasgow and Clyde
Glasgow, G12 0XH
Royal Brompton & Harefield NHS Foundation Trust
London, SW3 6NP
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH
Canada2 sites
University of Alberta Hospital
Edmonton, Alberta, T6G 2B7
The Hospital for Sick Children
Toronto, Ontario
About this study
Official title: 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.
- Conditions
- Cardiomyopathy
- Keywords
- Genetic Cardiomyopathy, Pediatric, MYBPC3, Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy, Restrictive Cardiomyopathy, Non-Compaction cardiomyopathy
- Medical subject headings
- Cardiomyopathies, Cardiomyopathy, Hypertrophic, Cardiomyopathy, Dilated, Cardiomyopathy, Restrictive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 5 years for prospective group, n/a for retrospective group
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2021
- Study start1 Nov 2021
- Primary completionJun 2028 (estimated)
- Study completionJun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CardiomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tenaya Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05112237
- Sponsor's protocol ID
- TN-201-0003
- Acronym
- MyCLIMB
- Status last verified
- Sep 2026
- First submitted
- 27 Oct 2021
- Last updated
- 2 Sep 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective and Retrospective Registry and Biomarker Study to Evaluate the Natural History of Pediatric Patients With Cardiomyopathy Due to MYBPC3 Mutations
- ClinicalTrials.gov ID
- NCT05112237
- Acronym / sponsor ID
- MyCLIMB / TN-201-0003
- Conditions
- Cardiomyopathy
- Keywords
- Genetic Cardiomyopathy, Pediatric, MYBPC3, Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy, Restrictive Cardiomyopathy, Non-Compaction cardiomyopathy
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Tenaya TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 1 Nov 2021 (actual)
- Primary completion
- Jun 2028 (estimated)
- Study completion
- Jun 2028 (estimated)
- First posted
- 8 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Other
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 0 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients under the age of 18 years (at enrollment) with cardiomyopathy (CM) and pathogenic or likely pathogenic MYBPC3 genetic mutation
Key inclusion criteria (8 listed)
- 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).
- Infants who are homozygous or compound heterozygous for the known pathogenic truncating MYBPC3 mutations are eligible.
- 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).
Key exclusion criteria (4 listed)
- 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
- Concurrent participation in an interventional clinical trial unless approved by the sponsor.
- Severe noncardiac disease anticipated to significantly reduce life expectancy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
29 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cardiomyopathy, run by Tenaya Therapeutics at 29 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 2 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States, Spain, United Kingdom, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Sep 2026.