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Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
NCT05112237 · View on ClinicalTrials.gov ↗
Study 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.
Conditions Studied
Study Locations (20)
New York
- Cohen Children's Medical Center — Lake Success
- Mount Sinai — New York
- Columbia University Irving Medical Center — New York
- Montefiore Medical Center, Albert Einstein College of Medicine — The Bronx
California
- Children's Hospital Los Angeles — Los Angeles
- University of California Davis Health — Sacramento
- Rady Children's Hospital - San Diego — San Diego
Ohio
- Cincinnati Children's Hospital Medical Center — Cincinnati
- Cleveland Clinic Main Campus — Cleveland
- DDC Clinic Center for Special Needs Children — Middlefield
Pennsylvania
- Cardiology Care for Children — Lancaster
- Children's Hospital of Philadelphia — Philadelphia
- University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh — Pittsburgh
Missouri
- Children's Mercy Hospital Kansas — Kansas City
- St. Louis Children's Hospital — St Louis
Arkansas
- Arkansas Children's Hospital — Little Rock
Colorado
- University of Colorado Hospital - Anschutz Medical Campus — Aurora
Delaware
- Nemours Alfred I. Dupont Hospital for Children — Wilmington
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 200 participants |
| Start Date | 2021-11-01 |
| Est. Completion | 2028-06 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05112237
The ClinicalTrials.gov registry entry for NCT05112237 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 200 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Tenaya Therapeutics, which has 35 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Cardiomyopathy appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05112237 reports 20 study locations spanning 10 distinct geographic areas — top geographies include New York, California, Ohio. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05112237 about?
NCT05112237 is a clinical study titled "Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy". 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, p...
What is the current status of trial NCT05112237?
This trial is currently recruiting. The enrollment target is 200 participants. The study started on 2021-11-01. Estimated completion is 2028-06.
What conditions does trial NCT05112237 study?
This clinical trial studies the following conditions: Cardiomyopathy. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT05112237?
This trial is sponsored by Tenaya Therapeutics, which has 35 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05112237 being conducted?
This trial has 20 study locations across Arkansas, California, Colorado, Delaware, Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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