At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Not specified by the sponsor
- How long the study runs
- Study runs about 79 months (dates as stated)
- About the drug or intervention
- Not specified by the sponsor
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Observing health over time
- Ages
- 0 Years to 18 Years
- Who
- All
- Number of participants
- 200
- Started
- 2021-11-01
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for cardiomyopathy
- • Clinical study - 200 participants
- • 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)
Who can take part?
- • Ages 0 Years to 18 Years
- • Diagnosed with cardiomyopathy
Where?
- • Glasgow - NHS Greater Glasgow and Clyde
- • London - Royal Brompton & Harefield NHS Foundation Trust
- • London - Great Ormond Street Hospital for Children NHS Foundation Trust
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 0 Years - 18 Years
- Who can join: All genders
What the study is looking for
- ✓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...
Who cannot take part
- ✗Patient received heart transplantation or died \>10 years before study initiation. For homozygous or biallelic...
- ✗Prospective
- ✗Inclusion Criteria:
- ✗For Infants:
- ✗Infants who are homozygous or compound heterozygous for the known pathogenic truncating MYBPC3 mutations are eligible.
See the full criteria
Where Is This Study? (3 UK sites)
NHS Greater Glasgow and Clyde
Glasgow G12 0XH, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust
London SW3 6NP, United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London WC1N 3JH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700