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

To be considered for this study, children and young people must be under 18 years old. They need to have had a genetic test that confirmed a change in their MYBPC3 gene, which is known to cause heart problems.

For information taken from past medical records, the child must have been under 18 when the information was recorded or when they passed away. There's a special rule for very young babies with two copies of the faulty gene where older records can be included.

For new information being collected (going forward), children also need to be under 18 and have the MYBPC3 gene change. They must also have been diagnosed with cardiomyopathy. If a child is already taking part in another study where a new treatment is being tested, they might not be able to join this one unless specific approval is given. Also, if a child has another serious illness that is expected to significantly shorten their life, they would not be eligible.

This study primarily involves collecting information from your child's existing medical records. This means researchers will look at past details about their condition, treatments they've received, and how they progressed. For some children, especially very young infants with specific gene changes or those joining the study currently, there might also be a plan to collect new information ongoingly. This would involve following your child's health and treatment over time through their regular medical appointments and records. You would not need to attend extra visits or take any new experimental medication for this study. The total duration of participation for those followed over time would depend on the study plan and your child's medical care, but it focuses on gathering routine medical data.