RecruitingNCT05158738

Inherited Cardiac cONditions In Kids

Inherited Cardiac cONditions In Kids: Understanding the Genetics and Outcomes of Paediatric Inherited CardiacConditions


Sponsor

Imperial College London

Enrollment

300 participants

Start Date

Sep 1, 2022

Study Type

OBSERVATIONAL

Conditions

Summary

All patients with heart disease should have the opportunity to participate in research into their condition, to advance knowledge and treatment. The investigators have built an online registry and database - The Heart Hive - to connect research-willing participants (with heart muscle disease) with active researchers and projects. Participants enrol and upload their own data through the website.This study uses The Heart Hive platform to study cardiomyopathies - heart muscle disease. These are progressive diseases, and there is a need to better understand what factors affect the chances of developing cardiomyopathy, and how the condition progresses. The study will collect information about participants diagnosis, DNA for genetic analysis, and then follow participants' clinical progress. The study will identify genetic variants that cause cardiomyopathy, and determine which specific genetic or environmental factors predict disease severity, progression and response to treatment, with an overall objective of identifying new and personalised treatments for patients with this disease.


Eligibility

Inclusion Criteria5

  • Males or females with a confirmed diagnosis of childhood onset (\<16 years) PCM
  • Males or females with childhood onset (\<16 years) of a rare inherited cardiac condition likely to be a monogenic condition
  • Capacity for parents to provide informed consent
  • Genotype negative following local standard diagnostic ICC gene panel
  • Family members of patients with ICC, both affected and unaffected

Exclusion Criteria6

  • Parents who lack capacity to provide consent on behalf of their children/themselves
  • Onset over 16 years
  • Significant teratogen exposure (including maternal diabetes) likely to contribute to cardiac dysfunction (following discussion with Cardiologist)
  • Significant coronary heart disease likely to contribute to cardiac dysfunction (following discussion with Cardiologist)
  • Other secondary causes of cardiac dysfunction likely to explain the phenotype of the patient
  • Patients with a confirmed genetic diagnosis (patients with variants of uncertain significance are not excluded).

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Interventions

OTHERPatients with inherited cardiac condition, onset <16 years old and Parents

Patients with a confirmed diagnosis of an inherited cardiac condition, onset \<16 years old and Parents will have samples that undergo whole genome sequencing and biomarker analysis


Locations(3)

Royal Brompton Hospital

London, United Kingdom

Great Ormond Street Hospital for Children

London, United Kingdom

Harefield Hospital

Uxbridge, United Kingdom

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NCT05158738


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