Risk Stratification in Children and Adolescents With Primary Cardiomyopathy

• Conditions: Primary Cardiomyopathy; Dilated Cardiomyopathy; Arrhythmogenic Right Ventricular Cardiomyopathy; Hypertrophic Cardiomyopathy; Left Ventricular Noncompaction; Restrictive Cardiomyopathy

• Registration Number: NCT03572569
• Lead Sponsor: German Heart Institute

Brief Summary

RIKADA is a prospective study performing systematic family screening including clinical and genetic testing in pediatric patients with primary cardiomyopathy and their first-degree relatives with the aim to facilitate risk stratification.

Detailed Description

RIKADA is a long-term prospective study performing in-depth phenotype and genotype characterization in children and adolescents with primary cardiomyopathy and their first-degree family members. Family screening contains complete cardiac work-up with medical history, physical examination, 12-lead-/Holter-electrocardiogram, cardiopulmonary exercise testing, echocardiography, cardiovascular magnetic resonance (CMR) and laboratory including genetic testing. The aim is to facilitate early identification of at-risk individuals and contribute to patient-specific follow-up and therapy regimes preventing progressive heart failure and arrhythmia in pediatric CMP.

Study Timeline

• Study Start: 2013-01
• Status Verified: 2018-06
• Study First Submitted: 2018-06-01
• Study First Submitted QC: 2018-06-27
• Last Update Submitted: 2018-06-27
• Study First Posted: 2018-06-28
• Last Update Posted: 2018-06-28
• Primary Completion: 2020-12
• Study Completion: 2020-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 200

Inclusion Criteria

1. Index patients:

   • Age ≤18 years
   • written informed consent of parents/legal guardians
   • diagnosis of primary cardiomypathy:
   • DCM: left ventricular (LV) systolic dysfunction and dilatation greater than two standard deviations (SD) above the mean of a normal population
   • HCM: LV hypertrophy and septal wall thickness above two SD
   • RCM: diastolic dysfunction and concordant atrial enlargement
   • LVNC: separation of the myocardium into a compacted (C) and a non- compacted (NC) layer with an NC/C ratio >2 in echocardiography and/or >2.3 in CMR
   • ARVC: according to the revised Task Force Criteria

2. First-degree family members (parents and siblings):

   • Age ≥3 years
   • written informed consent of parents/legal guardians and siblings ≥18 years

Exclusion Criteria

• unwillingness to give consent
• myocardial inflammation / myocarditis
• systemic disease with cardiac involvement (secondary cardiomyopathy)
• structural congenital heart disease

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
major cardiovascular events	from date of enrollment until the date of death, mechanical circulatory support or heart transplantation, assessed up to 8 years	death, need for mechanical circulatory support or heart transplantation

Trial Locations

Locations (2)

Charité - Universitätsmedizin Berlin; 🇩🇪 Berlin, Germany

German Heart Institute; 🇩🇪 Berlin, Germany