Clinical trial NCT04036799: PRecIsion Medicine in CardiomyopathY
Official title: Development of a Risk Calculator to Predict Sudden Cardiac Death in Children With Hypertrophic Cardiomyopathy
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Hypertrophic Cardiomyopathy
Sudden Cardiac Death
Keywords: Pediatric
Risk Prediction
Lead sponsor: The Hospital for Sick Children
Sponsor class: OTHER
Collaborators: name: Stanford University
name: Children's Hospital of Philadelphia
name: New York Presbyterian Hospital
name: University of Texas Southwestern Medical Center
name: University of Michigan
name: Children's Hospital Medical Center, Cincinnati
name: Provincial Health Services Authority
name: Stollery Children's Hospital
name: Children's Hospital of Eastern Ontario
name: Royal Children's Hospital
name: Children's Healthcare of Atlanta
name: Primary Children's Hospital
name: Baylor College of Medicine
name: The Cleveland Clinic
name: Monroe Carell Jr. Children's Hospital at Vanderbilt
name: Children's Hospital Colorado
name: Indiana University Health
name: OHSU Doernbecher Children's Hospital
name: Children's Hospital Los Angeles
First posted: 2019-07-30
Enrollment: 572
Sex: ALL
Minimum age (years): 0
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Individuals meeting eligibility criteria at first presentation were included.

Brief summary: This is a retrospective cohort study of pediatric hypertrophic cardiomyopathy (HCM) patients using chart and registry review methodology. The studies objective is to develop and validate a sudden cardiac death (SCD) risk calculator that is age-appropriate for children with HCM that includes clinical and genetic factors.


Eligibility criteria: Inclusion Criteria:

* phenotype-positive patients diagnosed with hypertrophic cardiomyopathy
* phenotype-negative, genotype positive patients considered at risk for developing hypertrophic cardiomyopathy

Exclusion Criteria:

* Neuromuscular, metabolic, syndromic (other than Noonan Syndrome and related RAS-opathies) or endocrine (including infants of diabetic mothers) causes of HCM
* (Other treatable causes of left ventricular hypertrophy (systemic hypertension, anatomic defects causing left ventricular outflow tract obstruction e.g. aortic stenosis, subAS, subaortic membrane, coarctation)

Primary outcomes:
measure: Number of Participants With a Composite Sudden Cardiac Death Event
timeFrame: Time to a composite sudden cardiac death event during 5-year follow-up
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
References:
citation: Miron A, Lafreniere-Roula M, Steve Fan CP, Armstrong KR, Dragulescu A, Papaz T, Manlhiot C, Kaufman B, Butts RJ, Gardin L, Stephenson EA, Howard TS, Aziz PF, Balaji S, Ladouceur VB, Benson LN, Colan SD, Godown J, Henderson HT, Ingles J, Jeewa A, Jefferies JL, Lal AK, Mathew J, Jean-St-Michel E, Michels M, Nakano SJ, Olivotto I, Parent JJ, Pereira AC, Semsarian C, Whitehill RD, Wittekind SG, Russell MW, Conway J, Richmond ME, Villa C, Weintraub RG, Rossano JW, Kantor PF, Ho CY, Mital S. A Validated Model for Sudden Cardiac Death Risk Prediction in Pediatric Hypertrophic Cardiomyopathy. Circulation. 2020 Jul 21;142(3):217-229. doi: 10.1161/CIRCULATIONAHA.120.047235. Epub 2020 May 18.