Trial Outcomes & Findings for PRecIsion Medicine in CardiomyopathY (NCT NCT04036799)

Results Overview

The composite SCD event includes post diagnosis SCD, aborted SCD (including ventricular fibrillation, sustained ventricular tachycardia), primary ICD insertion with appropriate shock, secondary ICD insertion

Recruitment status

ACTIVE_NOT_RECRUITING

Target enrollment

572 participants

Primary outcome timeframe

Time to a composite sudden cardiac death event during 5-year follow-up

Results posted on

2025-05-21

Participant Flow

This was a multicenter retrospective observational cohort study of pediatric patients with clinically diagnosed childhood-onset HCM. The primary cohort included patients from 11 sites participating in the PRIMaCY study (Precision Medicine for Cardiomyopathy), an international registry of patients with pediatric HCM launched in 2017. These included 4 Canadian, 6 US, and 1 Australian site. For model validation, we used data from the Sarcomeric Human Cardiomyopathy Registry.

Participant milestones

Participant milestones
Measure	Hypertrophic Cardiomyopathy Patients with a clinical diagnosis of HCM defined as having an LV posterior wall or septal thickness z score ≥2.
Overall Study STARTED	572
Overall Study COMPLETED	572
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Hypertrophic Cardiomyopathy n=572 Participants Patients included were those with a clinical diagnosis of HCM defined as having LV posterior wall or septal thickness z score ≥2, age \<18 years at the time of diagnosis, absence of a SCD event before diagnosis, and at least 1 follow-up assessment after diagnosis.
Age, Continuous	9.8 years n=572 Participants
Sex: Female, Male Female	178 Participants n=572 Participants
Sex: Female, Male Male	394 Participants n=572 Participants
Region of Enrollment United States	235 participants n=572 Participants
Region of Enrollment Canada	273 participants n=572 Participants
Region of Enrollment Australia	64 participants n=572 Participants

PRIMARY outcome

Timeframe: Time to a composite sudden cardiac death event during 5-year follow-up

Population: Patients with a clinical diagnosis of HCM defined as having an LV posterior wall or septal thickness z score ≥2, age \<18 years at the time of diagnosis, absence of a sudden cardiac death event before diagnosis, and at least 1 follow-up assessment after diagnosis.

The composite SCD event includes post diagnosis SCD, aborted SCD (including ventricular fibrillation, sustained ventricular tachycardia), primary ICD insertion with appropriate shock, secondary ICD insertion

Outcome measures

Outcome measures
Measure	Hypertrophic Cardiomyopathy n=572 Participants Phenotype-positive HCM diagnosed between 1987 and 2018
Number of Participants With a Composite Sudden Cardiac Death Event	53 Participants

Adverse Events

Hypertrophic Cardiomyopathy

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Seema Mital, Staff Cardiologist

Hospital for Sick Children

Phone: 416-813-7418

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place