Hypertrophic CardiomyopathyPRIMaCYNCT04036799

PRecIsion Medicine in CardiomyopathY

An observational study of Hypertrophic Cardiomyopathy and Sudden Cardiac Death, run by The Hospital for Sick Children.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Trial 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… Read more about this study

Who is it for?: Ages 0 to 18 years
Conditions the study is for: Hypertrophic Cardiomyopathy, Sudden Cardiac Death
How many people: 572 participants
Where: No sites listed yet
Study dates: 1 Jan 2017to Dec 2026
Phase: Observational
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 0 to 18 years
All sexes
No healthy volunteers
Child, Adult

Study population: Individuals meeting eligibility criteria at first presentation were included.

Who may be able to join

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

Who cannot join

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)

About this study

Official title: Development of a Risk Calculator to Predict Sudden Cardiac Death in Children With Hypertrophic Cardiomyopathy

Conditions: Hypertrophic Cardiomyopathy, Sudden Cardiac Death
Keywords: Pediatric, Risk Prediction
Medical subject headings: Cardiomyopathy, Hypertrophic, Death, Sudden, Cardiac

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT04036799
Study group	Role	What they receive
Hypertrophic Cardiomyopathy		Not described

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With a Composite Sudden Cardiac Death Event
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
Measured: Time to a composite sudden cardiac death event during 5-year follow-up

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Jan 2017Dec 2026

First posted30 Jul 2019
Study start1 Jan 2017
Primary completion1 Jun 2020
Study completionDec 2026 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Hypertrophic CardiomyopathyStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Observational
Observational model: Cohort
Time perspective: Retrospective
Enrolment: 572 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04036799
Sponsor's protocol ID: 1000055819
Acronym: PRIMaCY
Status last verified: May 2025
First submitted: 23 Jul 2019
Last updated: 21 May 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Publications

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.
PubMed 32418493

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

Not yet recruiting
NHLBI SESAME (SEptal Scoring Along Midline Endocardium) Multi-Center Prospective Study Using Improvised Off-Label Devices
Hypertrophic Cardiomyopathy, Left Ventricular Outflow Obstruction
RecruitingPhase 2
A Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients With Symptomatic Non-obstructive Hypertrophic Cardiomyopathy
Non-obstructive Hypertrophic Cardiomyopathy
Not yet recruiting
Human Experimental Models of Pain (HEMP)
Acute Pain
Active, not recruitingPhase 3
A Study to Learn More About How Well Aficamten Works in Japanese Participants With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Obstructive Hypertrophic Cardiomyopathy
RecruitingPhase 1
Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of HTX-001
Nonobstructive Hypertrophic Cardiomyopathy

All Hypertrophic Cardiomyopathy trials

Study at a glance

From the registry record, last updated by the sponsor on 21 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Development of a Risk Calculator to Predict Sudden Cardiac Death in Children With Hypertrophic Cardiomyopathy
ClinicalTrials.gov ID: NCT04036799
Acronym / sponsor ID: PRIMaCY / 1000055819
Conditions: Hypertrophic Cardiomyopathy, Sudden Cardiac Death
Keywords: Pediatric, Risk Prediction
Study type: Observational
Recruitment status: Active, not recruiting
Enrolment: 572 participants (actual)
Lead sponsor: The Hospital for Sick ChildrenUniversity, hospital or other
Collaborators: Stanford University, Children's Hospital of Philadelphia, New York Presbyterian Hospital, University of Texas Southwestern Medical Center, University of Michigan, Children's Hospital Medical Center, Cincinnati, Provincial Health Services Authority British Columbia, Stollery Children's Hospital, Children's Hospital of Eastern Ontario, Royal Children's Hospital, Children's Healthcare of Atlanta, Primary Children's Hospital, Baylor College of Medicine, The Cleveland Clinic, Monroe Carell Jr. Children's Hospital at Vanderbilt, Children's Hospital Colorado, Indiana University Health, OHSU Doernbecher Children's Hospital, Children's Hospital Los Angeles
Study start: 1 Jan 2017 (actual)
Primary completion: 1 Jun 2020 (actual)
Study completion: Dec 2026 (estimated)
First posted: 30 Jul 2019 (actual)
Results: Posted 11 Mar 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Observational model: Cohort
Time perspective: Retrospective
Number of arms: 1

Arm	Type	Receives
Hypertrophic Cardiomyopathy	–	–

Who may qualify

Ages: 0 to 18 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult
Study population: Individuals meeting eligibility criteria at first presentation were included.

Key inclusion criteria (2 listed)

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

Key exclusion criteria (2 listed)

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)

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Primary outcome

Number of Participants With a Composite Sudden Cardiac Death Event
Time frame: Time to a composite sudden cardiac death event during 5-year follow-up
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

Results and safety

Results posted to the registry on 11 Mar 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Hypertrophic Cardiomyopathy	572	572

Primary outcome: Number of Participants With a Composite Sudden Cardiac Death Event

Time frame: Time to a composite sudden cardiac death event during 5-year follow-up. Count of participants. Unit: Participants

Measure	Hypertrophic Cardiomyopathy n = 572
Value	53

Hypertrophic Cardiomyopathy: Cumulative proportion of events at 5 yrs: 9.1

Population analysed: 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.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Hypertrophic Cardiomyopathy	0 of 0	0 of 0	0

Limitations and caveats (from the sponsor): The study has limitations inherent to any retrospective analysis, that includes missing data, survivor bias, and lower uptake of genetic testing in the earlier era. Missing echocardiographic data were addressed through a re-review of echocardiograms where available and through imputation where this was not possible. We deliberately did not perform an echocardiographic core laboratory analysis because a real-world, point-of-care tool has to rely on locally acquired data.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 21 May 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 32418493Miron 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.
Individual participant dataSharing planned: No

Related trials for Hypertrophic Cardiomyopathy

Not yet recruiting
NHLBI SESAME (SEptal Scoring Along Midline Endocardium) Multi-Center Prospective Study Using Improvised Off-Label Devices
Hypertrophic Cardiomyopathy, Left Ventricular Outflow Obstruction
RecruitingPhase 2
A Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients With Symptomatic Non-obstructive Hypertrophic Cardiomyopathy
Non-obstructive Hypertrophic Cardiomyopathy
Not yet recruiting
Human Experimental Models of Pain (HEMP)
Acute Pain
Active, not recruitingPhase 3
A Study to Learn More About How Well Aficamten Works in Japanese Participants With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Obstructive Hypertrophic Cardiomyopathy
RecruitingPhase 1
Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of HTX-001
Nonobstructive Hypertrophic Cardiomyopathy

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

Questions about this trial

What is this clinical trial studying?

An observational study of Hypertrophic Cardiomyopathy and Sudden Cardiac Death, run by The Hospital for Sick Children.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 21 May 2025. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 Mar 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With a Composite Sudden Cardiac Death Event (time frame: Time to a composite sudden cardiac death event during 5-year follow-up). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04036799, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 May 2025.