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Myosin Inhibitor

Aficamten for Pediatric Hypertrophic Cardiomyopathy (CEDAR-HCM Trial)

Phase 2 & 3

Recruiting

Research Sponsored by Cytokinetics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Body weight ≥ 45 kg for the initial cohort and then body weight ≥ 35 kg after at least 10 participants in the initial cohort have undergone dose titration up to Week 4 without observed events of LVEF < 50% at the starting dose of 5 mg qd

New York Heart Association (NYHA) Class ≥ II at screening

Must not have

History or evidence of any other clinically significant disorder, malignancy, active infection, or condition that would pose a risk to participant safety or interfere with the trial

Implantable cardioverter defibrillator (ICD) implantation within 6 weeks of screening or planned ICD implantation during the trial period

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 8 or week 12

Summary

"This trial aims to see how well aficamten works, how safe it is, and how the body processes it in children with a type of heart condition called obstructive hypertrophic cardiomyopathy

Who is the study for?

This trial is for young people aged 12 to under 18 with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). They must have a certain level of heart function and obstruction, be in NYHA Class II or higher, and on stable doses of specific heart medications. Genetic testing should confirm oHCM or assume it if no other conditions are present. Participants need to weigh at least 35 kg after the initial cohort.

What is being tested?

The study tests Aficamten's effectiveness, safety, and pharmacokinetics (how the drug moves through the body) in pediatric patients with oHCM. It compares Aficamten against a placebo – an inactive substance designed to test if the real drug works.

What are the potential side effects?

While not specified here, side effects may include those common to heart medications such as dizziness, low blood pressure, changes in heartbeat rhythm or rate; however individual experiences can vary.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My weight is at least 45 kg, or 35 kg after the first 10 participants.

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My heart condition limits my physical activity.

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I am between 12 and 17 years old.

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My heart's muscle is thickened, and tests show specific heart function and pressure levels.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I don't have any other serious health issues that could affect my safety in the trial.

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I have had or will have an ICD implant within 6 weeks of screening or during the trial.

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I have previously taken aficamten or mavacamten.

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I do not have serious heart valve problems.

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I have a history of weak heart muscle or stress-related heart condition.

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I am undergoing or planning to undergo stereotactic radiotherapy.

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I have a history of heart rhythm problems.

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I have used drugs that can affect heart muscle.

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My heart's pumping ability is below 40%, causing a treatment pause.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 8 or week 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 8 or week 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 8 or week 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Maximum observed concentration (Cmax) of aficamten

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: AficamtenExperimental Treatment1 Intervention

Participants in this arm will receive a single daily oral dose of aficamten with dose levels (5 mg to 20 mg) guided by echocardiography assessments, for 12 weeks during the double-blinded period, and then for another 52 weeks during the open-label extension period.

Group II: PlaceboPlacebo Group2 Interventions

Participants in this arm will receive placebo for 12 weeks during the double-blinded period, and then will receive aficamten for 52 weeks during the open-label extension period.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Aficamten

2023

Completed Phase 1

~50

0 / 13Eligibility criteria met