CEDAR-HCM

A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM)

Hypertrophic cardiomyopathy (HCM) is a chronic genetic heart disease in which the heart muscle is abnormally thickened and prevents the heart from pumping blood efficiently.  In some patients with HCM, the thickened heart muscle obstructs blood from leaving the heart.  When this occurs, it is called obstructive HCM.

Patients diagnosed with obstructive HCM may not exhibit symptoms in the early stages of the condition but may develop them over time, including signs and symptoms that can disrupt daily life, such as:

  • chest pain
  • dizziness
  • shortness of breath
  • fatigue
  • fainting
  • palpitations (a sensation of rapid fluttering or pounding heartbeat)
  • heart murmurs (unusual sound heard during heartbeat, extra beats or whooshing/swishing sounds which may be faint or loud)
  • swelling of ankles, legs, abdomen, or veins in the neck.

The severity of these signs and symptoms varies widely between people with obstructive HCM and can even vary day-to-day.

About CEDAR-HCM

Clinical trial participation period: Now and ongoing

A clinical research trial

CEDAR-HCM is a study to compare the effects of aficamten and placebo on the level of obstruction that limits blood flow out of the heart in young participants with obstructive HCM. Aficamten has not been established to be safe and effective by the FDA in this patient population, but it may help reduce symptoms of obstructive of HCM by decreasing excess pumping of the heart muscle.

Participants will be randomly assigned to receive either aficamten (the investigational drug) or placebo (a substance that does not have any medicine in it).

Abbreviated participation criteria

  • 12 to <18 years old
    • This study may be opened to younger participants (age 6 to <12) at a later date depending upon the results from this and other research studies.
  • Body weight ≥ 35 kg
  • Diagnosed with obstructive HCM
  • Have symptoms related to obstructive HCM

Additional participation criteria will apply. Full details regarding the participation criteria can be found at https://clinicaltrials.gov/study/NCT06412666.

What is CEDAR-HCM?

Clinical trial overview

CEDAR-HCM is a clinical trial of aficamten compared to placebo in pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy.

The clinical trial will compare the effects of aficamten and placebo on the level of obstruction that limits blood flow out of the heart in young participants with obstructive HCM. The effectiveness of aficamten and placebo will be compared by measuring the level of obstruction that limits blood flow out of the heart with an echocardiogram (Echo), a visualization of the heart using ultrasound. Participants will also be asked to complete questionnaires regarding the status of their general health, obstructive HCM symptoms, and quality of life. The safety and tolerability of aficamten at different, increasing dose levels will also be studied. Finally, the study will measure the amount of aficamten in the blood at various times and the effect the research medicine may have on participants’ obstructive HCM.

Clinical trial duration

There are three parts to this study. If all parts are completed, participation will last up to 222 weeks (about 4 years and 3 months):

  • Period 1 (Double-Blind): There will be a screening period to determine if patients are eligible to participate. All screening procedures will be completed within 56 days (about 8 weeks). Eligible participants who enroll in the study will receive aficamten or placebo for 12 weeks (3 months). The study medicine each participant receives will be determined by chance (like rolling dice). The chance of receiving aficamten will be 2 in 3 (66%), and the chance of receiving placebo will be 1 in 3 (33%). At the end of the 12-week treatment, there will be a 2-week “wash-out” where participants will not take any study drug before starting Period 2.
  • Period 2 (Open-Label): Participants who complete Period 1, and are eligible, will have the opportunity to receive aficamten for 52 weeks (1 year). No placebo is used in Period 2.
  • Period 3 (Open-Label): Participants who complete Period 2 will have the opportunity to continue to receive aficamten for an additional 144 weeks (about 2 years and 9 months). Period 3 will have a flexible follow-up schedule with alternating phone and clinic visits. No placebo is used in Period 3.

Study medicine doses

In period 1, participants who receive aficamten may receive up to four possible dose levels (5, 10, 15, or 20 mg) over the first 6 weeks. The starting dose will be 5 mg at Day 1 and may increase to 10 mg at Week 2, 15 mg at Week 4, and up to 20 mg at Week 6, depending on how well the drug is tolerated.

In Period 2, participants will receive 52-weeks of aficamten

In Period 3, participants will receive 144-weeks of aficamten

Clinical Trial Locations

Clinical trial sites for CEDAR-HCM are recruiting participants across the United States.

Check Eligibility

CEDAR-HCM site locations:

Visit clinicaltrials.gov (NCT06412666) to view contacts & locations.