Skip to main content
UK clinical trials - updated daily from ClinicalTrials.gov
TrialConnect
← Back to Search
ACTIVE NOT RECRUITINGPhase2/Phase3

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

Sponsor: Cytokinetics

NCT ID: NCT06412666

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Aficamten (drug), Placebo (drug)
How long the study runs
Study runs about 68 months (dates as stated)
About the drug or intervention
Aficamten — drug: Oral Tablet · Placebo — drug: Oral Tablet
Patient visit burden
Not specified by the sponsor
Type of study
Testing a treatment
Ages
12 Years to 17 Years
Who
All
Number of participants
65
Started
2024-05-29
Last checked
2026-08

Plain English Summary

What is this study?

  • • Testing a new treatment for pediatric
  • • Phase2/Phase3 - 65 participants
  • • The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM)

Who can take part?

  • • Ages 12 Years to 17 Years
  • • Diagnosed with pediatric

Where?

  • • Liverpool - Alder Hey Children's Hospital
  • • London - Evelina Children's Hospital
  • • London - Great Ormond Street Hospital for Children

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

More detail

The overall objective of the trial is to determine the efficacy, safety, and tolerability of administration of aficamten in adolescents (12 to \< 18 years old) and children (6 to \< 12 years old) with symptomatic oHCM. Adolescents and children will be studied in a staged approach involving established favorable pharmacodynamic and safety profiles of aficamten in adolescents followed by further pharmacokinetic modeling to inform the dosing regimen in children. The 12 to \<18 years old cohort has enrolled all participants and is no longer recruiting, the 6 to \< 12 year old cohort is not yet recruiting. The trial will consist of 3 periods: 1. Period 1 is the randomized, double-blind, placebo-controlled treatment period that will assess the efficacy, safety and tolerability of aficamten in pediatric participants. 2. Period 2 is the open-label extension trial that will assess the long-term safety of aficamten in pediatric participants, and further assess efficacy and tolerability. 3. Period 3 is the long-term extension trial that will assess the long-term safety of aficamten in pediatric participants, and further assess efficacy and tolerability.

PediatricSymptomatic Obstructive Hypertrophic Cardiomyopathy

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 12 Years - 17 Years
  • Who can join: All genders

Biomarkers mentioned

presence of positivehistory or positive

What the study is looking for

  • ✓Period 1: Treatment Period
  • ✓Males and females between 12 and \< 18 years of age at screening and at Day 1.
  • ✓Body weight ≥ 35 kg
  • ✓Diagnosed with oHCM based on the following at screening:
  • ✓Left ventricular (LV) hypertrophy with nondilated LV chamber in the absence of other heart disease.

Who cannot take part

  • ✗Period 1: Treatment Period
  • ✗Any of the following criteria will exclude potential participants from the trial:
  • ✗Significant valvular heart disease.
  • ✗Moderate or severe valvular aortic stenosis or fixed subaortic obstruction.
  • ✗Mitral regurgitation that is greater than mild in severity and not due to systolic anterior motion of the mitral...
See the full criteria
Inclusion Criteria: * Period 1: Treatment Period * Males and females between 12 and \< 18 years of age at screening and at Day 1. * Body weight ≥ 35 kg * Diagnosed with oHCM based on the following at screening: * Left ventricular (LV) hypertrophy with nondilated LV chamber in the absence of other cardiac disease. * Core laboratory confirmation of LV end-diastolic wall thickness that meets a threshold of: * Z-score \> 2.5 in the absence of family history OR * Z-score \> 2 in the presence of positive family history or positive genetic test. * Core laboratory confirmation of LVEF ≥ 60% AND Valsalva LVOT-G ≥ 50 mmHg. * oHCM of sarcomeric origin confirmed by genetic testing or, if unable to confirm by genetic testing, oHCM of sarcomeric origin may be presumed in the absence of history of metabolic disorders, mitochondrial cardiomyopathies, neuromuscular disease, malformation syndromes, infiltrative diseases/inflammation, and endocrine disorders (such as Fabry's disease, Noonan syndrome with left ventricular hypertrophy, and amyloid-cardiomyopathy). * New York Heart Association (NYHA) Class ≥ II at screening. * Adequate acoustic windows for echocardiography. * Participants on beta blockers, verapamil, diltiazem, or disopyramide should have been on stable doses for more than 4 weeks prior to randomization. * Period 2: Open-Label Extension * Completed Period 1. If unable to complete Period 1 due to circumstances not related to compliance or safety, the Medical Monitor may review and determine eligibility. * LVEF ≥ 55% after washout * Period 3: Long-term Extension * Completed Period 2 Exclusion Criteria: * Period 1: Treatment Period Any of the following criteria will exclude potential participants from the trial: * Significant valvular heart disease. * Moderate or severe valvular aortic stenosis or fixed subaortic obstruction. * Mitral regurgitation that is greater than mild in severity and not due to systolic anterior motion of the mitral valve (per judgment of Principal Investigator or designee). * Evidence of fixed left-sided obstruction (eg, subaortic membrane, aortic valve stenosis, or coarctation of the aorta). * History of LV systolic dysfunction (LVEF \< 45%) or stress cardiomyopathy at any time during their clinical course. * History of congenital heart disease other than oHCM (may be enrolled if not hemodynamically significant in the judgement of the Principal Investigator and study Medical Monitor). * Has been treated with SRT (surgical myectomy or percutaneous alcohol septal ablation) within the preceding 6 months or has plans for either treatment during the trial period. * History of paroxysmal or persistent atrial fibrillation or atrial flutter. * History of syncope, symptomatic ventricular arrhythmia, or sustained ventricular tachyarrhythmia within 3 months prior to screening. * History or evidence of any other clinically significant disorder, malignancy, active infection, other condition, or disease that, in the opinion of the Principal Investigator (or designee) or the Medical Monitor, would pose a risk to participant safety or interfere with the trial evaluation, procedures, or completion. * Current or previous use of drugs known to cause cardiomyopathy (eg, anthracyclines, monoclonal antibodies \[trastuzumab\], alkylating agents \[cyclophosphamide\], and tyrosine kinase inhibitors \[sunitinib and imatinib\]). * Currently participating in another investigational device or drug trial or received an investigational device or drug \< 1 month (or 5 half-lives for drugs, whichever is longer) prior to screening. * Implantable cardioverter defibrillator (ICD) implantation within 6 weeks of screening or planned ICD implantation during the trial period. * Has received prior treatment with aficamten or mavacamten. * Currently listed for heart transplantation or anticipated to be listed for heart transplantation in the next 12 months.

Where Is This Study? (3 UK sites)

Alder Hey Children's Hospital

Liverpool, United Kingdom

Hospital R&D contact (matched)

Kelly Davies

research@alderhey.nhs.uk0151 2525570

Evelina Children's Hospital

London SW3 6NP, United Kingdom

Great Ormond Street Hospital for Children

London WC1N 3BH, United Kingdom

Hospital R&D contact (matched)

Main Email: Research.Governance@gosh.nhs.uk

Research.Governance@gosh.nhs.uk0207 905 2700
Data sourced from ClinicalTrials.gov · Last verified: 2026-08