Phase 3 Cardiomyopathy Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• People who are aged 12 and under 18 years old at the time of screening and at the start of the trial.
• People who weigh at least 45 kg (for the first group of participants), or at least 35 kg once the trial has confirmed safety data from at least 10 earlier participants.
• People whose heart scans (echocardiograms), reviewed by a central laboratory, show thickened heart walls, a non-enlarged left heart chamber, no other heart disease, and specific measurements of wall thickness based on age and family history.
• People whose heart scans show that the heart is pumping well (at least 60% efficiency) and that there is significant blockage of blood flow out of the heart during a specific breathing test (Valsalva manoeuvre), measuring at least 50 mmHg.
• People diagnosed with obstructive hypertrophic cardiomyopathy (oHCM) caused by a gene variant, confirmed by genetic testing — or, if genetic testing is not possible, where the condition is not linked to certain other known diseases or syndromes (confirm with trial site).
• People whose heart condition causes at least some symptoms during daily life, as classified by a standard medical scale (New York Heart Association Class II or higher).
• People whose heart can be clearly seen on an ultrasound scan (adequate acoustic windows).
• People already taking certain heart medications (beta blockers, verapamil, diltiazem, or disopyramide) who have been on a stable dose for more than 4 weeks before joining the trial.
• People who have completed the first part of the trial may be eligible for the second (open-label extension) part, provided their heart pumping function remains at 55% or above after a washout period.
• People who have completed the second part of the trial may be eligible for the third (long-term extension) part.

Who may not be able to join:

• People with significant valve problems in the heart, including moderate or severe narrowing of the aortic valve, fixed blockages below the aortic valve, or a leaking mitral valve (beyond mild) not caused by a specific movement of the valve.
• People with a history of the heart's main pumping chamber working poorly (pumping less than 45% efficiency) or a type of heart condition called stress cardiomyopathy, at any point in their life.
• People with a history of heart defects present from birth, other than oHCM (though some cases may be considered if judged to be not serious, at the discretion of the trial's principal investigator and medical monitor).
• People who have had a heart procedure to reduce the thickened heart muscle (surgical myectomy or alcohol septal ablation) in the past 6 months, or who are planning to have one during the trial.
• People with a history of an irregular heart rhythm involving the upper chambers of the heart (atrial fibrillation or atrial flutter).
• People who have fainted, had significant irregular heartbeats, or had a dangerous fast heart rhythm in the 3 months before screening.
• People with another serious medical condition, active infection, or illness that the trial doctors believe could put them at risk or affect the trial results.
• People who have taken or are currently taking certain medications known to damage the heart muscle (such as some chemotherapy drugs — confirm with trial site).
• People currently taking part in another clinical trial, or who have received an experimental drug or device within 1 month (or 5 half-lives of the drug, whichever is longer) before screening.
• People who have had a heart device (implantable defibrillator/ICD) implanted within 6 weeks of screening, or who are planning to have one implanted during the trial.
• People who have previously been treated with aficamten or mavacamten.
• People who are on a waiting list for a heart transplant, or who are expected to be placed on one within the next 12 months.

Important: Always verify eligibility with the trial site directly before applying.