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A study of aficamten (CK-3773274) in a Pediatric Population with Symptomatic Obstructive Hypertrophic Cardiomyopathy
EU CTIS ID: 2024-511377-30-00
What this study is testing
P1: To assess the effect of aficamten compared with placebo on change from baseline in Valsalva LVOT-G P2: To determine the safety of aficamten in pediatric participants with symptomatic oHCM
- Phase II and Phase III (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- P1: Males and females between 12 and < 18 years of age at screening and at Day 1.
- P3: completed period 2
- P1: 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.
- P1: Diagnosed with oHCM per the following criteria, confirmed at the time of 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 (Ommen 2020)>2.5 in the absence of family history or Z-score (Ommen 2020)>2 in the presence of positive family history or positive genetic test. Core laboratory confirmation of LVEF ≥60% AND Valsalva LVOT-G ≥50mmHg.
- P1: 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).
- P1: New York Heart Association (NYHA) Class ≥ II at screening.
You likely can't join if
- P1: 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).
- P1: 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.
- P1: History of paroxysmal or persistent atrial fibrillation or atrial flutter.
- P1: History of syncope, symptomatic ventricular arrhythmia, or sustained ventricular tachyarrhythmia within 3 months prior to screening.
- P1: 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.
- P1: 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]).
See the full eligibility criteria
Who can join
- P1: Males and females between 12 and < 18 years of age at screening and at Day 1.
- P3: completed period 2
- P1: 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.
- P1: Diagnosed with oHCM per the following criteria, confirmed at the time of 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 (Ommen 2020)>2.5 in the absence of family history or Z-score (Ommen 2020)>2 in the presence of positive family history or positive genetic test. Core laboratory confirmation of LVEF ≥60% AND Valsalva LVOT-G ≥50mmHg.
- P1: 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).
- P1: New York Heart Association (NYHA) Class ≥ II at screening.
- P1: Adequate acoustic windows for echocardiography.
- P1: Participants on beta blockers, verapamil, diltiazem, or disopyramide should have been on stable doses or more than 4 weeks prior to randomization.
- P2: 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.
- P2: LVEF ≥ 55% after washout.
What rules you out
- P1: 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).
- P1: 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.
- P1: History of paroxysmal or persistent atrial fibrillation or atrial flutter.
- P1: History of syncope, symptomatic ventricular arrhythmia, or sustained ventricular tachyarrhythmia within 3 months prior to screening.
- P1: 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.
- P1: 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]).
- P1: 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.
- P1: Implantable cardioverter defibrillator (ICD) implantation within 6 weeks of screening or planned ICD implantation during the trial period.
- P1: Has received prior treatment with aficamten or mavacamten.
- P1: Currently listed for heart transplantation or anticipated to be listed for heart transplantation in the next 12 months.
- P1: Does not assent/consent to participate in the CMR substudy.
- P1: Inability to tolerate CMR without sedation.
- P1: Has an ICD or cardiac pacemaker.
- P1: History of LV systolic dysfunction (LVEF < 45%) or stress cardiomyopathy at any time during their clinical course.
- P1: 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).
- P1: Hypersensitivity to aficamten or any of the excipients
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
- Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom; Canada. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.