Phase
Condition
Circulation Disorders
Treatment
Placebo
Aficamten
Clinical Study ID
Ages 12-17 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Period 1: Treatment Period
Males and females between 12 and < 18 years of age at screening and at Day 1.
Body weight ≥ 45 kg for the initial cohort and then body weight ≥ 35 kg afterat least 10 participants in the initial cohort have undergone dose titration upto Week 4 without observed events of LVEF < 50% at the starting dose of 5 mgqd.
Core laboratory confirmation of the following oHCM echocardiographic criteriaat screening:
Left ventricular (LV) hypertrophy with nondilated LV chamber in the absence of othercardiac disease.
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 genetictest.
LVEF ≥ 60% AND Valsalva LVOT-G ≥ 50 mmHg.
oHCM of sarcomeric origin confirmed by genetic testing or, if unable to confirmby genetic testing, oHCM of sarcomeric origin may be presumed in the absence ofhistory of metabolic disorders, mitochondrial cardiomyopathies, neuromusculardisease, malformation syndromes, infiltrative diseases/inflammation, andendocrine disorders (such as Fabry's disease, Noonan syndrome with leftventricular 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 shouldhave 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 relatedto compliance or safety, the Medical Monitor may review and determine eligibility.
LVEF ≥ 55% after washout.
Exclusion
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 tosystolic anterior motion of the mitral valve (per judgment of PrincipalInvestigator or designee).
Evidence of fixed left-sided obstruction (eg, subaortic membrane, aortic valvestenosis, or coarctation of the aorta).
History of LV systolic dysfunction (LVEF < 45%) or stress cardiomyopathy at any timeduring their clinical course.
History of congenital heart disease other than oHCM (may be enrolled if nothemodynamically significant in the judgement of the Principal Investigator and studyMedical Monitor).
Has been treated with SRT (surgical myectomy or percutaneous alcohol septalablation) within the preceding 6 months or has plans for either treatment during thetrial period.
History of paroxysmal or persistent atrial fibrillation or atrial flutter.
History of syncope, symptomatic ventricular arrhythmia, or sustained ventriculartachyarrhythmia within 3 months prior to screening.
History or evidence of any other clinically significant disorder, malignancy, activeinfection, other condition, or disease that, in the opinion of the PrincipalInvestigator (or designee) or the Medical Monitor, would pose a risk to participantsafety 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], andtyrosine kinase inhibitors [sunitinib and imatinib]).
Currently participating in another investigational device or drug trial or receivedan investigational device or drug < 1 month (or 5 half-lives for drugs, whichever islonger) prior to screening.
Implantable cardioverter defibrillator (ICD) implantation within 6 weeks ofscreening 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 hearttransplantation in the next 12 months.
Study Design
Study Description
Connect with a study center
The Hospital for Sick Children (SickKids)
Toronto, Ontario M5G 1E8
CanadaSite Not Available
Careggi University Hospital
Florence,
ItalyActive - Recruiting
Unidad de Cardiología Infantil; Hospital Universitario da Coruña
A Coruña,
SpainActive - Recruiting
Alder Hey Children's Hospital
Liverpool,
United KingdomActive - Recruiting
Great Ormond Street Hospital for Children
London, WC1N 3BH
United KingdomActive - Recruiting
Children's Hospital Los Angeles
Los Angeles, California 90027
United StatesSite Not Available
University of California, Los Angeles (UCLA)
Los Angeles, California 90095
United StatesActive - Recruiting
Children's Hospital Colorado
Aurora, Colorado 80045
United StatesActive - Recruiting
Children's National Hospital
Washington, District of Columbia 20010
United StatesActive - Recruiting
Baptist Health South Florida
Miami, Florida 33176
United StatesSite Not Available
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois 60611
United StatesSite Not Available
University of Iowa
Iowa City, Iowa 52242
United StatesActive - Recruiting
Children's Hospital New Orleans
New Orleans, Louisiana 70018
United StatesSite Not Available
University of Michigan
Ann Arbor, Michigan 48109
United StatesActive - Recruiting
Children's Hospital of Michigan
Detroit, Michigan 48201
United StatesActive - Recruiting
Mayo Clinic
Rochester, Minnesota 55905
United StatesActive - Recruiting
Children's Mercy Hospital
Kansas City, Missouri 64108
United StatesActive - Recruiting
University of Nebraska Medical Center
Omaha, Nebraska 68198
United StatesSite Not Available
Morristown Medical Center
Morristown, New Jersey 07960
United StatesActive - Recruiting
Columbia University
New York, New York 10027
United StatesSite Not Available
NYP/Columbia University Medical Center
New York, New York 10027
United StatesSite Not Available
Duke Clinical Research Institute
Durham, North Carolina 27701
United StatesSite Not Available
Oregon Health & Science University
Portland, Oregon 97239
United StatesActive - Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
LeBonheur Children's Hospital
Memphis, Tennessee 38103
United StatesSite Not Available
Vanderbilt University Medical Center
Nashville, Tennessee 37235
United StatesActive - Recruiting
UT Southwestern
Dallas, Texas 75390
United StatesSite Not Available
Children's Wisconsin
Milwaukee, Wisconsin 53226
United StatesSite Not Available
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