Phase
Condition
Circulation Disorders
Treatment
Aficamten
Placebo
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 notrelated to compliance or safety, the Medical Monitor may review and determineeligibility.
LVEF ≥ 55% after washout.
Period 3: Long-term Extension • Completed Period 2.
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
CanadaActive - Recruiting
The Hospital for Sick Children (SickKids)
Toronto 6167865, Ontario 6093943 M5G 1E8
CanadaSite Not Available
Careggi University Hospital
Florence,
ItalyActive - Recruiting
Careggi University Hospital
Florence 3176959,
ItalySite Not Available
NHO Kagoshima Medical Center
Kagoshima,
JapanActive - Recruiting
NHO Kagoshima Medical Center
Kagoshima 1860827,
JapanSite Not Available
University of Osaka Hospital
Osaka,
JapanActive - Recruiting
University of Osaka Hospital
Osaka 1853909,
JapanSite Not Available
Kitasato University Hospital
Sagamihara,
JapanActive - Recruiting
Kitasato University Hospital
Sagamihara 11611609,
JapanSite Not Available
National Cerebral and Cardiovascular Center
Suita,
JapanActive - Recruiting
National Cerebral and Cardiovascular Center
Suita 1851483,
JapanSite Not Available
Juntendo University Hospital
Tokyo,
JapanActive - Recruiting
Juntendo University Hospital
Tokyo 1850147,
JapanSite Not Available
Unidad de Cardiología Infantil; Hospital Universitario da Coruña
A Coruña,
SpainActive - Recruiting
Unidad de Cardiología Infantil; Hospital Universitario da Coruña
A Coruña 3119841,
SpainSite Not Available
Hospital Sant Joan de Deu
Barcelona,
SpainActive - Recruiting
Hospital Sant Joan de Deu
Barcelona 3128760,
SpainSite Not Available
Alder Hey Children's Hospital
Liverpool,
United KingdomActive - Recruiting
Alder Hey Children's Hospital
Liverpool 2644210,
United KingdomSite Not Available
Evelina Children's Hospital
London, SW3 6NP
United KingdomActive - Recruiting
Great Ormond Street Hospital for Children
London, WC1N 3BH
United KingdomActive - Recruiting
Royal Brompton Hospital
London, SW3 6NP
United KingdomSite Not Available
Evelina Children's Hospital
London 2643743, SW3 6NP
United KingdomSite Not Available
Great Ormond Street Hospital for Children
London 2643743, WC1N 3BH
United KingdomActive - Recruiting
Phoenix Children's Hospital
Phoenix, Arizona 85016
United StatesActive - Recruiting
Phoenix Children's Hospital
Phoenix 5308655, Arizona 5551752 85016
United StatesSite Not Available
Children's Hospital Los Angeles
Los Angeles, California 90027
United StatesActive - Recruiting
University of California, Los Angeles (UCLA)
Los Angeles, California 90095
United StatesActive - Recruiting
UCSF Benioff Children's Hospital
San Francisco, California 94158
United StatesSite Not Available
Children's Hospital Los Angeles
Los Angeles 5368361, California 5332921 90027
United StatesSite Not Available
University of California, Los Angeles (UCLA)
Los Angeles 5368361, California 5332921 90095
United StatesActive - Recruiting
UCSF Benioff Children's Hospital
San Francisco 5391959, California 5332921 94158
United StatesSite Not Available
Children's Hospital Colorado
Aurora, Colorado 80045
United StatesActive - Recruiting
Children's Hospital Colorado
Aurora 5412347, Colorado 5417618 80045
United StatesSite Not Available
Children's National Hospital
Washington, District of Columbia 20010
United StatesSite Not Available
Children's National Hospital
Washington D.C., District of Columbia 20010
United StatesActive - Recruiting
Children's National Hospital
Washington D.C. 4140963, District of Columbia 4138106 20010
United StatesSite Not Available
Baptist Health South Florida
Miami, Florida 33176
United StatesSite Not Available
Nicklaus Children's Hospital
Miami, Florida 33155
United StatesActive - Recruiting
Nicklaus Children's Hospital
Miami 4164138, Florida 4155751 33155
United StatesSite Not Available
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois 60611
United StatesActive - Recruiting
Ann & Robert H. Lurie Children's Hospital
Chicago 4887398, Illinois 4896861 60611
United StatesSite Not Available
University of Iowa
Iowa City, Iowa 52242
United StatesSite Not Available
University of Iowa
Iowa City 4862034, Iowa 4862182 52242
United StatesSite Not Available
Children's Hospital New Orleans
New Orleans, Louisiana 70018
United StatesActive - Recruiting
Children's Hospital New Orleans
New Orleans 4335045, Louisiana 4331987 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
University of Michigan
Ann Arbor 4984247, Michigan 5001836 48109
United StatesSite Not Available
Children's Hospital of Michigan
Detroit 4990729, Michigan 5001836 48201
United StatesSite Not Available
Mayo Clinic
Rochester, Minnesota 55905
United StatesActive - Recruiting
Mayo Clinic
Rochester 5043473, Minnesota 5037779 55905
United StatesSite Not Available
Children's Mercy Hospital
Kansas City, Missouri 64108
United StatesActive - Recruiting
Children's Mercy Hospital
Kansas City 4393217, Missouri 4398678 64108
United StatesSite Not Available
University of Nebraska Medical Center
Omaha, Nebraska 68198
United StatesActive - Recruiting
University of Nebraska Medical Center
Omaha 5074472, Nebraska 5073708 68198
United StatesSite Not Available
Morristown Medical Center
Morristown, New Jersey 07960
United StatesActive - Recruiting
Morristown Medical Center
Morristown 5101427, New Jersey 5101760 07960
United StatesSite Not Available
Children's Hospital at Montefiore
Bronx, New York 10467
United StatesSite Not Available
Columbia University
New York, New York 10027
United StatesSite Not Available
NYP/Columbia University Medical Center
New York, New York 10027
United StatesActive - Recruiting
Children's Hospital at Montefiore
The Bronx, New York 10467
United StatesActive - Recruiting
NYP/Columbia University Medical Center
New York 5128581, New York 5128638 10027
United StatesSite Not Available
Children's Hospital at Montefiore
The Bronx 5110266, New York 5128638 10467
United StatesSite Not Available
Duke Clinical Research Institute
Durham, North Carolina 27701
United StatesActive - Recruiting
Duke Clinical Research Institute
Durham 4464368, North Carolina 4482348 27701
United StatesSite Not Available
Oregon Health & Science University
Portland, Oregon 97239
United StatesActive - Recruiting
Oregon Health & Science University
Portland 5746545, Oregon 5744337 97239
United StatesSite Not Available
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesActive - Recruiting
Children's Hospital of Philadelphia
Philadelphia 4560349, Pennsylvania 6254927 19104
United StatesSite Not Available
LeBonheur Children's Hospital
Memphis, Tennessee 38103
United StatesActive - Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee 37235
United StatesActive - Recruiting
LeBonheur Children's Hospital
Memphis 4641239, Tennessee 4662168 38103
United StatesSite Not Available
Vanderbilt University Medical Center
Nashville 4644585, Tennessee 4662168 37235
United StatesSite Not Available
Dell Children's Hospital
Austin, Texas 78723
United StatesActive - Recruiting
UT Southwestern
Dallas, Texas 75390
United StatesActive - Recruiting
Dell Children's Hospital
Austin 4671654, Texas 4736286 78723
United StatesSite Not Available
UT Southwestern
Dallas 4684888, Texas 4736286 75390
United StatesSite Not Available
Children's Wisconsin
Milwaukee, Wisconsin 53226
United StatesSite Not Available
Children's Wisconsin
Milwaukee 5263045, Wisconsin 5279468 53226
United StatesSite Not Available

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