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AzTrials

Obstructive Hypertrophic Cardiomyopathy (oHCM)Phase 3SEQUOIA-HCMNCT05186818

Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM

A Phase 3 study testing Aficamten (5 mg, 10 mg, 15 mg, and 20 mg) in people with Obstructive Hypertrophic Cardiomyopathy (oHCM), run by Cytokinetics at 114 sites in 14 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

The purpose of this study is to evaluate the efficacy and safety of aficamten (CK-3773274) versus placebo in adults with symptomatic hypertrophic cardiomyopathy (HCM) and left ventricular outflow tract obstruction. Read more about this study

Who is it for?
Ages 18 to 85 years
What is being tested?
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
282 participants
Where
114 sitesin 14 countries
Study dates
1 Feb 2022to 18 Dec 2023
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Males and females between 18 and 85 years of age, inclusive, at screening.
  • Body mass index <35 kg/m2.
  • Diagnosed with HCM per the following criteria:
  • Has LV hypertrophy and non-dilated LV chamber in the absence of other cardiac disease and
  • Has an end-diastolic LV wall thickness as measured by the echocardiography core laboratory of:
  • ≥15 mm in one or more myocardial segments OR
Show all 13Show fewer
  • ≥13 mm in one or more wall segments and a known-disease-causing gene mutation or positive family history of HCM
  • Has resting LVOT-G ≥30 mmHg and post-Valsalva LVOT G ≥50 mmHg during screening as determined by the echocardiography core laboratory.
  • LVEF ≥60% at screening as determined by the echocardiography core laboratory.
  • New York Heart Association (NYHA) Functional Class II or III at screening.
  • Hemoglobin ≥10g/dL at screening.
  • Respiratory exchange ratio (RER) ≥1.05 and pVO2 ≤90% predicted on the screening CPET per the core laboratory.
  • Patients on beta-blockers, verapamil, diltiazem, or disopyramide should have been on stable doses for >6 weeks prior to randomization and anticipate remaining on the same medication regimen during the trial. Patients treated with disopyramide must also be concomitantly treated with a beta blocker and/or calcium channel blocker.

Who cannot join

  • Known or suspected infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics oHCM (eg, Noonan syndrome, Fabry disease, amyloidosis).
  • Significant valvular heart disease (per investigator judgment).
  • Moderate-severe valvular aortic stenosis.
  • Moderate-severe mitral regurgitation not due to systolic anterior motion of the mitral valve.
  • History of LV systolic dysfunction (LVEF <45%) or stress cardiomyopathy at any time during their clinical course.
  • Inability to exercise on a treadmill or bicycle (eg, orthopedic limitations).
Show all 13Show fewer
  • Has been treated with septal reduction therapy (surgical myectomy or percutaneous alcohol septal ablation) or has plans for either treatment during the trial period.
  • Documented paroxysmal atrial fibrillation during the screening period.
  • Paroxysmal or permanent atrial fibrillation is only excluded IF:
  • rhythm restoring treatment (eg, direct-current cardioversion, atrial fibrillation ablation procedure, or antiarrhythmic therapy) has been required ≤6 months prior to screening.
  • rate control and anticoagulation have not been achieved for at least 6 months prior to screening.
  • History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to screening.
  • Has received prior treatment with CK-3773274 or mavacamten.

Trial locations and contacts

114 sites in 14 countries.

114 sites in 14 countries

United States48 sites

  • Alaska Heart and Vascular Institute

    Anchorage, Alaska, 99508

  • Michigan Medicine

    Ann Arbor, Michigan, 48109

  • Emory Clinic

    Atlanta, Georgia, 30322

  • MedStar Medical Group Cardiology at Union Memorial

    Baltimore, Maryland, 21218

  • Medstar Franklin Square Medical Center

    Baltimore, Maryland, 21237

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115

  • Lahey Hospital & Medical Center

    Burlington, Massachusetts, 01805

  • Sanger Heart and Vascular Institute

    Charlotte, North Carolina, 10595

  • Sanger Heart and Vascular Institute - HCM Clinic

    Charlotte, North Carolina, 28204

  • University of Virginia Health System University Hospital

    Charlottesville, Virginia, 22908

  • Northwestern University

    Chicago, Illinois, 60614

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • The Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210

  • The University of Texas Southwestern Medical Center

    Dallas, Texas, 75390

  • Henry Ford Medical Center

    Detroit, Michigan, 48202

  • Piedmont Fayette Hospital

    Fayetteville, Georgia, 30214

  • Holy Cross Hospital / Cardiology Associates

    Fort Lauderdale, Florida, 33308

  • Stern Cardiovascular Foundation, Inc

    Germantown, Tennessee, 38138

  • Spectrum Health Medical Group Cardiovascular Medicine

    Grand Rapids, Michigan, 49525

  • Houston Methodist Hospital

    Houston, Texas, 77030

  • University of Iowa

    Iowa City, Iowa, 52242

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224

  • Saint Luke's Hospital of Kansas City

    Kansas City, Missouri, 64111

  • UC San Diego Health - Sulpizio Cardiovascular Center

    La Jolla, California, 92037

  • Cedars-Sinai Medical Center - Smidt Heart Institute Clinic

    Los Angeles, California, 90048

  • Froedtert Hospital

    Milwaukee, Wisconsin, 53226

  • M Health Fairview University of Minnesota Medical Center - East Bank

    Minneapolis, Minnesota, 55455

  • Morristown Medical Center

    Morristown, New Jersey, 07960

  • Duke Health Center Arringdon

    Morrisville, North Carolina, 27560

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Yale School of Medicine

    New Haven, Connecticut, 06511

  • Yale University School of Medicine

    New Haven, Connecticut, 06519

  • NYU Langone Health

    New York, New York, 10016

  • Mount Sinai Hospital

    New York, New York, 10029

  • Columbia University Medical Center/NY Presbyterian Hospital

    New York, New York, 10032

  • Hospital of the University of Pennsylvania (University of Pennsylvania School of Medicine)

    Philadelphia, Pennsylvania, 19104

  • UPMC Presbyterian

    Pittsburgh, Pennsylvania, 15213

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • University of Utah Hospital

    Salt Lake City, Utah, 84132

  • University of California San Francisco

    San Francisco, California, 94143

  • University of Washington Medical

    Seattle, Washington, 98195

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • Stanford University Hospital

    Stanford, California, 94305

  • Ascension St. John Clinical Research Institute

    Tulsa, Oklahoma, 74104

  • Westchester Medical Center

    Valhalla, New York, 10595

  • MedStar Washington Hospital Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

China9 sites

  • Beijing Chao-Yang Hospital, Capital Medical University

    Beijing, Beijing Municipality, 100020

  • Beijing Anzhen Hospital

    Beijing, Beijing Municipality, 100029

  • Peking University Third Hospital

    Beijing, Beijing Municipality, 10019

  • The First Hospital of Jinlin University

    Changchun, Jinlin, 130031

  • Xiangya Hospital of the Central South University

    Changsha, Hunan, 410008

  • Nanfang Hospital Southern Medical University

    Guanzhou, Guangdong, 516006

  • Union Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology

    Wuhan, Hubei, 430022

  • The First Affiliated Hospital of Xi'an Jiaotong University

    Xi’an, Shanxi, 710061

  • The First Affiliated Hospital of Zhengzhou University

    Zhengzhou, Henan, 450018

Other trials in China

Germany9 sites

  • Kerckhoff-Klinik GmbH Abteilung Administration Forschung und Lehre

    Bad Nauheim, 61231

  • Charite-Universitaetsmedizin Berlin Campus Virchow-Klinikum (CVK) Medizinische Klinik mit Schwerpunkt Kardiologie

    Berlin, 13353

  • Universitaetsklinikum Essen Westdeutsches Herz-Gefaesszentrum

    Essen, 45147

  • Klinikumder Georg-August-Universitaet

    Göttingen, 37075

  • Universitaetsmedizin Goettingen

    Göttingen, 37075

  • Universitaetsklinikum Heidelberg

    Heidelberg

  • Universitaetsklinikum Jena Klinik fuer Innere Medizin 1

    Jena, 07747

  • Unniversitaetsklinikum Magdeburg Klinik fur Kardiologie und Anglologie

    Magdeburg, 39120

  • Universitätsklinikum Würzburg

    Würzburg

Other trials in Germany

France8 sites

  • CHU de Marseille - Hopital de la Timone Service de cardiologie

    Marseille, 13005

  • CHU de Nantes

    Nantes, 44093

  • Hopital Lariboisiere, Service de Cardiologie

    Paris, 75010

  • Hôpital Européen Georges Pompidou

    Paris, 75015

  • CHU Pitie-Salpetriere, Centre de reference maladies cardiaques hereditaires ou rares

    Paris

  • CHU de Bordeaux Hopital Cardiologique Haut-Leveque

    Pessac, 33600

  • CHU du Haut Lveque Cardiologie

    Pessac, 33604

  • Hopital Universitaire de Rangueil (CHU de Toulouse) Service de Cardiologie

    Toulouse, Cedex, 31059

Other trials in France

United Kingdom8 sites

  • Queen Elizabeth Hospital Birmingham University Hospitals Birmingham NHS Foundation Trust

    Edgbaston, Birmingham, B15 2GW

  • Queen Elizabeth University Hospital - HWS Greater Glasgow and Clyde

    Glasgow, G1 4TF

  • Liverpool Heart and Chest Hospital - Liverpool Heart and Chest Hospital NHS Foundation Trust- HWS Greater Glasgow and Clyde

    Liverpool, L14 3LB

  • Barts Health NS Trust

    London, EC1A 7BE

  • St George's University Hospitals NHS Foundation Trust

    London, SW17 0QT

  • Royal Brompton Hospital

    London, SW3 6NP

  • Oxford Centre for Clinical Magnetic Resonance Research

    Oxford, OX3 9DU

  • Radcliffe Department of Medicine

    Oxford, OX3 9DU

Other trials in United Kingdom

Spain7 sites

  • Complejo Hospitalario Universitario

    A Coruña, La Coruna, 15006

  • Hospital Clinic de Barcelona

    Barcelona, 08036

  • Hospital Universitario Puerta de Hierro

    Madrid, Majadahonda

  • Hospital Clinico Universitario Virgen de la Arrixaca

    Murcia

  • Hospital Universitario Son Llatzer Secretaria de Cardiologia

    Palma, 07198

  • Hospital Universitario de Salamanca

    Salamanca, 37007

  • Hospital Universitario Virgen Macarena-merge

    Seville, 41009

Other trials in Spain

Israel6 sites

  • The Barzilai University Medical Center

    Ashkelon, 78100

  • The Barzilai University Medical Center

    Ashkelon, 7830604

  • Hadassah Medical Center- Ein Kerem

    Jerusalem, 9112001

  • The Olga & Lev Leviev Heart Center The Chaim Sheba Medical Center

    Ramat Gan, Tel Hasomer, 5265601

  • Kaplan Medical Center

    Rehovot, 76100

  • Ziv Medical Center

    Safed, 13100

Other trials in Israel

Denmark4 sites

  • Aalborg University Hospital, Department of Cardiology

    Aalborg, 9100

  • Department of Cardiology Aarhus University Hospital

    Aarhus, 8200

  • Hjertecentret (The Heart Center)

    Copenhagen, 2100

  • Copenhagen University Hospital

    Copenhagen, 2400

Other trials in Denmark

Italy4 sites

  • UOC Cardiologica Azienda Ospedaliera Spedali Civili di Brescia

    Brescia, 25123

  • Azienda Ospedaliero Universitaria Careggi Dipartimento Cardiotoracovascolare - Cardiomiopatie Unit

    Florence, 50134

  • Fondazione toscana Gabriele Monesterio per la ricarca medica Dipartimento Cardiotoracico UOC Cardiologia e Medicina Cardiovasculare Ospedale San Cataldo

    Pisa, 56124

  • Dipartimento di Medicina Clinica e Molecolare Universita Sapienza di Roma Unita di Terapia Intensiva Cardiologica Azienda Ospedaliero Universitaria Sant'Andrea

    Roma, 1035-39

Other trials in Italy

Netherlands3 sites

  • Amsterdam UMC, location AMC

    Amsterdam, 1105

  • University Hospital Maastricht

    Maastricht, 6229HX

  • Erasmus Medical Center

    Rotterdam, 3015

Other trials in Netherlands

Poland3 sites

  • Kardio Brynow S.C

    Katowice

  • Krakowskie Centrum

    Krakow

  • Narodowy Instytut Kardiologii Stefana Kardynała Wyszynskiego- Panstwowy Instytut Badawczy

    Warsaw, Masovian Voivodeship, 04-628

Other trials in Poland

Czechia2 sites

  • Charles University

    Prague, 128 08

  • Interni klinika kardiologie a angiologie

    Prague, 12808

Other trials in Czechia

Portugal2 sites

  • Centro Hospitalar Do Baixo Vouga, EPE AV ARTUR RAVARA

    Aveiro, 3814-501

  • Hospital da Luz

    Lisbon, 1500-650

Other trials in Portugal

Hungary1 site

  • Semmelweis Egyetem Varosmajori Sziv es Ergyogyasazati Klinika

    Budapest

Other trials in Hungary

About this study

Official title: A Phase 3, Multi-Center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Efficacy and Safety of CK-3773274 in Adults With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction

The purpose of this study is to evaluate the efficacy and safety of aficamten (CK-3773274) versus placebo in adults with symptomatic hypertrophic cardiomyopathy (HCM) and left ventricular outflow tract obstruction.

Read the detailed description

CY 6031 was a Phase 3, randomized, placebo-controlled, double-blind, multi-center trial in participants with symptomatic oHCM. Eligible participants were randomized in a 1:1 ratio to receive aficamten or placebo. Randomization was stratified by use of beta-blockers (yes or no) and cardiopulmonary exercise testing (CPET) exercise modality (treadmill or bicycle). Enrollment limits were applied as follows: participants taking beta-blockers were capped at approximately 70% of total enrollment; participants taking disopyramide were capped at approximately 10% of total enrollment; participants with persistent atrial fibrillation (AF) at screening were capped at approximately 15% of total enrollment; and participants using the bicycle CPET exercise modality were capped at approximately 50% of total enrollment.

Investigational product (IP) was administered orally once daily (QD) with or without food for 24 weeks. During the initial 6 weeks of the treatment period, IP doses were individually titrated at Weeks 2, 4, and 6 based on echocardiography-guided criteria. Dose escalation at Weeks 2, 4, and 6 occurred only if a participant had a Valsalva left ventricular outflow tract gradient (LVOT-G) ≥ 30 mmHg and a biplane left ventricular ejection fraction (LVEF) ≥ 55%. Echocardiograms were performed at each subsequent visit during the trial, and the IP dose was down-titrated if the LVEF was < 50%. The primary endpoint of peak oxygen uptake (pVO2) was measured by CPET at screening and at the end of treatment (Week 24). A participant's background HCM therapy was individually optimized according to local practice prior to enrollment in the study.

Being tested
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg), Placebo to match aficamten
Keywords
Obstructive Hypertrophic Cardiomyopathy, oHCM, CK-3773274, CK-274, Aficamten, SEQUOIA-HCM, CY 6031, SEQUOIA
Medical subject headings
Cardiomyopathy, Hypertrophic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Aficamten (5 mg, 10 mg, 15 mg, and 20 mg)Drug

    Also called CK-3773274

    Aficamten tablets were administered orally once daily.

  • Placebo to match aficamtenDrug

    Also called Placebo

    Placebo tablets were administered orally once daily.

Study groups in NCT05186818
Study groupRoleWhat they receive
Aficamten up to 20 mgExperimentalParticipants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Receives: Drug: Aficamten (5 mg, 10 mg, 15 mg, and 20 mg)
Placebo to match aficamtenPlacebo comparatorParticipants received placebo for up to 24 weeks.
Receives: Drug: Placebo to match aficamten

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change From Baseline in pVO2 at Week 24

    The effect of CK-3773274 on exercise capacity in participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) was determined through changes in peak oxygen uptake (pVO2) after 24 weeks of treatment. pVO2 was measured by cardiopulmonary exercise testing (CPET) on a treadmill or bicycle. A higher pVO2 indicates better cardiorespiratory fitness.

    Measured: Baseline to Week 24

Secondary outcomes (10)

  1. Change From Baseline in KCCQ-CSS at Week 24

    The effect of aficamten on patient health status was assessed using the Kansas City Cardiomyopathy Questionnaire (KCCQ), a patient-reported outcome designed to assess the physical limitations, symptoms, self-efficacy, social limitation, and quality of life of patients with heart failure symptoms. The KCCQ-Clinical Symptoms Score (KCCQ-CSS) is scored on a scale from 0 to 100, with higher scores indicating better physical functioning and symptoms.

    Measured: Baseline to Week 24

  2. Change From Baseline in KCCQ-CSS at Week 12

    The effect of aficamten on patient health status was assessed using the Kansas City Cardiomyopathy Questionnaire (KCCQ), a patient-reported outcome designed to assess the physical limitations, symptoms, self-efficacy, social limitation, and quality of life of patients with heart failure symptoms. The KCCQ-Clinical Symptoms Score (KCCQ-CSS) is scored on a scale from 0 to 100, with higher scores indicating better physical functioning and symptoms.

    Measured: Baseline to Week 12

  3. Proportion of Participants With ≥1 Class Improvement in New York Heart Association (NYHA) Functional Class From Baseline to Week 24

    The effect of aficamten on NYHA functional classification was evaluated through changes observed from baseline through 24 weeks of treatment.

    Measured: Baseline to Week 24

  4. Proportion of Participants With ≥1 Class Improvement in NYHA Functional Class From Baseline to Week 12

    The effect of aficamten on NYHA functional classification was evaluated through changes observed from baseline through 12 weeks of treatment.

    Measured: Baseline to Week 12

  5. Change From Baseline in Valsalva Left Ventricular Outflow Tract Gradient (LVOT-G) at Week 24

    The effect of aficamten treatment on Valsalva LVOT-G was evaluated through changes from baseline to Week 24.

    Measured: Baseline to Week 24

  6. Change From Baseline in Valsalva LVOT-G at Week 12

    The effect of aficamten treatment on Valsalva LVOT-G was evaluated through changes from baseline to Week 12.

    Measured: Baseline to Week 12

  7. Proportion of Participants With Valsalva LVOT G <30 mmHg at Week 24

    The effect of aficamten treatment on Valsalva LVOT-G was evaluated through changes from baseline to Week 24.

    Measured: Baseline to Week 24

  8. Proportion of Participants With Valsalva LVOT G <30 mmHg at Week 12

    The effect of aficamten treatment on Valsalva LVOT-G was evaluated through changes from baseline to Week 12.

    Measured: Baseline to Week 12

  9. Duration of SRT Eligibility During the 24-week Treatment Period for Participants Who Were SRT Eligible at Baseline

    The effect of aficamten treatment on the duration of septal reduction therapy (SRT) eligibility was evaluated over the 24- week treatment period.

    Measured: Baseline to Week 24

  10. Change From Baseline to Week 24 in Total Workload During CPET

    Effect of aficamten on intensity of exercise (based on speed, incline, participant weight, etc.) during CPET. Workload is an indication of the energy expended during the exercise test.

    Measured: Baseline to Week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Jan 2022
  2. Study start1 Feb 2022
  3. Primary completion10 Nov 2023
  4. Study completion18 Dec 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
282 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05186818
Sponsor's protocol ID
CY 6031
Other IDs
2021-003536-92
Acronym
SEQUOIA-HCM
Status last verified
Feb 2025
First submitted
23 Dec 2021
Last updated
3 Mar 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Masri A, Lewis GD, Barriales-Villa R, Claggett BL, Coats CJ, Elliott P, Hagege A, Kulac IJ, Garcia-Pavia P, Fifer MA, Meder B, Olivotto I, Nassif MA, Lakdawala NK, Owens AT, Heitner SB, Jacoby DL, Sohn R, Kupfer S, Malik FI, Wohltman A, Maron MS. The Threshold for a Clinically Meaningful Improvement in Cardiopulmonary Exercise Testing Measures for Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy. J Card Fail. 2026 Aug 20:S1071-9164(26)00500-2. doi: 10.1016/j.cardfail.2026.08.011. Online ahead of print.

    PubMed 42624353

  • Kaski JP, Kantor PF, Nakano SJ, Olivotto I, Russell MW, Godown J, Chiu M, German P, Heitner SB, Jacoby DL, Kupfer S, Lutz J, Maharao N, Malik FI, Melloni C, Nieto Morales PF, Simkins T, Wei J, Ho CY; CEDAR-HCM Investigators. Efficacy and Safety of Aficamten in Children and Adolescents With Obstructive Hypertrophic Cardiomyopathy: Study Design and Rationale of CEDAR-HCM. Circ Heart Fail. 2026 Feb;19(2):e013418. doi: 10.1161/CIRCHEARTFAILURE.125.013418. Epub 2025 Dec 5.

    PubMed 41347307

  • Campain J, Griskowitz C, Newlands C, Claggett BL, Kulac IJ, McGinnis S, Giverts I, Moreno F, Minasian A, Prasad C, Rupert L, Landsteiner I, Iskenderian N, Coats CJ, Lee MMY, Maron MS, Owens AT, Jacoby DL, Heitner SB, Kupfer S, Malik FI, Wohltman A, Malhotra R, Lewis GD. Characterization and Application of Novel Exercise Recovery Patterns That Reflect Cardiac Performance: A Substudy of the SEQUOIA-HCM Trial. Circulation. 2025 Oct 7;152(14):990-1002. doi: 10.1161/CIRCULATIONAHA.124.073585. Epub 2025 Sep 5.

    PubMed 40910168

  • Maharao N, Xu D, Simkins TJ, Bowles O, Liu G, Benattia Y, Griffith A, Heitner SB, Kupfer S, German P. Clinical Evaluation of the Effect of Aficamten on QT/QTc Interval in Healthy Participants. Clin Transl Sci. 2025 Apr;18(4):e70218. doi: 10.1111/cts.70218.

    PubMed 40200648

  • Maron MS, Masri A, Nassif ME, Barriales-Villa R, Abraham TP, Arad M, Cardim N, Choudhury L, Claggett B, Coats CJ, Dungen HD, Garcia-Pavia P, Hagege AA, Januzzi JL, Kulac I, Lee MMY, Lewis GD, Ma CS, Michels M, Oreziak A, Owens AT, Spertus JA, Solomon SD, Tfelt-Hansen J, van Sinttruije M, Veselka J, Watkins HC, Jacoby DL, Heitner SB, Kupfer S, Malik FI, Meng L, Wohltman A, Olivotto I; SEQUOIA-HCM Investigators. Impact of Aficamten on Disease and Symptom Burden in Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM. J Am Coll Cardiol. 2024 Nov 5;84(19):1821-1831. doi: 10.1016/j.jacc.2024.09.003. Epub 2024 Sep 30.

    PubMed 39352339

  • Lee MMY, Masri A, Nassif ME, Barriales-Villa R, Abraham TP, Claggett BL, Coats CJ, Gimeno JR, Kulac IJ, Landsteiner I, Ma C, Maron MS, Olivotto I, Owens AT, Solomon SD, Veselka J, Jacoby DL, Heitner SB, Kupfer S, Malik FI, Meng L, Wohltman A, Lewis GD; SEQUOIA-HCM Investigators. Aficamten and Cardiopulmonary Exercise Test Performance: A Substudy of the SEQUOIA-HCM Randomized Clinical Trial. JAMA Cardiol. 2024 Nov 1;9(11):990-1000. doi: 10.1001/jamacardio.2024.2781.

    PubMed 39230885

  • Sherrod CF 4th, Saberi S, Nassif ME, Claggett BL, Coats CJ, Garcia-Pavia P, Januzzi JL, Lewis GD, Ma C, Maron MS, Miao ZM, Olivotto I, Veselka J, Butzner M, Jacoby DL, Heitner SB, Kupfer S, Malik FI, Meng L, Wohltman A, Spertus JA. Effect of Aficamten on Health Status Outcomes in Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM. J Am Coll Cardiol. 2024 Nov 5;84(19):1773-1785. doi: 10.1016/j.jacc.2024.08.014. Epub 2024 Sep 1.

    PubMed 39217569

  • Masri A, Cardoso RN, Abraham TP, Claggett BL, Coats CJ, Hegde SM, Kulac IJ, Lee MMY, Maron MS, Merkely B, Michels M, Olivotto I, Oreziak A, Jacoby DL, Heitner SB, Kupfer S, Malik FI, Meng L, Solomon SD, Wohltman A, Kwong RY, Kramer CM; SEQUOIA-HCM Investigators. Effect of Aficamten on Cardiac Structure and Function in Obstructive Hypertrophic Cardiomyopathy: SEQUOIA-HCM CMR Substudy. J Am Coll Cardiol. 2024 Nov 5;84(19):1806-1817. doi: 10.1016/j.jacc.2024.08.015. Epub 2024 Sep 1.

    PubMed 39217563

  • Hegde SM, Claggett BL, Wang X, Jering K, Prasad N, Roshanali F, Masri A, Nassif ME, Barriales-Villa R, Abraham TP, Cardim N, Coats CJ, Kramer CM, Maron MS, Michels M, Olivotto I, Saberi S, Jacoby DL, Heitner SB, Kupfer S, Meng L, Wohltman A, Malik FI, Solomon SD; SEQUOIA-HCM Investigators. Impact of Aficamten on Echocardiographic Cardiac Structure and Function in Symptomatic Obstructive Hypertrophic Cardiomyopathy. J Am Coll Cardiol. 2024 Nov 5;84(19):1789-1802. doi: 10.1016/j.jacc.2024.08.002. Epub 2024 Sep 1.

    PubMed 39217556

  • Coats CJ, Masri A, Barriales-Villa R, Abraham TP, Brinkley DM, Claggett BL, Hagege A, Hegde SM, Ho CY, Kulac IJ, Lee MMY, Maron MS, Olivotto I, Owens AT, Solomon SD, Tfelt-Hansen J, Watkins H, Jacoby DL, Heitner SB, Kupfer S, Malik FI, Meng L, Wohltman A, Januzzi JL. Cardiac biomarkers and effects of aficamten in obstructive hypertrophic cardiomyopathy: the SEQUOIA-HCM trial. Eur Heart J. 2024 Nov 8;45(42):4464-4478. doi: 10.1093/eurheartj/ehae590.

    PubMed 39217447

  • Coats CJ, Masri A, Nassif ME, Barriales-Villa R, Arad M, Cardim N, Choudhury L, Claggett B, Dungen HD, Garcia-Pavia P, Hagege AA, Januzzi JL, Lee MMY, Lewis GD, Ma CS, Maron MS, Miao ZM, Michels M, Olivotto I, Oreziak A, Owens AT, Spertus JA, Solomon SD, Tfelt-Hansen J, van Sinttruije M, Veselka J, Watkins H, Jacoby DL, German P, Heitner SB, Kupfer S, Lutz JD, Malik FI, Meng L, Wohltman A, Abraham TP; SEQUOIA-HCM Investigators *. Dosing and Safety Profile of Aficamten in Symptomatic Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM. J Am Heart Assoc. 2024 Aug 6;13(15):e035993. doi: 10.1161/JAHA.124.035993. Epub 2024 Jul 26.

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  • Maron MS, Masri A, Nassif ME, Barriales-Villa R, Arad M, Cardim N, Choudhury L, Claggett B, Coats CJ, Dungen HD, Garcia-Pavia P, Hagege AA, Januzzi JL, Lee MMY, Lewis GD, Ma CS, Michels M, Olivotto I, Oreziak A, Owens AT, Spertus JA, Solomon SD, Tfelt-Hansen J, van Sinttruije M, Veselka J, Watkins H, Jacoby DL, Heitner SB, Kupfer S, Malik FI, Meng L, Wohltman A, Abraham TP; SEQUOIA-HCM Investigators. Aficamten for Symptomatic Obstructive Hypertrophic Cardiomyopathy. N Engl J Med. 2024 May 30;390(20):1849-1861. doi: 10.1056/NEJMoa2401424. Epub 2024 May 13.

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  • Coats CJ, Maron MS, Abraham TP, Olivotto I, Lee MMY, Arad M, Cardim N, Ma CS, Choudhury L, Dungen HD, Garcia-Pavia P, Hagege AA, Lewis GD, Michels M, Oreziak A, Owens AT, Tfelt-Hansen J, Veselka J, Watkins HC, Heitner SB, Jacoby DL, Kupfer S, Malik FI, Meng L, Wohltman A, Masri A; SEQUOIA-HCM Investigators. Exercise Capacity in Patients With Obstructive Hypertrophic Cardiomyopathy: SEQUOIA-HCM Baseline Characteristics and Study Design. JACC Heart Fail. 2024 Jan;12(1):199-215. doi: 10.1016/j.jchf.2023.10.004. Epub 2023 Nov 29.

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Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Aficamten (5 mg, 10 mg, 15 mg, and 20 mg) in people with Obstructive Hypertrophic Cardiomyopathy (oHCM), run by Cytokinetics at 114 sites in 14 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 3 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 114 sites in United States, China, Germany, France, United Kingdom and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 3 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in pVO2 at Week 24 (time frame: Baseline to Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05186818, which also lists 13 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Mar 2026.

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