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Symptomatic Hypertrophic Cardiomyopathy (HCM)Phase 2/3FOREST-HCMNCT04848506

Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Aficamten in Adults With HCM

A Phase 2/3 study testing Aficamten (5 - 20 mg) in people with Symptomatic Hypertrophic Cardiomyopathy (HCM), run by Cytokinetics at 141 sites in 19 countries.

Enrolling by invitation

Enrolling by invitation. The study team selects and invites participants, usually from an existing group of patients.

Sites and contacts
Official record
Search all trials

Trial summary

The purpose of this study is to collect long-term safety and tolerability data for aficamten. Read more about this study

Who is it for?
Ages 18 to 85 years
What is being tested?
Aficamten (5 - 20 mg)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
900 participants
Where
141 sitesin 19 countries
Study dates
6 May 2021to Mar 2029
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

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

  • Completion of a Cytokinetics trial investigating aficamten
  • LVEF ≥ 55% at the Screening Visit

Who cannot join

  • Has received treatment with mavacamten: (a) within 56 days prior to dosing and (b) has not received approval for participation from the Medical Monitor.
  • Has participated in another investigational device or drug study or received an investigational device or drug < 1 month (or 5 half-lives for drugs, whichever is longer) prior to screening. Other investigational procedures while participating in this study are not permitted.
  • Since completion of a previous trial of aficamten has:
  • Developed new-onset paroxysmal or permanent atrial fibrillation requiring rhythm restoring treatment (eg, direct-current cardioversion, ablation procedure, or antiarrhythmic therapy) < 30 days prior to screening. Patient may re-screen for CY 6022 after 30 days if heart rate (HR) < 100 bpm and/or rhythm is stable > 30 days
  • Undergone septal reduction therapy (surgical myectomy or transcatheter alcohol ablation)
  • Had a confirmed LVEF < 40% with an associated dose interruption during participation in a prior study with aficamten
  • History of implantable ICD placement within 30 days prior to screening.

Trial locations and contacts

141 sites in 19 countries. Contact the study team or a site near you to ask about taking part.

141 sites in 19 countries

United States57 sites

  • Alaska Heart and Vascular Institute

    Anchorage, Alaska, 99508

  • Michigan Medicine - University of Michigan

    Ann Arbor, Michigan, 48109

  • University of Michigan Health System (UMHS) - University Hospital (University of Michigan Medical Center)

    Ann Arbor, Michigan, 48109

  • Emory Clinic

    Atlanta, Georgia, 30322

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35233

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115

  • Lahey Hospital & Medical Center

    Burlington, Massachusetts, 01805

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

  • Sanger Heart & Vascular Institute, Atrium Health

    Charlotte, North Carolina, 28204

  • University of Virginia Health System University Hospital

    Charlottesville, Virginia, 22908

  • Northwestern University

    Chicago, Illinois, 60611

  • The Linder Research Center at The Christ Hospital

    Cincinnati, Ohio, 45219

  • University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • The University of Texas Southwestern Medical Center at Dallas

    Dallas, Texas, 75390

  • Henry Ford Hospital

    Detroit, Michigan, 48202

  • Duke Cardiology at Southpoint

    Durham, North Carolina, 27710

  • Inova Health Care Services

    Falls Church, Virginia, 22042

  • Piedmont Fayette Hospital

    Fayetteville, Georgia, 30214

  • Holy Cross Hospital / Cardiology Associates

    Fort Lauderdale, Florida, 33308

  • Greenville Memorial Hospital

    Greenville, South Carolina, 29605

  • The Queen's Medical Center

    Honolulu, Hawaii, 96813

  • CHI St. Luke's Health Baylor College of Medicine Medical Center

    Houston, Texas, 77030

  • Houston Methodist Hospital

    Houston, Texas, 77030

  • The University of Iowa Hospitals & Clinics

    Iowa City, Iowa, 52242

  • The University of Kansas Medical Center

    Kansas City, Kansas, 66160

  • Saint Luke's Hospital of Kansas City

    Kansas City, Missouri, 64111

  • UC San Diego Health - Sulpizio Cardiovascular Center

    La Jolla, California, 92037

  • Cedar-Sinai Medical Center

    Los Angeles, California, 90048

  • Cedars-Sinai Medical Center (Smidt Heart Institute)

    Los Angeles, California, 90048

  • UCLA Health System - UCLA Cardiovascular Center

    Los Angeles, California, 90095

  • Northwell Health North Shore University Hospital

    Manhasset, New York, 11030

  • Aurora St. Luke's Medical Center

    Milwaukee, Wisconsin, 53215

  • University of Minnesota

    Minneapolis, Minnesota, 55455

  • WVUMedicine

    Morgantown, West Virginia, 26506

  • Morristown Medical Center

    Morristown, New Jersey, 07960

  • Intermountain Medical Center

    Murray, Utah, 84107

  • Ascension Saint Thomas Heart West

    Nashville, Tennessee, 37205

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Yale New Haven Hospital

    New Haven, Connecticut, 06519

  • NYU Langone Health

    New York, New York, 10016

  • Weill Cornell Medicine

    New York, New York, 10021

  • Mount Sinai Hospital

    New York, New York, 10029

  • Columbia University Medical Center/New York Presbyterian Hospital

    New York, New York, 10032

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

    Philadelphia, Pennsylvania, 19104

  • Mayo Clinic Building - Phoenix

    Phoenix, Arizona, 85054

  • UPMC Presbyterian

    Pittsburgh, Pennsylvania, 15213

  • Providence St. Vincent Medical Center

    Portland, Oregon, 97225

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • University of Utah Health

    Salt Lake City, Utah, 84132

  • UCSF Medical Center

    San Francisco, California, 94143

  • Virginia Mason Medical Center

    Seattle, Washington, 98101

  • Ascension St. John Clinical Research Institute

    Tulsa, Oklahoma, 76104

  • Westchester Medical Center

    Valhalla, New York, 10595

  • MedStar Washington Hospital Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

Spain12 sites

  • Complejo Hospitalario Universitario A Coruna

    A Coruña, 15006

  • Hospital Clinic Barcelona

    Barcelona, 08036

  • Hospital General Universitario Gregorio Marañón (HGUGM)

    Madrid, 28007

  • Hospital Clinico Universitario San Carlos

    Madrid, 28040

  • Hospital Universitario Puerta de Hierro Majadahonda

    Madrid, 28220

  • Hospital Clinico Universitario Virgen de la Arrixaca

    Madrid, 30120

  • Hospital Universitario Virgen de la Victoria

    Málaga, 29010

  • Hospital Universitario Son Llatzer

    Palma, 07198

  • Complexo Hospitalario Universitario De Vigo

    Pontevedra, 36312

  • Hospital Universitario de Salamanca

    Salamanca, 37007

  • Hospital Universitario Virgen Macarena-merge

    Seville, 41009

  • Hospital Universitario Virgen del Rocío

    Seville, 41013

Other trials in Spain

France9 sites

  • Hopital de la Timone Service de cardiologie

    Marseille, 130005

  • Clinique Du Millenaire

    Montpellier, 340000

  • CHU Montpellier - Hopital Arnaud de Villeneuve

    Montpellier, 34295

  • CHU de Nantes Institut Du Thorax et du systeme nerveux Clinique Cardiologique et des Maladies Vasculaires

    Nantes, 44093

  • Hopital Lariboisiere

    Paris, 75010

  • Hopital Europeen Georges Pompidou

    Paris, 75015

  • Hopital cardiologique de Haut-Leveque

    Pessac, 33604

  • Centre hospitalier universitaire (CHU) de Rennes-Hopital Pontchaillou

    Rennes, 35033

  • Centre Hospitalier universitaire de Toulouse - Rangueil Hospital

    Toulouse, 31059

Other trials in France

United Kingdom8 sites

  • Queen Elizabeth Hospital Birmingham University Hospitals Birmingham NHS Foundation Trust

    Birmingham, B15 2GW

  • Ninewells Hospital and Medical School

    Dundee, DD2 1SG

  • Glasgow Clinical Research Facility Neuroscience Institute

    Glasgow, G51 4TF

  • Liverpool Heart and Chest Hospital

    Liverpool, L14 3PE

  • St Bartholomew's Hospital

    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 (OCMR) Division of Cardiovascular Medicine Radcliffe Department of Medicine University of Oxford

    Oxford, OX3 9DU

Other trials in United Kingdom

Italy7 sites

  • Unit Cardiomiopatie, Dipartimento Cardio ToracoVascolare

    Florence, 50134

  • IRCCS Policlinico San Donato

    Milan, 20097

  • ASST Grande Ospedale Metropolitano Niguarda

    Milan, 20162

  • Azienda Ospedaliera Universitaria Federico II di Napoli

    Naples, 80131

  • 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, 00189

  • Azienda Sanitaria Universitaria Giuliano Isontina

    Trieste, 34149

Other trials in Italy

Brazil6 sites

  • Nucleo de Pesquisa Clinica NUPEC

    Belo Horizonte, Minas Gerais, 30220

  • Associacao Lar Sao Francisco de Assis na Providencia de Deus - Hospital Universitario Sao Francisco na Providencia de Deus (HUSF)

    Bragança Paulista, São Paulo, 12916-542

  • IPECC - Instituto De Pesquisa Clinica de Campinas

    Campinas, São Paulo, 13060-080

  • Centro Avancado de Pesquisa-Estudo para-Diagnostico-CAPED

    Ribeirão Preto, São Paulo, 14026-020

  • Universidade de Sao Paulo (USP) Hospital das Clinicas da Faculdade de Medicina Instituto do Coracao (InCor)

    São Paulo, São Paulo, 5403000

  • Instituto Dante Pazzanese de Cardiologia

    São Paulo, 04012-909

Other trials in Brazil

Germany6 sites

  • Charite-Universitaetsmedizin Berlin

    Berlin, 13353

  • Universitaetsklinikum Essen

    Essen, 45147

  • Universitaetsmedizin Goettingen Klinik fur Kardiologie und Pneumologie

    Goettigen, 37075

  • Universitaesklinkum Heidelberg

    Heidelberg, 69120

  • Universitaetsklinikum Jena

    Jena, 07747

  • Universitaetskinikum Wuerzburg Deutsches Zentrum fur Herzinsuffzienz Wurzburg (DZHI) Comprehensive Heart Failure Center (CHFC)

    Würzburg, 97078

Other trials in Germany

Canada5 sites

  • Royal Alexandra Hospital

    Edmonton, T5H 3V9

  • QEII Health Science Centre (Halifax Infirmary site)

    Halifax, B3H 3A7

  • Montreal Heart Institute

    Montreal, Quebec, H1T 1C8

  • Institut universitaire de cardiologie et de pneumologie de Quebec-Universite Laval

    Québec, Quebec, G1V 4G5

  • St. Boniface General Hospital

    Winnipeg, Manitoba, R2H 2A6

Other trials in Canada

Denmark5 sites

  • Department of Cardiology Aalborg University Hospital

    Aalborg, 9000

  • Department of Cardiology Aarhus University Hospital

    Aarhus, 8200

  • The Heart Center, Department of Cardiology

    Copenhagen, 2100

  • Copenhagen University Hospital

    Copenhagen, 2400

  • Odense University Hospital

    Odense, 5000

Other trials in Denmark

Israel5 sites

  • The Barzilai University Medical Center

    Ashkelon, 7830604

  • Hadassah Medical Center- Ein Kerem

    Jerusalem, 9112001

  • Meir Medical Center

    Kfar Saba, 4428164

  • The Chaim Sheba Medical Center

    Ramat Gan, 5265601

  • Ziv Medical Center

    Safed, 1311001

Other trials in Israel

Greece4 sites

  • Evangelismos General Hospital of Athens

    Athens, 10676

  • "Hippokration" General Hospital of Athens

    Athens, 11527

  • Onassis Hospital

    Athens, 17674

  • University General Hospital "ATTIKON"

    Attiki, 12462

Other trials in Greece

Portugal4 sites

  • Centro Hospitalar Do Baixo Vouga, EPE Cardiology Department

    Aveiro, 3814-501

  • Unidade Local De Saude De Sao Jose E.P.E

    Lisbon, 1150-199

  • Hospital Cuf Descobertas S.A.

    Lisbon, 1998-018

  • Hospital da Luz

    Lisbon

Other trials in Portugal

Australia3 sites

  • Flinders Medical Centre, Cardiology Research

    Bedford Park, 5042

  • The Prince Charles Hospital

    Chermside, 4032

  • Fiona Stanley Hospital, AHF Unit

    Murdoch, 6150

Other trials in Australia

Netherlands3 sites

  • Amsterdam UMC

    Amsterdam, 1105

  • Maastricht University Medical Center (MUMC)

    Maastricht, 6229 HX

  • Erasmus Medical Center Department of Cardiology

    Rotterdam, 3015

Other trials in Netherlands

Hungary2 sites

  • Semmelweis Egyetem Varosmajori Sziv es Ergyogyaszati Klinika

    Budapest, 1122

  • Szegedi Tudományegyetem, Belgyógyászati Klinika, Kardiológiai Központ

    Szeged, 6725

Other trials in Hungary

Poland2 sites

  • Kardio Brynow S.C.

    Katowice, 40-555

  • Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego - Panstwowy Instytut Badawczy Centrum Zaburzen Rytmu Serca

    Warsaw, 04-628

Other trials in Poland

Argentina1 site

  • Instituto Cardiovascular de Buenos Aires(ICBA)

    Buenos Aires, C1428

Other trials in Argentina

Czechia1 site

  • Il. Interni klinika kardiologie a angiologie

    Prague, 128 08

Other trials in Czechia

Iceland1 site

  • Landspitali University Hospital

    Reykjavik, 101

Other trials in Iceland

About this study

Official title: A Follow-Up, Open-Label, Research Evaluation of Sustained Treatment With Aficamten (CK-3773274) in Hypertrophic Cardiomyopathy (HCM)

The purpose of this study is to collect long-term safety and tolerability data for aficamten.

Being tested
Aficamten (5 - 20 mg)
Keywords
Obstructive Hypertrophic Cardiomyopathy, oHCM, CK-3773274, CK-274, Non obstructive hypertrophic cardiomyopathy, nHCM, HCM, Hypertrophic cardiomyopathy, Aficamten, REDWOOD-OLE, CY 6022, FOREST-HCM, Forest
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 - 20 mg)Drug

    Aficamten tablets, 5-20 mg daily, administered orally. Participants may continue on Aficamten until end of treatment criteria defined in the protocol is met.

Study groups in NCT04848506
Study groupRoleWhat they receive
AficamtenExperimentalParticipants in this arm take aficamten starting at the lowest prespecified dose and titrating up to their individual maximum tolerated dose. During titration phase, clinic visits will occur approximately every 2-6 weeks. In the maintenance phase clinic visits will occur every 24 weeks, with safety check-in occurring every 12 weeks between visits by phone or, if desirable, in the clinic. After Week 48, the visits can be yearly if participants meet criteria.
Receives: Drug: Aficamten (5 - 20 mg)

What is being measured

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

  1. Incidence of adverse events observed during dosing of aficamten in patients with HCM

    Patient incidence of reported Adverse Events (AEs)

    Measured: Baseline to End of study, up to 5 years

  2. Incidence of serious adverse events observed during dosing of aficamten in patients with HCM

    Patient incidence of reported Serious Adverse Events (SAEs)

    Measured: Baseline to End of study, up to 5 years

  3. Incidence of left ventricular ejection fraction (LVEF) < 50% observed during dosing of aficamten in patients with HCM

    Patient incidence of reported LVEF <50%

    Measured: Baseline to End of study, up to 5 years

Secondary outcomes (12)

  1. Long-term effects of aficamten on left ventricular outflow tract gradient (LVOT G) in patients with obstructive Hypertrophic Cardiomyopathy (oHCM)

    Peak LVOT-G at rest; applicable only to patients with oHCM

    Measured: Baseline through the end of participation at 12-24 week intervals

  2. Long-term effects of aficamten on resting LVOT-G

    Proportion of patients with resting LVOT-G < 30 mmHg; applicable only to patients with oHCM

    Measured: Baseline through the end of participation at 12-24 week intervals

  3. Long-term effects of aficamten on post Valsalva LVOT-G

    Proportion of patients with post-Valsalva LVOT-G < 50 mmHg; applicable only to patients with oHCM

    Measured: Baseline through the end of participation at 12-24 week intervals

  4. Long-term effects of aficamten on post Valsalva LVOT-G

    Proportion of patients with post-Valsalva LVOT-G < 30 mmHg; applicable only to patients with oHCM

    Measured: Baseline through the end of participation at 12-24 week intervals

  5. Long-term effects of aficamten on left ventricular ejection fraction (LVEF) and post-Valsalva LVOT-G

    Proportion of patients with LVEF ≥ 50%, resting LVOT-G < 30 mmHg, and post-Valsalva LVOT-G < 50 mmHg; applicable only to patients with oHCM

    Measured: Baseline through the end of participation at 12-24 week intervals

  6. Long-term effects of aficamten on time to first resting LVOT-G < 30 mmHg through last follow-up

    Applicable only to patients with oHCM

    Measured: Time to the following event through last follow-up, up to 5 years

  7. Long-term effects of aficamten on time to first post-Valsalva LVOT-G < 50 mmHg through last follow-up

    Applicable only to patients with oHCM

    Measured: Time to the following event through last follow-up, up to 5 years

  8. Long-term effects of aficamten on time to first post-Valsalva LVOT-G < 30 mmHg through last follow-up

    Applicable only to patients with oHCM

    Measured: Time to the following event through last follow-up, up to 5 years

  9. Long-term effects of aficamten on time to first LVEF ≥ 50%, resting LVOT-G < 30 mmHg, and post-Valsalva LVOT-G < 50 mmHg through last follow-up

    Applicable only to patients with oHCM

    Measured: Time to the following event through last follow-up, up to 5 years

  10. Change from baseline values in N-terminal pro-B-type natriuretic peptide (NT-proBNP)

    Measured: Baseline to end of collection, up to 5 years

  11. Change from baseline values in high sensitivity cardiac troponin I (hs-cTnI)

    Measured: Baseline to end of collection, up to 5 years

  12. Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) score

    Measured: Baseline to end of collection, up to 5 years

Timeline

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

  1. First posted19 Apr 2021
  2. Study start6 May 2021
  3. Primary completionMar 2029 (estimated)
  4. Study completionMar 2029 (estimated)

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 2/3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
900 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04848506
Sponsor's protocol ID
CY 6022
Other IDs
2020-003571-17, 2023-508963-58-00, 2023
Acronym
FOREST-HCM
Status last verified
Oct 2026
First submitted
9 Apr 2021
Last updated
13 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Chen ML, Hodovan J, Masri A, Liu V, Kim MO, Melloni C, Jacoby DL, Heitner SB, Abraham TP. Aficamten is associated with improvements in cardiac mechanics in obstructive hypertrophic cardiomyopathy: Results from FOREST-HCM. Prog Cardiovasc Dis. 2026 Aug 25:S0033-0620(26)00098-8. doi: 10.1016/j.pcad.2026.08.013. Online ahead of print.

    PubMed 42641848

  • Saberi S, Abraham TP, Choudhury L, Barriales-Villa R, Elliott PM, Nassif ME, Oreziak A, Owens AT, Tower-Rader A, Rader F, Garcia-Pavia P, Olivotto I, Coats CJ, Fifer MA, Sherrid MV, Solomon SD, Watkins H, Heitner SB, Jacoby DL, Kupfer S, Malik FI, Melloni C, Meng L, Wei J, Maron MS, Masri A; FOREST-HCM Steering Committee and Investigators. Aficamten Treatment for Symptomatic Obstructive Hypertrophic Cardiomyopathy: 48-Week Results From FOREST-HCM. JACC Heart Fail. 2025 Aug;13(8):102496. doi: 10.1016/j.jchf.2025.03.040. Epub 2025 Jun 19.

    PubMed 40540987

  • Masri A, Maron MS, Abraham TP, Nassif ME, Barriales-Villa R, Bilen O, Coats CJ, Elliott P, Garcia-Pavia P, Massera D, Olivotto I, Oreziak A, Owens AT, Saberi S, Solomon SD, Tower-Rader A, Heitner SB, Jacoby DL, Melloni C, Wei J, Sherrid MV; REDWOOD-HCM, SEQUOIA-HCM, and FOREST-HCM Investigators. Concomitant Aficamten and Disopyramide in Symptomatic Obstructive Hypertrophic Cardiomyopathy. JACC Heart Fail. 2026 Feb;14(2):102441. doi: 10.1016/j.jchf.2025.03.008. Epub 2025 Apr 2.

    PubMed 40285763

  • Masri A, Choudhury L, Barriales-Villa R, Elliott P, Maron MS, Nassif ME, Oreziak A, Owens AT, Saberi S, Tower-Rader A, Rader F, Garcia-Pavia P, Olivotto I, Nagueh SF, Wang A, Heitner SB, Jacoby DL, Kupfer S, Malik FI, Melloni C, Meng L, Wei J, Sherrid MV, Abraham TP; FOREST-HCM Investigators. Standard-of-Care Medication Withdrawal in Patients With Obstructive Hypertrophic Cardiomyopathy Receiving Aficamten in FOREST-HCM. J Am Coll Cardiol. 2024 Nov 5;84(19):1839-1849. doi: 10.1016/j.jacc.2024.09.002.

    PubMed 39477631

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Aficamten (5 - 20 mg) in people with Symptomatic Hypertrophic Cardiomyopathy (HCM), run by Cytokinetics at 141 sites in 19 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 2 inclusion and 7 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: enrolling by invitation, as of the sponsor's last update on 13 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 141 sites in United States, Spain, France, United Kingdom, Italy and 14 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

This study is still under way. Results are not available in the source record yet.

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?

Incidence of adverse events observed during dosing of aficamten in patients with HCM (time frame: Baseline to End of study, up to 5 years). The study lists 3 primary outcomes in all. 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 NCT04848506, which also lists 4 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 13 Oct 2026.

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