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

Recruitment Status
ENROLLING BY INVITATION
(See Contacts and Locations)Verified December 2024 by Cytokinetics
Sponsor
Cytokinetics
Information Provided by (Responsible Party)
Cytokinetics
Clinicaltrials.gov Identifier
NCT04848506
Other Study ID Numbers:
CY 6022
First Submitted
April 8, 2021
First Posted
April 18, 2021
Last Update Posted
January 21, 2026
Last Verified
December 2024

ClinicalTrials.gov processed this data on January 2026. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study Description

Condition or DiseaseIntervention/Treatment
Symptomatic Hypertrophic Cardiomyopathy (HCM)
Drug: Aficamten (5 - 20 mg)

Study Design

Study TypeInterventional
Actual Enrollment900 participants
Design AllocationN/A
Interventional ModelSingle Group Assignment
MaskingNone (Open Label)
Primary PurposeTreatment
Official TitleA Follow-Up, Open-Label, Research Evaluation of Sustained Treatment With Aficamten (CK-3773274) in Hypertrophic Cardiomyopathy (HCM)
Study Start DateMay 5, 2021
Actual Primary Completion Date1yr 10mos from now
Actual Study Completion Date1yr 10mos from now

Groups and Cohorts

Group/CohortIntervention/Treatment
Aficamten up to 20 mg
Patients in this arm take daily dose of aficamten. Each patient will start at the lowest prespecified dose and titrate up to their maximum tolerated dose.
Drug: Aficamten (5 - 20 mg)
Aficamten tablets administered orally. 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.

Outcome Measures

Primary Outcome Measures
  1. Incidence of adverse events observed during dosing of aficamten in patients with HCM
    Patient incidence of reported Adverse Events (AEs)
Secondary Outcome Measures
  1. Incidence of serious adverse events observed during dosing of aficamten in patients with HCM
    Patient incidence of reported Serious Adverse Events (SAEs)
  2. Incidence of left ventricular ejection fraction (LVEF) < 50% observed during dosing of aficamten in patients with HCM
    Patient incidence of reported LVEF \&lt;50%
  3. Long-term effects of aficamten on left ventricular outflow tract gradient (LVOT G) in patients with oHCM
    Peak LVOT-G at rest; applicable only to patients with oHCM
  4. Long-term effects of aficamten on resting LVOT-G
    Proportion of patients with resting LVOT-G \&lt; 30 mmHg; applicable only to patients with oHCM
  5. Long-term effects of aficamten on post Valsalva LVOT-G
    Proportion of patients with post-Valsalva LVOT-G \&lt; 50 mmHg; applicable only to patients with oHCM
  6. Long-term effects of aficamten on post Valsalva LVOT-G
    Proportion of patients with post-Valsalva LVOT-G \&lt; 30 mmHg; applicable only to patients with oHCM
  7. Long-term effects of aficamten on left ventricular ejection fraction (LVEF) and post-Valsalva LVOT-G
    Proportion of patients with LVEF &ge; 50%, resting LVOT-G \&lt; 30 mmHg, and post-Valsalva LVOT-G \&lt; 50 mmHg; applicable only to patients with oHCM
  8. Long-term effects of aficamten on time to first resting LVOT-G < 30 mmHg through last follow-up
    Applicable only to patients with oHCM
  9. 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
  10. 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
  11. 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

Eligibility Criteria

Ages Eligible for Study(Adult, Older Adult)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
Inclusion Criteria
Completion of a Cytokinetics trial investigating aficamten
LVEF ≥ 55% at the Screening Visit
Exclusion Criteria
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.

Contacts and Locations

Sponsors and CollaboratorsCytokinetics
Locations
University of Alabama at Birmingham | Birmingham Alabama, United States, 35233Alaska Heart and Vascular Institute | Anchorage Alaska, United States, 99508Mayo Clinic Building - Phoenix | Phoenix Arizona, United States, 85054UC San Diego Health - Sulpizio Cardiovascular Center | La Jolla California, United States, 92037Cedar-Sinai Medical Center | Los Angeles California, United States, 90048Cedars-Sinai Medical Center (Smidt Heart Institute) | Los Angeles California, United States, 90048UCSF Medical Center | San Francisco California, United States, 94143Yale New Haven Hospital | New Haven Connecticut, United States, 06519MedStar Washington Hospital Center | Washington D.C. District of Columbia, United States, 20010Holy Cross Hospital / Cardiology Associates | Fort Lauderdale Florida, United States, 33308Emory Clinic | Atlanta Georgia, United States, 30322Piedmont Fayette Hospital | Fayetteville Georgia, United States, 30214Northwestern University | Evanston Illinois, United States, 60208The University of Kansas Medical Center | Kansas City Kansas, United States, 66160Massachusetts General Hospital | Boston Massachusetts, United States, 02114Brigham and Women's Hospital | Boston Massachusetts, United States, 02115Lahey Hospital & Medical Center | Burlington Massachusetts, United States, 01805Michigan Medicine - University of Michigan | Ann Arbor Michigan, United States, 48109University of Michigan Health System (UMHS) - University Hospital (University of Michigan Medical Center) | Ann Arbor Michigan, United States, 48109Henry Ford Hospital | Detroit Michigan, United States, 48202Saint Luke's Hospital of Kansas City | Kansas City Missouri, United States, 64111Morristown Medical Center | Morristown New Jersey, United States, 07960Northwell Health North Shore University Hospital | Manhasset New York, United States, 11030NYU Langone Health | New York New York, United States, 10016Weill Cornell Medicine | New York New York, United States, 10021Mount Sinai Hospital | New York New York, United States, 10029Columbia University Medical Center/New York Presbyterian Hospital | New York New York, United States, 10032Westchester Medical Center | Valhalla New York, United States, 10595Sanger Heart & Vascular Institute - HCM Clinic | Charlotte North Carolina, United States, 28204Duke Cardiology at Southpoint | Durham North Carolina, United States, 27710The Linder Research Center at The Christ Hospital | Cincinnati Ohio, United States, 45219University Hospitals Cleveland Medical Center | Cleveland Ohio, United States, 44106Cleveland Clinic | Cleveland Ohio, United States, 44195Ascension St. John Clinical Research Institute | Tulsa Oklahoma, United States, 76104Providence St. Vincent Medical Center | Portland Oregon, United States, 97225Oregon Health & Science University | Portland Oregon, United States, 97239Hospital of the University of Pennsylvania (University of Pennsylvania School of Medicine) | Philadelphia Pennsylvania, United States, 19104UPMC Presbyterian | Pittsburgh Pennsylvania, United States, 15213Medical University of South Carolina | Charleston South Carolina, United States, 29425Greenville Memorial Hospital | Greenville South Carolina, United States, 29605Ascension Saint Thomas Heart West | Nashville Tennessee, United States, 37205Vanderbilt University Medical Center | Nashville Tennessee, United States, 37232The University of Texas Southwestern Medical Center at Dallas | Dallas Texas, United States, 75390CHI St. Luke's Health Baylor College of Medicine Medical Center | Houston Texas, United States, 77030Houston Methodist Hospital | Houston Texas, United States, 77030Intermountain Medical Center | Murray Utah, United States, 84107University of Utah Health | Salt Lake City Utah, United States, 84132University of Virginia Health System University Hospital | Charlottesville Virginia, United States, 22908Inova Health Care Services | Falls Church Virginia, United States, 22042Virginia Mason Medical Center | Seattle Washington, United States, 98101WVUMedicine | Morgantown West Virginia, United States, 26506Aurora St. Luke's Medical Center | Milwaukee Wisconsin, United States, 53215Instituto Cardiovascular de Buenos Aires(ICBA) | Buenos Aires , Argentina, C1428Flinders Medical Centre, Cardiology Research | Bedford Park , Australia, 5042The Prince Charles Hospital | Chermside , Australia, 4032Fiona Stanley Hospital, AHF Unit | Murdoch , Australia, 6150Instituto Dante Pazzanese de Cardiologia | São Paulo , Brazil, 04012-909Institut universitaire de cardiologie et de pneumologie de Quebec-Universite Laval | Québec Quebec, Canada, G1V 4G5Royal Alexandra Hospital | Edmonton , Canada, T5H 3V9QEII Health Science Centre (Halifax Infirmary site) | Halifax , Canada, B3H 3A7Il. Interni klinika kardiologie a angiologie | Prague , Czechia, 128 08Department of Cardiology Aalborg University Hospital | Aalborg , Denmark, 9000Department of Cardiology Aarhus University Hospital | Aarhus , Denmark, 8200The Heart Center, Department of Cardiology | Copenhagen , Denmark, 2100Copenhagen University Hospital | Copenhagen , Denmark, 2400Odense University Hospital | Odense , Denmark, 5000Hopital de la Timone Service de cardiologie | Marseille , France, 130005Clinique Du Millenaire | Montpellier , France, 340000CHU Montpellier - Hopital Arnaud de Villeneuve | Montpellier , France, 34295CHU de Nantes Institut Du Thorax et du systeme nerveux Clinique Cardiologique et des Maladies Vasculaires | Nantes , France, 44093Hopital Lariboisiere | Paris , France, 75010Hopital Europeen Georges Pompidou | Paris , France, 75015Hopital cardiologique de Haut-Leveque | Pessac , France, 33604Centre hospitalier universitaire (CHU) de Rennes-Hopital Pontchaillou | Rennes , France, 35033Centre Hospitalier universitaire de Toulouse - Rangueil Hospital | Toulouse , France, 31059Charite-Universitaetsmedizin Berlin | Berlin , Germany, 13353Universitaetsklinikum Essen | Essen , Germany, 45147Universitaetsmedizin Goettingen Klinik fur Kardiologie und Pneumologie | Goettigen , Germany, 37075Universitaesklinkum Heidelberg | Heidelberg , Germany, 69120Universitaetsklinikum Jena | Jena , Germany, 07747Universitaetskinikum Wuerzburg Deutsches Zentrum fur Herzinsuffzienz Wurzburg (DZHI) Comprehensive Heart Failure Center (CHFC) | Würzburg , Germany, 97078Evangelismos General Hospital of Athens | Athens , Greece, 10676"Hippokration" General Hospital of Athens | Athens , Greece, 11527Onassis Hospital | Athens , Greece, 17674University General Hospital "ATTIKON" | Attiki , Greece, 12462Semmelweis Egyetem Varosmajori Sziv es Ergyogyaszati Klinika | Budapest , Hungary, 1122Szegedi Tudományegyetem, Belgyógyászati Klinika, Kardiológiai Központ | Szeged , Hungary, 6725Landspitali University Hospital | Reykjavik , Iceland, 101The Barzilai University Medical Center | Ashkelon , Israel, 7830604Hadassah Medical Center- Ein Kerem | Jerusalem , Israel, 9112001Meir Medical Center | Kfar Saba , Israel, 4428164The Chaim Sheba Medical Center | Ramat Gan , Israel, 5265601Ziv Medical Center | Safed , Israel, 1311001Unit Cardiomiopatie, Dipartimento Cardio ToracoVascolare | Florence , Italy, 50134IRCCS Policlinico San Donato | Milan , Italy, 20097ASST Grande Ospedale Metropolitano Niguarda | Milan , Italy, 20162Azienda Ospedaliera Universitaria Federico II di Napoli | Naples , Italy, 80131Fondazione toscana Gabriele Monesterio per la ricarca medica Dipartimento Cardiotoracico UOC Cardiologia e Medicina Cardiovasculare Ospedale San Cataldo | Pisa , Italy, 56124Dipartimento di Medicina Clinica e Molecolare Universita Sapienza di Roma Unita di Terapia Intensiva Cardiologica Azienda Ospedaliero Universitaria Sant'Andrea | Roma , Italy, 00189Azienda Sanitaria Universitaria Giuliano Isontina | Trieste , Italy, 34149Amsterdam UMC | Amsterdam , Netherlands, 1105Maastricht University Medical Center (MUMC) | Maastricht , Netherlands, 6229 HXErasmus Medical Center Department of Cardiology | Rotterdam , Netherlands, 3015Kardio Brynow S.C. | Katowice , Poland, 40-555Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego - Panstwowy Instytut Badawczy Centrum Zaburzen Rytmu Serca | Warsaw , Poland, 04-628Centro Hospitalar Do Baixo Vouga, EPE Cardiology Department | Aveiro , Portugal, 3814-501Unidade Local De Saude De Sao Jose E.P.E | Lisbon , Portugal, 1150-199Hospital Cuf Descobertas S.A. | Lisbon , Portugal, 1998-018Hospital da Luz | Lisbon , Portugal, Complejo Hospitalario Universitario A Coruna | A Coruña , Spain, 15006Hospital Clinic Barcelona | Barcelona , Spain, 08036Hospital General Universitario Gregorio Marañón (HGUGM) | Madrid , Spain, 28007Hospital Clinico Universitario San Carlos | Madrid , Spain, 28040Hospital Universitario Puerta de Hierro Majadahonda | Madrid , Spain, 28220Hospital Clinico Universitario Virgen de la Arrixaca | Madrid , Spain, 30120Hospital Universitario Virgen de la Victoria | Málaga , Spain, 29010Hospital Universitario Son Llatzer | Palma , Spain, 07198Complexo Hospitalario Universitario De Vigo | Pontevedra , Spain, 36312Hospital Universitario de Salamanca | Salamanca , Spain, 37007Hospital Universitario Virgen Macarena-merge | Seville , Spain, 41009Hospital Universitario Virgen del Rocío | Seville , Spain, 41013Queen Elizabeth Hospital Birmingham University Hospitals Birmingham NHS Foundation Trust | Birmingham , United Kingdom, B15 2GWNinewells Hospital and Medical School | Dundee , United Kingdom, DD2 1SGGlasgow Clinical Research Facility Neuroscience Institute | Glasgow , United Kingdom, G51 4TFLiverpool Heart and Chest Hospital | Liverpool , United Kingdom, L14 3PESt Bartholomew's Hospital | London , United Kingdom, EC1A 7BESt George's University Hospitals NHS Foundation Trust | London , United Kingdom, SW17 0QTRoyal Brompton Hospital | London , United Kingdom, SW3 6NPOxford Centre for Clinical Magnetic Resonance Research (OCMR) Division of Cardiovascular Medicine Radcliffe Department of Medicine University of Oxford | Oxford , United Kingdom, OX3 9DU
Investigators
Study Director: Cytokinetics, MD, Cytokinetics