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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-003571-17 | EudraCT Number | ||
| 2023-508963-58-00 | Other Identifier | EU CTR Number |
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The purpose of this study is to collect long-term safety and tolerability data for aficamten.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Aficamten up to 20 mg | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Aficamten (5 - 20 mg) | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of adverse events observed during dosing of aficamten in patients with HCM | Patient incidence of reported Adverse Events (AEs) | Baseline to End of study, up to 5 years |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of serious adverse events observed during dosing of aficamten in patients with HCM | Patient incidence of reported Serious Adverse Events (SAEs) | Baseline to End of study, up to 5 years |
| Incidence of left ventricular ejection fraction (LVEF) < 50% observed during dosing of aficamten in patients with HCM |
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Inclusion Criteria:
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:
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.
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| Name | Affiliation | Role |
|---|---|---|
| Cytokinetics, MD | Cytokinetics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | United States | ||
| Alaska Heart and Vascular Institute |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40540987 | Derived | 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. | |
| 40285763 |
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|
Patient incidence of reported LVEF <50% |
| Baseline to End of study, up to 5 years |
| 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 | Baseline through the end of participation at 12-24 week intervals |
| Long-term effects of aficamten on resting LVOT-G | Proportion of patients with resting LVOT-G < 30 mmHg; applicable only to patients with oHCM | Baseline through the end of participation at 12-24 week intervals |
| 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 | Baseline through the end of participation at 12-24 week intervals |
| 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 | Baseline through the end of participation at 12-24 week intervals |
| 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 | Baseline through the end of participation at 12-24 week intervals |
| Long-term effects of aficamten on time to first resting LVOT-G < 30 mmHg through last follow-up | Applicable only to patients with oHCM | Time to the following event through last follow-up, up to 5 years |
| 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 | Time to the following event through last follow-up, up to 5 years |
| 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 | Time to the following event through last follow-up, up to 5 years |
| 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 | Time to the following event through last follow-up, up to 5 years |
| Anchorage |
| Alaska |
| 99508 |
| United States |
| Mayo Clinic Building - Phoenix | Phoenix | Arizona | 85054 | United States |
| UC San Diego Health - Sulpizio Cardiovascular Center | La Jolla | California | 92037 | United States |
| Cedar-Sinai Medical Center | Los Angeles | California | 90048 | United States |
| Cedars-Sinai Medical Center (Smidt Heart Institute) | Los Angeles | California | 90048 | United States |
| UCSF Medical Center | San Francisco | California | 94143 | United States |
| Yale New Haven Hospital | New Haven | Connecticut | 06519 | United States |
| MedStar Washington Hospital Center | Washington D.C. | District of Columbia | 20010 | United States |
| Holy Cross Hospital / Cardiology Associates | Fort Lauderdale | Florida | 33308 | United States |
| Emory Clinic | Atlanta | Georgia | 30322 | United States |
| Piedmont Fayette Hospital | Fayetteville | Georgia | 30214 | United States |
| Northwestern University | Evanston | Illinois | 60208 | United States |
| The University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Brigham and Women's Hospital | Boston | Massachusetts | 02115 | United States |
| Lahey Hospital & Medical Center | Burlington | Massachusetts | 01805 | United States |
| Michigan Medicine - University of Michigan | Ann Arbor | Michigan | 48109 | United States |
| University of Michigan Health System (UMHS) - University Hospital (University of Michigan Medical Center) | Ann Arbor | Michigan | 48109 | United States |
| Henry Ford Hospital | Detroit | Michigan | 48202 | United States |
| Saint Luke's Hospital of Kansas City | Kansas City | Missouri | 64111 | United States |
| Morristown Medical Center | Morristown | New Jersey | 07960 | United States |
| Northwell Health North Shore University Hospital | Manhasset | New York | 11030 | United States |
| NYU Langone Health | New York | New York | 10016 | United States |
| Weill Cornell Medicine | New York | New York | 10021 | United States |
| Mount Sinai Hospital | New York | New York | 10029 | United States |
| Columbia University Medical Center/New York Presbyterian Hospital | New York | New York | 10032 | United States |
| Westchester Medical Center | Valhalla | New York | 10595 | United States |
| Sanger Heart & Vascular Institute - HCM Clinic | Charlotte | North Carolina | 28204 | United States |
| Duke Cardiology at Southpoint | Durham | North Carolina | 27710 | United States |
| The Linder Research Center at The Christ Hospital | Cincinnati | Ohio | 45219 | United States |
| University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| Ascension St. John Clinical Research Institute | Tulsa | Oklahoma | 76104 | United States |
| Providence St. Vincent Medical Center | Portland | Oregon | 97225 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Hospital of the University of Pennsylvania (University of Pennsylvania School of Medicine) | Philadelphia | Pennsylvania | 19104 | United States |
| UPMC Presbyterian | Pittsburgh | Pennsylvania | 15213 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Greenville Memorial Hospital | Greenville | South Carolina | 29605 | United States |
| Ascension Saint Thomas Heart West | Nashville | Tennessee | 37205 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| The University of Texas Southwestern Medical Center at Dallas | Dallas | Texas | 75390 | United States |
| CHI St. Luke's Health Baylor College of Medicine Medical Center | Houston | Texas | 77030 | United States |
| Houston Methodist Hospital | Houston | Texas | 77030 | United States |
| Intermountain Medical Center | Murray | Utah | 84107 | United States |
| University of Utah Health | Salt Lake City | Utah | 84132 | United States |
| University of Virginia Health System University Hospital | Charlottesville | Virginia | 22908 | United States |
| Inova Health Care Services | Falls Church | Virginia | 22042 | United States |
| Virginia Mason Medical Center | Seattle | Washington | 98101 | United States |
| WVUMedicine | Morgantown | West Virginia | 26506 | United States |
| Aurora St. Luke's Medical Center | Milwaukee | Wisconsin | 53215 | United States |
| Instituto Cardiovascular de Buenos Aires(ICBA) | Buenos Aires | C1428 | Argentina |
| Flinders Medical Centre, Cardiology Research | Bedford Park | 5042 | Australia |
| The Prince Charles Hospital | Chermside | 4032 | Australia |
| Fiona Stanley Hospital, AHF Unit | Murdoch | 6150 | Australia |
| Instituto Dante Pazzanese de Cardiologia | São Paulo | 04012-909 | Brazil |
| Institut universitaire de cardiologie et de pneumologie de Quebec-Universite Laval | Québec | Quebec | G1V 4G5 | Canada |
| Royal Alexandra Hospital | Edmonton | T5H 3V9 | Canada |
| QEII Health Science Centre (Halifax Infirmary site) | Halifax | B3H 3A7 | Canada |
| Il. Interni klinika kardiologie a angiologie | Prague | 128 08 | Czechia |
| Department of Cardiology Aalborg University Hospital | Aalborg | 9000 | Denmark |
| Department of Cardiology Aarhus University Hospital | Aarhus | 8200 | Denmark |
| The Heart Center, Department of Cardiology | Copenhagen | 2100 | Denmark |
| Copenhagen University Hospital | Copenhagen | 2400 | Denmark |
| Odense University Hospital | Odense | 5000 | Denmark |
| Hopital de la Timone Service de cardiologie | Marseille | 130005 | France |
| Clinique Du Millenaire | Montpellier | 340000 | France |
| CHU Montpellier - Hopital Arnaud de Villeneuve | Montpellier | 34295 | France |
| CHU de Nantes Institut Du Thorax et du systeme nerveux Clinique Cardiologique et des Maladies Vasculaires | Nantes | 44093 | France |
| Hopital Lariboisiere | Paris | 75010 | France |
| Hopital Europeen Georges Pompidou | Paris | 75015 | France |
| Hopital cardiologique de Haut-Leveque | Pessac | 33604 | France |
| Centre hospitalier universitaire (CHU) de Rennes-Hopital Pontchaillou | Rennes | 35033 | France |
| Centre Hospitalier universitaire de Toulouse - Rangueil Hospital | Toulouse | 31059 | France |
| Charite-Universitaetsmedizin Berlin | Berlin | 13353 | Germany |
| Universitaetsklinikum Essen | Essen | 45147 | Germany |
| Universitaetsmedizin Goettingen Klinik fur Kardiologie und Pneumologie | Goettigen | 37075 | Germany |
| Universitaesklinkum Heidelberg | Heidelberg | 69120 | Germany |
| Universitaetsklinikum Jena | Jena | 07747 | Germany |
| Universitaetskinikum Wuerzburg Deutsches Zentrum fur Herzinsuffzienz Wurzburg (DZHI) Comprehensive Heart Failure Center (CHFC) | Würzburg | 97078 | Germany |
| Evangelismos General Hospital of Athens | Athens | 10676 | Greece |
| "Hippokration" General Hospital of Athens | Athens | 11527 | Greece |
| Onassis Hospital | Athens | 17674 | Greece |
| University General Hospital "ATTIKON" | Attiki | 12462 | Greece |
| Semmelweis Egyetem Varosmajori Sziv es Ergyogyaszati Klinika | Budapest | 1122 | Hungary |
| Szegedi Tudományegyetem, Belgyógyászati Klinika, Kardiológiai Központ | Szeged | 6725 | Hungary |
| Landspitali University Hospital | Reykjavik | 101 | Iceland |
| The Barzilai University Medical Center | Ashkelon | 7830604 | Israel |
| Hadassah Medical Center- Ein Kerem | Jerusalem | 9112001 | Israel |
| Meir Medical Center | Kfar Saba | 4428164 | Israel |
| The Chaim Sheba Medical Center | Ramat Gan | 5265601 | Israel |
| Ziv Medical Center | Safed | 1311001 | Israel |
| Unit Cardiomiopatie, Dipartimento Cardio ToracoVascolare | Florence | 50134 | Italy |
| IRCCS Policlinico San Donato | Milan | 20097 | Italy |
| ASST Grande Ospedale Metropolitano Niguarda | Milan | 20162 | Italy |
| Azienda Ospedaliera Universitaria Federico II di Napoli | Naples | 80131 | Italy |
| Fondazione toscana Gabriele Monesterio per la ricarca medica Dipartimento Cardiotoracico UOC Cardiologia e Medicina Cardiovasculare Ospedale San Cataldo | Pisa | 56124 | Italy |
| Dipartimento di Medicina Clinica e Molecolare Universita Sapienza di Roma Unita di Terapia Intensiva Cardiologica Azienda Ospedaliero Universitaria Sant'Andrea | Roma | 00189 | Italy |
| Azienda Sanitaria Universitaria Giuliano Isontina | Trieste | 34149 | Italy |
| Amsterdam UMC | Amsterdam | 1105 | Netherlands |
| Maastricht University Medical Center (MUMC) | Maastricht | 6229 HX | Netherlands |
| Erasmus Medical Center Department of Cardiology | Rotterdam | 3015 | Netherlands |
| Kardio Brynow S.C. | Katowice | 40-555 | Poland |
| Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego - Panstwowy Instytut Badawczy Centrum Zaburzen Rytmu Serca | Warsaw | 04-628 | Poland |
| Centro Hospitalar Do Baixo Vouga, EPE Cardiology Department | Aveiro | 3814-501 | Portugal |
| Unidade Local De Saude De Sao Jose E.P.E | Lisbon | 1150-199 | Portugal |
| Hospital Cuf Descobertas S.A. | Lisbon | 1998-018 | Portugal |
| Hospital da Luz | Lisbon | Portugal |
| Complejo Hospitalario Universitario A Coruna | A Coruña | 15006 | Spain |
| Hospital Clinic Barcelona | Barcelona | 08036 | Spain |
| Hospital General Universitario Gregorio Marañón (HGUGM) | Madrid | 28007 | Spain |
| Hospital Clinico Universitario San Carlos | Madrid | 28040 | Spain |
| Hospital Universitario Puerta de Hierro Majadahonda | Madrid | 28220 | Spain |
| Hospital Clinico Universitario Virgen de la Arrixaca | Madrid | 30120 | Spain |
| Hospital Universitario Virgen de la Victoria | Málaga | 29010 | Spain |
| Hospital Universitario Son Llatzer | Palma | 07198 | Spain |
| Complexo Hospitalario Universitario De Vigo | Pontevedra | 36312 | Spain |
| Hospital Universitario de Salamanca | Salamanca | 37007 | Spain |
| Hospital Universitario Virgen Macarena-merge | Seville | 41009 | Spain |
| Hospital Universitario Virgen del Rocío | Seville | 41013 | Spain |
| Queen Elizabeth Hospital Birmingham University Hospitals Birmingham NHS Foundation Trust | Birmingham | B15 2GW | United Kingdom |
| Ninewells Hospital and Medical School | Dundee | DD2 1SG | United Kingdom |
| Glasgow Clinical Research Facility Neuroscience Institute | Glasgow | G51 4TF | United Kingdom |
| Liverpool Heart and Chest Hospital | Liverpool | L14 3PE | United Kingdom |
| St Bartholomew's Hospital | London | EC1A 7BE | United Kingdom |
| St George's University Hospitals NHS Foundation Trust | London | SW17 0QT | United Kingdom |
| Royal Brompton Hospital | London | SW3 6NP | United Kingdom |
| Oxford Centre for Clinical Magnetic Resonance Research (OCMR) Division of Cardiovascular Medicine Radcliffe Department of Medicine University of Oxford | Oxford | OX3 9DU | United Kingdom |
| Derived |
| 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. |
| 39477631 | Derived | 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. |
| ID | Term |
|---|---|
| D002312 | Cardiomyopathy, Hypertrophic |
| ID | Term |
|---|---|
| D009202 | Cardiomyopathies |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D001020 | Aortic Stenosis, Subvalvular |
| D001024 | Aortic Valve Stenosis |
| D000082862 | Aortic Valve Disease |
| D006349 | Heart Valve Diseases |
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