Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Aficamten in Adults With HCM
- Conditions
- Symptomatic Hypertrophic Cardiomyopathy (HCM)
- Interventions
- Registration Number
- NCT04848506
- Lead Sponsor
- Cytokinetics
- Brief Summary
The purpose of this study is to collect long-term safety and tolerability data for aficamten.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 900
- Completion of a Cytokinetics trial investigating aficamten
- LVEF ≥ 55% at the Screening Visit
-
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Aficamten up to 20 mg Aficamten (5 - 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.
- Primary Outcome Measures
Name Time Method Incidence of adverse events observed during dosing of aficamten in patients with HCM Baseline to End of study, up to 5 years Patient incidence of reported Adverse Events (AEs)
- Secondary Outcome Measures
Name Time Method Incidence of serious adverse events observed during dosing of aficamten in patients with HCM Baseline to End of study, up to 5 years Patient incidence of reported Serious Adverse Events (SAEs)
Incidence of left ventricular ejection fraction (LVEF) < 50% observed during dosing of aficamten in patients with HCM Baseline to End of study, up to 5 years Patient incidence of reported LVEF \<50%
Long-term effects of aficamten on left ventricular outflow tract gradient (LVOT G) in patients with oHCM Baseline through the end of participation at 12-24 week intervals Peak LVOT-G at rest; applicable only to patients with oHCM
Long-term effects of aficamten on resting LVOT-G Baseline through the end of participation at 12-24 week intervals Proportion of patients with resting LVOT-G \< 30 mmHg; applicable only to patients with oHCM
Long-term effects of aficamten on post Valsalva LVOT-G Baseline through the end of participation at 12-24 week intervals Proportion of patients with post-Valsalva LVOT-G \< 30 mmHg; applicable only to patients with oHCM
Long-term effects of aficamten on left ventricular ejection fraction (LVEF) and post-Valsalva LVOT-G Baseline through the end of participation at 12-24 week intervals Proportion of patients with LVEF ≥ 50%, resting LVOT-G \< 30 mmHg, and post-Valsalva LVOT-G \< 50 mmHg; applicable only to patients with oHCM
Long-term effects of aficamten on time to first resting LVOT-G < 30 mmHg through last follow-up Time to the following event through last follow-up, up to 5 years Applicable only to patients with oHCM
Long-term effects of aficamten on time to first post-Valsalva LVOT-G < 50 mmHg through last follow-up Time to the following event through last follow-up, up to 5 years Applicable only to patients with oHCM
Long-term effects of aficamten on time to first post-Valsalva LVOT-G < 30 mmHg through last follow-up Time to the following event through last follow-up, up to 5 years Applicable only to patients with oHCM
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 Time to the following event through last follow-up, up to 5 years Applicable only to patients with oHCM
Trial Locations
- Locations (90)
Charite-Universitaetsmedizin Berlin
🇩🇪Berlin, Germany
Universitaetsklinikum Essen
🇩🇪Essen, Germany
Universitaetsmedizin Goettingen Klinik fur Kardiologie und Pneumologie
🇩🇪Goettigen, Germany
Universitaetsmedizin Goettingen
🇩🇪Goettingen, Germany
Alaska Heart and Vascular Institute
🇺🇸Anchorage, Alaska, United States
Mayo Clinic Building - Phoenix
🇺🇸Phoenix, Arizona, United States
UC San Diego Health - Sulpizio Cardiovascular Center
🇺🇸La Jolla, California, United States
Cedar-Sinai Medical Center
🇺🇸Los Angeles, California, United States
Cedars-Sinai Medical Center (Smidt Heart Institute)
🇺🇸Los Angeles, California, United States
UCSF Medical Center
🇺🇸San Francisco, California, United States
Yale New Haven Hospital
🇺🇸New Haven, Connecticut, United States
MedStar Washington Hospital Center
🇺🇸Washington, District of Columbia, United States
Holy Cross Hospital / Cardiology Associates
🇺🇸Fort Lauderdale, Florida, United States
Emory Clinic
🇺🇸Atlanta, Georgia, United States
Piedmont Fayette Hospital
🇺🇸Fayetteville, Georgia, United States
Northwestern University
🇺🇸Evanston, Illinois, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
Lahey Hospital & Medical Center
🇺🇸Burlington, Massachusetts, United States
Michigan Medicine - University of Michigan
🇺🇸Ann Arbor, Michigan, United States
University of Michigan Health System (UMHS) - University Hospital (University of Michigan Medical Center)
🇺🇸Ann Arbor, Michigan, United States
Henry Ford Hospital
🇺🇸Detroit, Michigan, United States
Saint Luke's Hospital of Kansas City
🇺🇸Kansas City, Missouri, United States
Morristown Medical Center
🇺🇸Morristown, New Jersey, United States
NYU Langone Health
🇺🇸New York, New York, United States
Mount Sinai Hospital
🇺🇸New York, New York, United States
Columbia University Medical Center/New York Presbyterian Hospital
🇺🇸New York, New York, United States
Universitaesklinkum Heidelberg
🇩🇪Heidelberg, Germany
Westchester Medical Center
🇺🇸Valhalla, New York, United States
Sanger Heart & Vascular Institute - HCM Clinic
🇺🇸Charlotte, North Carolina, United States
Duke Cardiology at Southpoint
🇺🇸Durham, North Carolina, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Ascension St. John Clinical Research Institute
🇺🇸Tulsa, Oklahoma, United States
Providence St. Vincent Medical Center
🇺🇸Portland, Oregon, United States
Universitaetsklinikum Jena
🇩🇪Jena, Germany
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
Hospital of the University of Pennsylvania (University of Pennsylvania School of Medicine)
🇺🇸Philadelphia, Pennsylvania, United States
UPMC Presbyterian
🇺🇸Pittsburg, Pennsylvania, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
Ascension Saint Thomas Heart West
🇺🇸Nashville, Tennessee, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
The University of Texas Southwestern Medical Center at Dallas
🇺🇸Dallas, Texas, United States
CHI St. Luke's Health Baylor College of Medicine Medical Center
🇺🇸Houston, Texas, United States
Houston Methodist Hospital
🇺🇸Houston, Texas, United States
Intermountain Medical Center
🇺🇸Murray, Utah, United States
University of Utah Health
🇺🇸Salt Lake City, Utah, United States
University of Virginia Health System University Hospital
🇺🇸Charlottesville, Virginia, United States
Inova Health Care Services
🇺🇸Falls Church, Virginia, United States
Institut universitaire de cardiologie et de pneumologie de Quebec-Universite Laval
🇨🇦Quebec, Canada
Il. Interni klinika kardiologie a angiologie
🇨🇿Praha, Czechia
Department of Cardiology Aalborg University Hospital
🇩🇰Aalborg, Denmark
Department of Cardiology Aarhus University Hospital
🇩🇰Aarhus, Denmark
The Heart Center, Department of Cardiology
🇩🇰Copenhagen, Denmark
Copenhagen University Hospital
🇩🇰Copenhagen, Denmark
Hopital de la Timone Service de cardiologie
🇫🇷Marseille, France
CHU de Nantes Institut Du Thorax et du systeme nerveux Clinique Cardiologique et des Maladies Vasculaires
🇫🇷Nantes, France
Hopital Lariboisiere
🇫🇷Paris, France
Hopital Europeen Georges Pompidou
🇫🇷Paris, France
Hopital cardiologique de Haut-Leveque
🇫🇷Pessac, France
Centre hospitalier universitaire (CHU) de Rennes-Hopital Pontchaillou
🇫🇷Rennes, France
Universitaetskinikum Wuerzburg Deutsches Zentrum fur Herzinsuffzienz Wurzburg (DZHI) Comprehensive Heart Failure Center (CHFC)
🇩🇪Würzburg, Germany
Semmelweis Egyetem Varosmajori Sziv es Ergyogyaszati Klinika
🇭🇺Budapest, Hungary
The Barzilai University Medical Center
🇮🇱Ashkelon, Israel
Hadassah Medical Center- Ein Kerem
🇮🇱Jerusalem, Israel
The Chaim Sheba Medical Center
🇮🇱Ramat Gan, Israel
Ziv Medical Center
🇮🇱Safed, Israel
Unit Cardiomiopatie, Dipartimento Cardio ToracoVascolare
🇮🇹Firenze, Italy
Fondazione toscana Gabriele Monesterio per la ricarca medica Dipartimento Cardiotoracico UOC Cardiologia e Medicina Cardiovasculare Ospedale San Cataldo
🇮🇹Pisa, Italy
Dipartimento di Medicina Clinica e Molecolare Universita Sapienza di Roma Unita di Terapia Intensiva Cardiologica Azienda Ospedaliero Universitaria Sant'Andrea
🇮🇹Roma, Italy
Maastricht University Medical Center (MUMC)
🇳🇱Maastricht, Netherlands
Erasmus Medical Center Department of Cardiology
🇳🇱Rotterdam, Netherlands
Kardio Brynow S.C.
🇵🇱Katowice, Poland
Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego - Panstwowy Instytut Badawczy Centrum Zaburzen Rytmu Serca
🇵🇱Warszawa, Poland
Centro Hospitalar Do Baixo Vouga, EPE Cardiology Department
🇵🇹Aveiro, Portugal
Hospital da Luz
🇵🇹Lisboa, Portugal
Hospital Clinic Barcelona
🇪🇸Barcelona, Spain
Complejo Hospitalario Universitario A Coruna
🇪🇸Coruña, Spain
Hospital Universitario Puerta de Hierro Majadahonda
🇪🇸Madrid, Spain
Hospital Clinico Universitario Virgen de la Arrixaca
🇪🇸Madrid, Spain
Hospital Universitario Son Llatzer
🇪🇸Palma, Spain
Hospital Universitario de Salamanca
🇪🇸Salamanca, Spain
Hospital Universitario Virgen Macarena-merge
🇪🇸Sevilla, Spain
Hospital Universitario Virgen del Rocío
🇪🇸Sevilla, Spain
Queen Elizabeth Hospital Birmingham University Hospitals Birmingham NHS Foundation Trust
🇬🇧Birmingham, United Kingdom
Glasgow Clinical Research Facility Neuroscience Institute
🇬🇧Glasgow, United Kingdom
Liverpool Heart and Chest Hospital
🇬🇧Liverpool, United Kingdom
St Bartholomew's Hospital
🇬🇧London, United Kingdom
St George's University Hospitals NHS Foundation Trust
🇬🇧London, United Kingdom
Royal Brompton Hospital
🇬🇧London, United Kingdom
Oxford Centre for Clinical Magnetic Resonance Research (OCMR) Division of Cardiovascular Medicine Radcliffe Department of Medicine University of Oxford
🇬🇧Oxford, United Kingdom