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)
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
Scroll for more (80 remaining)Alaska Heart and Vascular Institute🇺🇸Anchorage, Alaska, United States