The purpose of this study is to collect long-term safety and tolerability data for aficamten.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
900
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.
Incidence of adverse events observed during dosing of aficamten in patients with HCM
Patient incidence of reported Adverse Events (AEs)
Time frame: Baseline to End of study, up to 5 years
Incidence of serious adverse events observed during dosing of aficamten in patients with HCM
Patient incidence of reported Serious Adverse Events (SAEs)
Time frame: 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
Patient incidence of reported LVEF \<50%
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Baseline through the end of participation at 12-24 week intervals
Long-term effects of aficamten on post Valsalva LVOT-G
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University of Alabama at Birmingham
Birmingham, Alabama, United States
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 D.C., District of Columbia, United States
Holy Cross Hospital / Cardiology Associates
Fort Lauderdale, Florida, United States
...and 119 more locations
Proportion of patients with post-Valsalva LVOT-G \< 30 mmHg; applicable only to patients with oHCM
Time frame: 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
Time frame: 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 frame: 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 frame: 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 frame: 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 frame: Time to the following event through last follow-up, up to 5 years