Trial Outcomes & Findings for Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM (NCT NCT05186818)
NCT ID: NCT05186818
Last Updated: 2026-03-03
Results Overview
The effect of CK-3773274 on exercise capacity in participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) was determined through changes in peak oxygen uptake (pVO2) after 24 weeks of treatment. pVO2 was measured by cardiopulmonary exercise testing (CPET) on a treadmill or bicycle. A higher pVO2 indicates better cardiorespiratory fitness.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
282 participants
Primary outcome timeframe
Baseline to Week 24
Results posted on
2026-03-03
Participant Flow
Participant milestones
| Measure |
Aficamten up to 20 mg
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Overall Study
STARTED
|
142
|
140
|
|
Overall Study
COMPLETED
|
137
|
136
|
|
Overall Study
NOT COMPLETED
|
5
|
4
|
Reasons for withdrawal
| Measure |
Aficamten up to 20 mg
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
2
|
|
Overall Study
Physician Decision
|
0
|
1
|
|
Overall Study
Protocol Violation
|
2
|
0
|
|
Overall Study
Withdrawal by Subject
|
2
|
0
|
|
Overall Study
Transportation issue due to COVID-19
|
0
|
1
|
Baseline Characteristics
Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM
Baseline characteristics by cohort
| Measure |
Aficamten up to 20 mg
n=142 Participants
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 Participants
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
Total
n=282 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
59.0 Years
n=41 Participants
|
60.0 Years
n=35 Participants
|
59.5 Years
n=76 Participants
|
|
Sex: Female, Male
Female
|
56 Participants
n=41 Participants
|
59 Participants
n=35 Participants
|
115 Participants
n=76 Participants
|
|
Sex: Female, Male
Male
|
86 Participants
n=41 Participants
|
81 Participants
n=35 Participants
|
167 Participants
n=76 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=76 Participants
|
|
Race/Ethnicity, Customized
Asian
|
29 Participants
n=41 Participants
|
25 Participants
n=35 Participants
|
54 Participants
n=76 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=76 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
3 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
3 Participants
n=76 Participants
|
|
Race/Ethnicity, Customized
White
|
108 Participants
n=41 Participants
|
115 Participants
n=35 Participants
|
223 Participants
n=76 Participants
|
|
Race/Ethnicity, Customized
More than one race
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=76 Participants
|
|
Race/Ethnicity, Customized
Other
|
2 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
2 Participants
n=76 Participants
|
|
Region of Enrollment
China
|
24 Participants
n=41 Participants
|
22 Participants
n=35 Participants
|
46 Participants
n=76 Participants
|
|
Region of Enrollment
Czechia
|
0 Participants
n=41 Participants
|
1 Participants
n=35 Participants
|
1 Participants
n=76 Participants
|
|
Region of Enrollment
Denmark
|
4 Participants
n=41 Participants
|
3 Participants
n=35 Participants
|
7 Participants
n=76 Participants
|
|
Region of Enrollment
France
|
9 Participants
n=41 Participants
|
8 Participants
n=35 Participants
|
17 Participants
n=76 Participants
|
|
Region of Enrollment
Germany
|
8 Participants
n=41 Participants
|
3 Participants
n=35 Participants
|
11 Participants
n=76 Participants
|
|
Region of Enrollment
Hungary
|
2 Participants
n=41 Participants
|
5 Participants
n=35 Participants
|
7 Participants
n=76 Participants
|
|
Region of Enrollment
Israel
|
4 Participants
n=41 Participants
|
5 Participants
n=35 Participants
|
9 Participants
n=76 Participants
|
|
Region of Enrollment
Italy
|
6 Participants
n=41 Participants
|
6 Participants
n=35 Participants
|
12 Participants
n=76 Participants
|
|
Region of Enrollment
Netherlands
|
1 Participants
n=41 Participants
|
6 Participants
n=35 Participants
|
7 Participants
n=76 Participants
|
|
Region of Enrollment
Poland
|
6 Participants
n=41 Participants
|
11 Participants
n=35 Participants
|
17 Participants
n=76 Participants
|
|
Region of Enrollment
Portugal
|
2 Participants
n=41 Participants
|
2 Participants
n=35 Participants
|
4 Participants
n=76 Participants
|
|
Region of Enrollment
Spain
|
17 Participants
n=41 Participants
|
15 Participants
n=35 Participants
|
32 Participants
n=76 Participants
|
|
Region of Enrollment
United Kingdom
|
10 Participants
n=41 Participants
|
8 Participants
n=35 Participants
|
18 Participants
n=76 Participants
|
|
Region of Enrollment
United States
|
49 Participants
n=41 Participants
|
45 Participants
n=35 Participants
|
94 Participants
n=76 Participants
|
|
pVO2 per CPET
|
18.4 mL/kg/min
STANDARD_DEVIATION 4.5 • n=41 Participants
|
18.6 mL/kg/min
STANDARD_DEVIATION 4.5 • n=35 Participants
|
18.5 mL/kg/min
STANDARD_DEVIATION 4.5 • n=76 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 24Population: Full Analysis Set (consisting of all randomized participants)
The effect of CK-3773274 on exercise capacity in participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) was determined through changes in peak oxygen uptake (pVO2) after 24 weeks of treatment. pVO2 was measured by cardiopulmonary exercise testing (CPET) on a treadmill or bicycle. A higher pVO2 indicates better cardiorespiratory fitness.
Outcome measures
| Measure |
Aficamten up to 20 mg
n=142 Participants
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 Participants
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Change From Baseline in pVO2 at Week 24
|
1.76 mL/kg/min
Standard Error 0.25
|
0.02 mL/kg/min
Standard Error 0.25
|
SECONDARY outcome
Timeframe: Baseline to Week 24Population: Full Analysis Set (consisting of all randomized participants)
The effect of aficamten on patient health status was assessed using the Kansas City Cardiomyopathy Questionnaire (KCCQ), a patient-reported outcome designed to assess the physical limitations, symptoms, self-efficacy, social limitation, and quality of life of patients with heart failure symptoms. The KCCQ-Clinical Symptoms Score (KCCQ-CSS) is scored on a scale from 0 to 100, with higher scores indicating better physical functioning and symptoms.
Outcome measures
| Measure |
Aficamten up to 20 mg
n=142 Participants
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 Participants
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Change From Baseline in KCCQ-CSS at Week 24
|
11.6 score on a scale
Standard Error 1.0
|
4.3 score on a scale
Standard Error 1.0
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: Full Analysis Set (consisting of all randomized participants)
The effect of aficamten on patient health status was assessed using the Kansas City Cardiomyopathy Questionnaire (KCCQ), a patient-reported outcome designed to assess the physical limitations, symptoms, self-efficacy, social limitation, and quality of life of patients with heart failure symptoms. The KCCQ-Clinical Symptoms Score (KCCQ-CSS) is scored on a scale from 0 to 100, with higher scores indicating better physical functioning and symptoms.
Outcome measures
| Measure |
Aficamten up to 20 mg
n=142 Participants
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 Participants
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Change From Baseline in KCCQ-CSS at Week 12
|
11.1 score on a scale
Standard Error 0.9
|
4.0 score on a scale
Standard Error 0.9
|
SECONDARY outcome
Timeframe: Baseline to Week 24Population: Full Analysis Set (consisting of all randomized participants)
The effect of aficamten on NYHA functional classification was evaluated through changes observed from baseline through 24 weeks of treatment.
Outcome measures
| Measure |
Aficamten up to 20 mg
n=142 Participants
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 Participants
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Proportion of Participants With ≥1 Class Improvement in New York Heart Association (NYHA) Functional Class From Baseline to Week 24
|
83 Participants
|
34 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: Full Analysis Set (consisting of all randomized participants)
The effect of aficamten on NYHA functional classification was evaluated through changes observed from baseline through 12 weeks of treatment.
Outcome measures
| Measure |
Aficamten up to 20 mg
n=142 Participants
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 Participants
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Proportion of Participants With ≥1 Class Improvement in NYHA Functional Class From Baseline to Week 12
|
69 Participants
|
25 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 24Population: Full Analysis Set (consisting of all randomized participants)
The effect of aficamten treatment on Valsalva LVOT-G was evaluated through changes from baseline to Week 24.
Outcome measures
| Measure |
Aficamten up to 20 mg
n=142 Participants
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 Participants
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Change From Baseline in Valsalva Left Ventricular Outflow Tract Gradient (LVOT-G) at Week 24
|
-48 mmHg
Standard Error 2.4
|
2 mmHg
Standard Error 2.4
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: Full Analysis Set (consisting of all randomized participants)
The effect of aficamten treatment on Valsalva LVOT-G was evaluated through changes from baseline to Week 12.
Outcome measures
| Measure |
Aficamten up to 20 mg
n=142 Participants
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 Participants
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Change From Baseline in Valsalva LVOT-G at Week 12
|
-46 mmHg
Standard Error 2.4
|
3 mmHg
Standard Error 2.4
|
SECONDARY outcome
Timeframe: Baseline to Week 24Population: Full Analysis Set (consisting of all randomized participants)
The effect of aficamten treatment on Valsalva LVOT-G was evaluated through changes from baseline to Week 24.
Outcome measures
| Measure |
Aficamten up to 20 mg
n=142 Participants
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 Participants
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Proportion of Participants With Valsalva LVOT G <30 mmHg at Week 24
|
70 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: Full Analysis Set (consisting of all randomized participants)
The effect of aficamten treatment on Valsalva LVOT-G was evaluated through changes from baseline to Week 12.
Outcome measures
| Measure |
Aficamten up to 20 mg
n=142 Participants
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 Participants
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Proportion of Participants With Valsalva LVOT G <30 mmHg at Week 12
|
74 Participants
|
8 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 24Population: Participants in the Full Analysis Set who were SRT eligible at baseline
The effect of aficamten treatment on the duration of septal reduction therapy (SRT) eligibility was evaluated over the 24- week treatment period.
Outcome measures
| Measure |
Aficamten up to 20 mg
n=32 Participants
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=29 Participants
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Duration of SRT Eligibility During the 24-week Treatment Period for Participants Who Were SRT Eligible at Baseline
|
35.3 days
Standard Error 7.9
|
113.4 days
Standard Error 8.1
|
SECONDARY outcome
Timeframe: Baseline to Week 24Population: Full Analysis Set (consisting of all randomized participants)
Effect of aficamten on intensity of exercise (based on speed, incline, participant weight, etc.) during CPET. Workload is an indication of the energy expended during the exercise test.
Outcome measures
| Measure |
Aficamten up to 20 mg
n=142 Participants
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 Participants
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Change From Baseline to Week 24 in Total Workload During CPET
|
13.4 watts
Standard Error 2.1
|
1.2 watts
Standard Error 2.1
|
Adverse Events
Aficamten up to 20 mg
Serious events: 8 serious events
Other events: 44 other events
Deaths: 0 deaths
Placebo to Match Aficamten
Serious events: 13 serious events
Other events: 46 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Aficamten up to 20 mg
n=142 participants at risk
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 participants at risk
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Blood and lymphatic system disorders
Microcytic anaemia
|
0.70%
1/142 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.00%
0/140 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Cardiac disorders
Acute coronary syndrome
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Cardiac disorders
Acute myocardial infarction
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Cardiac disorders
Arrhythmia supraventricular
|
0.70%
1/142 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.00%
0/140 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Cardiac disorders
Atrial fibrillation
|
0.70%
1/142 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 2 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Cardiac disorders
Cardiac failure congestive
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Cardiac disorders
Sinoatrial block
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Cardiac disorders
Ventricular fibrillation
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Nervous system disorders
Loss of consciousness
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Congenital, familial and genetic disorders
Hypertrophic cardiomyopathy
|
2.1%
3/142 • Number of events 3 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Nervous system disorders
Syncope
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Congenital, familial and genetic disorders
Thalassaemia
|
0.70%
1/142 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.00%
0/140 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Gastrointestinal disorders
Upper gastrointestinal haemorrhage
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Hepatobiliary disorders
Cholecystitis
|
0.70%
1/142 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.00%
0/140 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Infections and infestations
Cellulitis
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Infections and infestations
Pneumonia
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Infections and infestations
Sepsis
|
0.70%
1/142 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.00%
0/140 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Injury, poisoning and procedural complications
Radius fracture
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Nervous system disorders
Ischaemic stroke
|
0.70%
1/142 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.00%
0/140 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Injury, poisoning and procedural complications
Ulna fracture
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Injury, poisoning and procedural complications
Wrist fracture
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute lymphocytic leukaemia
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma of the oral cavity
|
0.00%
0/142 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.71%
1/140 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Nervous system disorders
Carotid artery stenosis
|
0.70%
1/142 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.00%
0/140 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.70%
1/142 • Number of events 1 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
0.00%
0/140 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
Other adverse events
| Measure |
Aficamten up to 20 mg
n=142 participants at risk
Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks.
Aficamten (5 mg, 10 mg, 15 mg, and 20 mg): Aficamten tablets were administered orally once daily.
|
Placebo to Match Aficamten
n=140 participants at risk
Participants received placebo for up to 24 weeks.
Placebo to match aficamten: Placebo tablets were administered orally once daily.
|
|---|---|---|
|
Nervous system disorders
Headache
|
7.7%
11/142 • Number of events 12 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
7.1%
10/140 • Number of events 13 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
5.6%
8/142 • Number of events 8 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
5.7%
8/140 • Number of events 8 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Vascular disorders
Hypertension
|
7.7%
11/142 • Number of events 11 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
2.1%
3/140 • Number of events 3 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Cardiac disorders
Angina pectoris
|
2.1%
3/142 • Number of events 4 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
5.0%
7/140 • Number of events 12 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Cardiac disorders
Palpitations
|
7.0%
10/142 • Number of events 12 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
2.9%
4/140 • Number of events 5 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
General disorders
Fatigue
|
2.1%
3/142 • Number of events 3 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
5.0%
7/140 • Number of events 9 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Infections and infestations
COVID-19
|
5.6%
8/142 • Number of events 8 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
6.4%
9/140 • Number of events 11 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
|
Infections and infestations
Upper respiratory tract infection
|
6.3%
9/142 • Number of events 13 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
8.6%
12/140 • Number of events 16 • 28 Weeks
All reported adverse events (AEs) were treatment-emergent, defined as investigator-reported events starting on or after the first dose of study drug and up to and including 28 days after the last dose of study drug. AEs were collected and reported by randomized treatment groups, Aficamten and Placebo. Participants were randomized to treatment, not dose level: Aficamten doses were individually titrated based on echocardiography-guided criteria.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Cytokinetics agreements with investigators vary; constant is Cytokinetics' right to review communications regarding trial results prior to public release. Cytokinetics does not prohibit investigators from publishing, but single-center publications must be postponed until after release of the first multi-center publication for the trial. Investigators may not disclose previously undisclosed confidential information other than study data and results from their site.
- Publication restrictions are in place
Restriction type: OTHER