Trial Outcomes & Findings for Study Evaluating the Pharmacokinetics of Mavacamten in Healthy Adult Chinese Subjects (NCT NCT05135871)
NCT ID: NCT05135871
Last Updated: 2024-07-25
Results Overview
Determination of pharmacokinetics parameters as measured by area under curve AUC(0-last), AUC(0-inf)
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
45 participants
Primary outcome timeframe
Predose, Day 1, Day 7, Day 10, Day 14, Day 21, Day 28, Day 35, Day 45, Day 67 and Day 75 post-dose
Results posted on
2024-07-25
Participant Flow
Participant milestones
| Measure |
Cohort 2: Mavacamten 25 mg
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 1: Mavacamten 15 mg
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
13
|
12
|
12
|
8
|
|
Overall Study
COMPLETED
|
12
|
12
|
12
|
8
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
0
|
0
|
Reasons for withdrawal
| Measure |
Cohort 2: Mavacamten 25 mg
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 1: Mavacamten 15 mg
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Overall Study
1 subject withdrew from the study before administration of the IMP due to pre-dose abnormal ECG
|
1
|
0
|
0
|
0
|
Baseline Characteristics
Study Evaluating the Pharmacokinetics of Mavacamten in Healthy Adult Chinese Subjects
Baseline characteristics by cohort
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 Participants
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 Participants
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1 13 subjects were assigned to Cohort 2 but 1 subject (Cohort 2) withdrew from the study before administration of the study drug due to pre-dose abnormal ECG and was excluded from the pharmacokinetic analysis set and safety analysis set.
|
Cohort 3: Mavacamten 15 mg
n=12 Participants
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 Participants
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Total
n=44 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
26.8 years
STANDARD_DEVIATION 4.99 • n=5 Participants
|
31.0 years
STANDARD_DEVIATION 7.12 • n=7 Participants
|
28.9 years
STANDARD_DEVIATION 6.32 • n=5 Participants
|
35.4 years
STANDARD_DEVIATION 4.31 • n=4 Participants
|
30.1 years
STANDARD_DEVIATION 6.44 • n=21 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
11 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
33 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Chinese
|
12 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
44 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Non-Chinese
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Han
|
12 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
44 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Non-Han
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Region of Enrollment
China
|
12 participants
n=5 Participants
|
12 participants
n=7 Participants
|
12 participants
n=5 Participants
|
8 participants
n=4 Participants
|
44 participants
n=21 Participants
|
|
Body mass index
|
24.34 kg/m^2
STANDARD_DEVIATION 2.444 • n=5 Participants
|
23.82 kg/m^2
STANDARD_DEVIATION 2.571 • n=7 Participants
|
23.26 kg/m^2
STANDARD_DEVIATION 2.434 • n=5 Participants
|
23.04 kg/m^2
STANDARD_DEVIATION 2.312 • n=4 Participants
|
23.67 kg/m^2
STANDARD_DEVIATION 2.421 • n=21 Participants
|
|
CYP2C19 genotype
Ultra-rapid metabolizer
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
CYP2C19 genotype
Rapid metabolizer
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
CYP2C19 genotype
Normal metabolizer
|
11 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
22 Participants
n=21 Participants
|
|
CYP2C19 genotype
Intermediate metabolizer
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
12 Participants
n=21 Participants
|
|
CYP2C19 genotype
Poor metabolizer
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
8 Participants
n=21 Participants
|
|
Left ventricular ejection fraction(%)
|
68.0 %
STANDARD_DEVIATION 5.03 • n=5 Participants
|
66.9 %
STANDARD_DEVIATION 396 • n=7 Participants
|
66.4 %
STANDARD_DEVIATION 3.99 • n=5 Participants
|
64.0 %
STANDARD_DEVIATION 5.58 • n=4 Participants
|
66.5 %
STANDARD_DEVIATION 4.63 • n=21 Participants
|
PRIMARY outcome
Timeframe: Predose, Day 1, Day 7, Day 10, Day 14, Day 21, Day 28, Day 35, Day 45, Day 67 and Day 75 post-dosePopulation: One subject (Cohort 3) was excluded from the pharmacokinetic analysis set due to a major protocol deviation (use of prohibited drug moderate-acting CYP2C19 inhibitor "Omeprazole") affecting the assessment of PK parameters.
Determination of pharmacokinetics parameters as measured by area under curve AUC(0-last), AUC(0-inf)
Outcome measures
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 Participants
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 Participants
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
n=11 Participants
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 Participants
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Area Under the Curve (AUC) (0-last), AUC(0-inf)
(AUC) (0-last)
|
9956 h*ng/mL
Standard Deviation 35.61
|
20040 h*ng/mL
Standard Deviation 50.49
|
18270 h*ng/mL
Standard Deviation 21.05
|
39980 h*ng/mL
Standard Deviation 23.60
|
|
Area Under the Curve (AUC) (0-last), AUC(0-inf)
AUC(0-inf)
|
10030 h*ng/mL
Standard Deviation 35.44
|
20280 h*ng/mL
Standard Deviation 52.47
|
18430 h*ng/mL
Standard Deviation 21.18
|
45810 h*ng/mL
Standard Deviation 25.51
|
PRIMARY outcome
Timeframe: Predose, Day 1, Day 7, Day 10, Day 14, Day 21, Day 28, Day 35, Day 45, Day 67 and Day 75 post-dosePopulation: One subject (Cohort 3) was excluded from the pharmacokinetic analysis set due to a major protocol deviation (use of prohibited drug moderate-acting CYP2C19 inhibitor "Omeprazole") affecting the assessment of PK parameters.
Determination of pharmacokinetics parameters as measured by maximum concentration (Cmax)
Outcome measures
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 Participants
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 Participants
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
n=11 Participants
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 Participants
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Maximum Concentration (Cmax)
|
395.4 ng/mL
Standard Deviation 39.95
|
571.8 ng/mL
Standard Deviation 25.69
|
485.2 ng/mL
Standard Deviation 34.96
|
435.7 ng/mL
Standard Deviation 35.66
|
PRIMARY outcome
Timeframe: Predose, Day 1, Day 7, Day 10, Day 14, Day 21, Day 28, Day 35, Day 45, Day 67 and Day 75 post-dosePopulation: One subject (Cohort 3) was excluded from the pharmacokinetic analysis set due to a major protocol deviation (use of prohibited drug moderate-acting CYP2C19 inhibitor "Omeprazole") affecting the assessment of PK parameters.
Determination of pharmacokinetics parameters as measured by time to maximum concentration (Tmax)
Outcome measures
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 Participants
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 Participants
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
n=11 Participants
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 Participants
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Time to Maximum Concentration (Tmax)
|
0.88 h
Interval 0.5 to 3.0
|
1.50 h
Interval 0.5 to 4.0
|
0.75 h
Interval 0.5 to 3.0
|
0.63 h
Interval 0.33 to 1.0
|
PRIMARY outcome
Timeframe: Predose, Day 1, Day 7, Day 10, Day 14, Day 21, Day 28, Day 35, Day 45, Day 67 and Day 75 post-dosePopulation: One subject (Cohort 3) was excluded from the pharmacokinetic analysis set due to a major protocol deviation (use of prohibited drug moderate-acting CYP2C19 inhibitor "Omeprazole") affecting the assessment of PK parameters.
Determination of pharmacokinetics parameters as measured by elimination half-life (T1⁄2)
Outcome measures
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 Participants
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 Participants
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
n=11 Participants
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 Participants
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Elimination Half-life (T1⁄2)
|
120.3 h
Standard Deviation 23.44
|
143.6 h
Standard Deviation 42.32
|
205.4 h
Standard Deviation 22.31
|
572.0 h
Standard Deviation 25.03
|
PRIMARY outcome
Timeframe: Predose, Day 1, Day 7, Day 10, Day 14, Day 21, Day 28, Day 35, Day 45, Day 67 and Day 75 post-dosePopulation: One subject (Cohort 3) was excluded from the pharmacokinetic analysis set due to a major protocol deviation (use of prohibited drug moderate-acting CYP2C19 inhibitor "Omeprazole") affecting the assessment of PK parameters.
Determination of pharmacokinetics parameters as measured by apparent volume of distribution (Vd/F)
Outcome measures
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 Participants
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 Participants
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
n=11 Participants
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 Participants
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Apparent Volume of Distribution (Vd/F)
|
259500 mL
Standard Deviation 29.93
|
255300 mL
Standard Deviation 16.60
|
241200 mL
Standard Deviation 23.13
|
270200 mL
Standard Deviation 26.95
|
PRIMARY outcome
Timeframe: Predose, Day 1, Day 7, Day 10, Day 14, Day 21, Day 28, Day 35, Day 45, Day 67 and Day 75 post-dosePopulation: One subject (Cohort 3) was excluded from the pharmacokinetic analysis set due to a major protocol deviation (use of prohibited drug moderate-acting CYP2C19 inhibitor "Omeprazole") affecting the assessment of PK parameters.
Determination of pharmacokinetics parameters as measured by apparent clearance (CL/F)
Outcome measures
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 Participants
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 Participants
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
n=11 Participants
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 Participants
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Apparent Clearance (CL/F)
|
1495 mL/h
Standard Deviation 35.44
|
1233 mL/h
Standard Deviation 52.47
|
814.0 mL/h
Standard Deviation 21.18
|
327.5 mL/h
Standard Deviation 25.51
|
SECONDARY outcome
Timeframe: Up to 75 daysPopulation: All 44 subjects included in the safety analysis set: cohort 1 12 subjects, cohort 2 12 subjects, cohort 3 12 subjects, cohort 4 8 subjects.
Safety assessments will be performed by incidence of adverse events during the whole study at specified time points
Outcome measures
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 Participants
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 Participants
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
n=12 Participants
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 Participants
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Number of Participants With Adverse Events
TEAE leading to death
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Adverse Events
Number of subjects with at least 1 AE
|
3 Participants
|
6 Participants
|
9 Participants
|
3 Participants
|
|
Number of Participants With Adverse Events
Number of subjects with at least 1 TEAE
|
3 Participants
|
6 Participants
|
9 Participants
|
3 Participants
|
|
Number of Participants With Adverse Events
Worst severity of TEAE(Mild)
|
3 Participants
|
6 Participants
|
8 Participants
|
3 Participants
|
|
Number of Participants With Adverse Events
Worst severity of TEAE(Moderate)
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Adverse Events
Worst severity of TEAE(Severe)
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Adverse Events
Drug-related TEAEs
|
0 Participants
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Adverse Events
TESAE
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Adverse Events
TEAE leading to study discontinuation
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Adverse Events
AESI
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Body weight (kg) will be measured at screening (baseline) and EOS(75 days).Population: All 44 subjects included in the safety analysis set: cohort 1 12 subjects, cohort 2 12 subjects, cohort 3 12 subjects, cohort 4 8 subjects.
Safety assessments will be performed by vital signs during the whole study at screening, Day-1, Day 3 and at the EOS Visit. A complete physical examination includes assessments of general appearance, skin, head and neck, chest, abdomen, back, spine, extremity neurological, and mental systems. Brief physical examination means chest examinations. Height (cm) and body weight (kg) will be measured at screening, and BMI (kg/m2) will be calculated. Body weight (kg) will be measured at EOS. The mean weight and the standard deviation are analysis for different cohort.
Outcome measures
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 Participants
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 Participants
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
n=12 Participants
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 Participants
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Body Weight at Screening and EOS
Change from Baseline
|
0.80 kg
Standard Deviation 2.899
|
0.70 kg
Standard Deviation 1.738
|
-0.36 kg
Standard Deviation 2.378
|
1.25 kg
Standard Deviation 2.184
|
|
Body Weight at Screening and EOS
Baseline
|
69.41 kg
Standard Deviation 12.699
|
62.78 kg
Standard Deviation 8.458
|
65.89 kg
Standard Deviation 11.157
|
68.26 kg
Standard Deviation 11.147
|
|
Body Weight at Screening and EOS
Day 75
|
70.21 kg
Standard Deviation 13.088
|
63.48 kg
Standard Deviation 9.405
|
65.53 kg
Standard Deviation 10.929
|
69.51 kg
Standard Deviation 11.530
|
SECONDARY outcome
Timeframe: Up to 75 daysPopulation: All 44 subjects included in the safety analysis set: cohort 1 12 subjects, cohort 2 12 subjects, cohort 3 12 subjects, cohort 4 8 subjects.
Safety assessments will be performed by physical examination findings during the whole study at specified time points
Outcome measures
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 Participants
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 Participants
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
n=12 Participants
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 Participants
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Physical Examination Findings
Number of abnormal but not clinically significant comprehensive physical examinations in baseline
|
5 participants
|
2 participants
|
4 participants
|
2 participants
|
|
Physical Examination Findings
Number of abnormal but not clinically significant comprehensive physical examinations in Day 75
|
5 participants
|
2 participants
|
4 participants
|
2 participants
|
|
Physical Examination Findings
The number of abnormal with clinically significant comprehensive physical examinations in Day 75
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: Up to 75 daysPopulation: All 44 subjects included in the safety analysis set: cohort 1 12 subjects, cohort 2 12 subjects, cohort 3 12 subjects, cohort 4 8 subjects.
Safety assessments will be performed by electrocardiogram (ECG) parameters findings during the whole study at specified time points
Outcome measures
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 Participants
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 Participants
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
n=12 Participants
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 Participants
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Heart Rate at Baseline and Day 75
Heart Rate-Baseline
|
63.3 beats/min
Standard Deviation 8.70
|
63.0 beats/min
Standard Deviation 10.44
|
63.3 beats/min
Standard Deviation 10.44
|
62.8 beats/min
Standard Deviation 9.27
|
|
Heart Rate at Baseline and Day 75
Heart Rate-D75
|
64.8 beats/min
Standard Deviation 7.12
|
66.4 beats/min
Standard Deviation 7.88
|
67.5 beats/min
Standard Deviation 5.35
|
66.8 beats/min
Standard Deviation 7.17
|
SECONDARY outcome
Timeframe: Up to 75 daysPopulation: All 44 subjects included in the safety analysis set: cohort 1 12 subjects, cohort 2 12 subjects, cohort 3 12 subjects, cohort 4 8 subjects.
Safety assessments will be performed by clinical laboratory tests data(including hematology and blood chemistry, coagulation and urinalysis parameters) during the whole study at specified time points
Outcome measures
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 Participants
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 Participants
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
n=12 Participants
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 Participants
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Clinical Laboratory Tests Data
Blood fibrinogen increased
|
0 participants
|
0 participants
|
1 participants
|
1 participants
|
|
Clinical Laboratory Tests Data
White blood cell count decreased
|
0 participants
|
1 participants
|
0 participants
|
1 participants
|
|
Clinical Laboratory Tests Data
Alanine aminotransferase increased
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Clinical Laboratory Tests Data
Neutrophil count increased
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Clinical Laboratory Tests Data
Neutrophil count decreased
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Clinical Laboratory Tests Data
Eosinophil percentage increased
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Clinical Laboratory Tests Data
Eosinophil count increased
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Clinical Laboratory Tests Data
Urobilinogen urine increased
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Clinical Laboratory Tests Data
White blood cell count increased
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Clinical Laboratory Tests Data
Hemoglobin decreased
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
Adverse Events
Cohort 1: Mavacamten 15 mg
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 2: Mavacamten 25 mg
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Cohort 3: Mavacamten 15 mg
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Cohort 4: Mavacamten 15 mg
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Cohort 1: Mavacamten 15 mg
n=12 participants at risk
Cohort 1: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 2: Mavacamten 25 mg
n=12 participants at risk
Cohort 2: 10 mg capsules x 1 and 15 mg capsules x 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 25 mg on Day 1
|
Cohort 3: Mavacamten 15 mg
n=12 participants at risk
Cohort 3: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
Cohort 4: Mavacamten 15 mg
n=8 participants at risk
Cohort 4: 15 mg capsules × 1 on Day 1
Mavacamten: Single fasted oral dose of Mavacamten 15 mg on Day 1
|
|---|---|---|---|---|
|
Infections and infestations
Upper respiratory tract infection
|
16.7%
2/12 • Number of events 2 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
25.0%
3/12 • Number of events 4 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
25.0%
3/12 • Number of events 3 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
12.5%
1/8 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Infections and infestations
Oral herpes
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Infections and infestations
Appendicitis
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
General disorders
Chest pain
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
16.7%
2/12 • Number of events 2 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
General disorders
Malaise
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Nervous system disorders
Headache
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Eye disorders
Conjunctival hyperaemia
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Gastrointestinal disorders
Haematochezia
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Gastrointestinal disorders
Mouth ulceration
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Investigations
Blood fibrinogen increased
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
16.7%
2/12 • Number of events 2 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Investigations
White blood cell count decreased
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
12.5%
1/8 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Investigations
Neutrophil count increased
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Investigations
Neutrophil count decreased
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
12.5%
1/8 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Investigations
Eosinophil percentage increased
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Investigations
Eosinophil count increased
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Investigations
Urobilinogen urine increased
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
12.5%
1/8 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Investigations
White blood cell count increased
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
8.3%
1/12 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/8 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
|
Investigations
Hemoglobin decreased
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
0.00%
0/12 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
12.5%
1/8 • Number of events 1 • The study took about 80 days to monitor the adverse event for each subject.
Consistent with clinicaltrials.gov definitions.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place