Trial Outcomes & Findings for A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM) (NCT NCT03442764)

NCT ID: NCT03442764

Last Updated: 2022-08-09

Results Overview

This is the percentage of participants who experienced at least one treatment emergent adverse event (TEAE)

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

59 participants

Primary outcome timeframe

From first dose to 8 weeks following last dose (Up to 24 weeks)

Results posted on

2022-08-09

Participant Flow

In total, 157 participants were assessed for eligibility and 59 were enrolled from 35 sites in the United States.

59 participants were randomized and 58 entered the treatment period.

Participant milestones

Participant milestones
Measure	Group 1 Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~200 ng/ml. Dose adjustments were done at Week 6 based on PK	Group 2 Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~500 ng/ml. Dose adjustments were done at Week 6 based on PK	Placebo Placebo Group
Overall Study STARTED	19	21	19
Overall Study COMPLETED	18	21	19
Overall Study NOT COMPLETED	1	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Group 1 Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~200 ng/ml. Dose adjustments were done at Week 6 based on PK	Group 2 Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~500 ng/ml. Dose adjustments were done at Week 6 based on PK	Placebo Placebo Group
Overall Study Adverse Event	1	0	0

Baseline Characteristics

A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Group 1 n=19 Participants Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~200 ng/ml. Dose adjustments were done at Week 6 based on PK	Group 2 n=21 Participants Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~500 ng/ml. Dose adjustments were done at Week 6 based on PK	Placebo n=19 Participants Placebo Group	Total n=59 Participants Total of all reporting groups
Age, Continuous	58.3 years STANDARD_DEVIATION 13.7 • n=5 Participants	50.0 years STANDARD_DEVIATION 14.7 • n=7 Participants	53.8 years STANDARD_DEVIATION 18.2 • n=5 Participants	53.9 years STANDARD_DEVIATION 15.7 • n=4 Participants
Sex: Female, Male Female	9 Participants n=5 Participants	12 Participants n=7 Participants	13 Participants n=5 Participants	34 Participants n=4 Participants
Sex: Female, Male Male	10 Participants n=5 Participants	9 Participants n=7 Participants	6 Participants n=5 Participants	25 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	5 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	18 Participants n=5 Participants	19 Participants n=7 Participants	15 Participants n=5 Participants	52 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants
Race (NIH/OMB) White	17 Participants n=5 Participants	18 Participants n=7 Participants	17 Participants n=5 Participants	52 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants	4 Participants n=4 Participants
NT-proBNP	1160.7 ng/L STANDARD_DEVIATION 859.02 • n=5 Participants	933.3 ng/L STANDARD_DEVIATION 719.41 • n=7 Participants	1164.1 ng/L STANDARD_DEVIATION 817.70 • n=5 Participants	1080.8 ng/L STANDARD_DEVIATION 791.97 • n=4 Participants
Troponin-I Elevated	6 participants n=5 Participants	7 participants n=7 Participants	6 participants n=5 Participants	19 participants n=4 Participants
Troponin-I Detectable Not Elevated	6 participants n=5 Participants	10 participants n=7 Participants	5 participants n=5 Participants	21 participants n=4 Participants
Troponin-I Undetectable	6 participants n=5 Participants	4 participants n=7 Participants	8 participants n=5 Participants	18 participants n=4 Participants
Troponin-I Missing	1 participants n=5 Participants	0 participants n=7 Participants	0 participants n=5 Participants	1 participants n=4 Participants
New York Heart Association (NYHA) Functional Classification Class II	15 Participants n=5 Participants	18 Participants n=7 Participants	13 Participants n=5 Participants	46 Participants n=4 Participants
New York Heart Association (NYHA) Functional Classification Class III	4 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants	13 Participants n=4 Participants

PRIMARY outcome

Timeframe: From first dose to 8 weeks following last dose (Up to 24 weeks)

Population: All treated participants

This is the percentage of participants who experienced at least one treatment emergent adverse event (TEAE)

Outcome measures

Outcome measures
Measure	Group 1 n=18 Participants Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~200 ng/ml. Dose adjustments were done at Week 6 based on PK	Group 2 n=21 Participants Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~500 ng/ml. Dose adjustments were done at Week 6 based on PK	Placebo n=19 Participants Placebo Group
Percentage of Participants Who Experienced at Least One Treatment Emergent Adverse Event (TEAE)	88.9 percentage of participants	90.5 percentage of participants	68.4 percentage of participants

PRIMARY outcome

Timeframe: From first dose to 8 weeks following last dose (Up to 24 weeks)

Population: All treated participants

This is the percentage of participants who experienced at least one serious treatment-emergent adverse event (STEAE)

Outcome measures

Outcome measures
Measure	Group 1 n=18 Participants Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~200 ng/ml. Dose adjustments were done at Week 6 based on PK	Group 2 n=21 Participants Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~500 ng/ml. Dose adjustments were done at Week 6 based on PK	Placebo n=19 Participants Placebo Group
Percentage of Participants Who Experienced at Least One Serious Treatment-emergent Adverse Event (STEAE)	11.1 percentage of participants	9.5 percentage of participants	21.1 percentage of participants

Adverse Events

Group 1 Mavacamten

Serious events: 2 serious events

Other events: 16 other events

Deaths: 0 deaths

Group 2 Mavacamten

Serious events: 2 serious events

Other events: 18 other events

Deaths: 0 deaths

Combined MAVA Treatment Group (Group 1+ Group 2)

Serious events: 4 serious events

Other events: 34 other events

Deaths: 0 deaths

Placebo

Serious events: 4 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Group 1 Mavacamten n=18 participants at risk Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~200 ng/ml (Group 1). Dose adjustments were done at Week 6 based on PK	Group 2 Mavacamten n=21 participants at risk Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~500 ng/ml (Group 2). Dose adjustments were done at Week 6 based on PK	Combined MAVA Treatment Group (Group 1+ Group 2) n=39 participants at risk Received active 16-week course of mavacamten doses titrated to achieve target drug concentrations of \~200 ng/ml (Group 1) or \~500 ng/ml (Group 2). Dose adjustments were done at Week 6 based on PK	Placebo n=19 participants at risk Placebo Group
Cardiac disorders Atrial fibrillation	0.00% 0/18 • From first dose to 8 weeks following last dose (Up to 24 weeks)	9.5% 2/21 • From first dose to 8 weeks following last dose (Up to 24 weeks)	5.1% 2/39 • From first dose to 8 weeks following last dose (Up to 24 weeks)	5.3% 1/19 • From first dose to 8 weeks following last dose (Up to 24 weeks)
Cardiac disorders Systolic dysfunction	0.00% 0/18 • From first dose to 8 weeks following last dose (Up to 24 weeks)	4.8% 1/21 • From first dose to 8 weeks following last dose (Up to 24 weeks)	2.6% 1/39 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/19 • From first dose to 8 weeks following last dose (Up to 24 weeks)
Cardiac disorders Angina pectoris	0.00% 0/18 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/21 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/39 • From first dose to 8 weeks following last dose (Up to 24 weeks)	5.3% 1/19 • From first dose to 8 weeks following last dose (Up to 24 weeks)
Cardiac disorders Atrial Flutter	0.00% 0/18 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/21 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/39 • From first dose to 8 weeks following last dose (Up to 24 weeks)	5.3% 1/19 • From first dose to 8 weeks following last dose (Up to 24 weeks)
Cardiac disorders Coronary artery disease	0.00% 0/18 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/21 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/39 • From first dose to 8 weeks following last dose (Up to 24 weeks)	5.3% 1/19 • From first dose to 8 weeks following last dose (Up to 24 weeks)
Musculoskeletal and connective tissue disorders Arthritis	5.6% 1/18 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/21 • From first dose to 8 weeks following last dose (Up to 24 weeks)	2.6% 1/39 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/19 • From first dose to 8 weeks following last dose (Up to 24 weeks)
Psychiatric disorders Mental status changes	5.6% 1/18 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/21 • From first dose to 8 weeks following last dose (Up to 24 weeks)	2.6% 1/39 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/19 • From first dose to 8 weeks following last dose (Up to 24 weeks)
Renal and urinary disorders Renal Failure	5.6% 1/18 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/21 • From first dose to 8 weeks following last dose (Up to 24 weeks)	2.6% 1/39 • From first dose to 8 weeks following last dose (Up to 24 weeks)	0.00% 0/19 • From first dose to 8 weeks following last dose (Up to 24 weeks)

Other adverse events

Additional Information

Bristol-Myers Squibb Study Director

Bristol-Myers Squibb

Phone: Please Email

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place