A Phase 3 interventional study of Mavacamten in Cardiomyopathy, Hypertrophic Obstructive, sponsored by Bristol-Myers Squibb. Completed at 20 sites in Japan. Open to participants aged 18 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-01-27.
Sponsored by Bristol-Myers Squibb · Phase 3, Interventional, and Treatment
The purpose of this study is to evaluate the effectiveness, safety, and tolerability of a 30-week course of mavacamten and the long-term effects of mavacamten in Japanese participants with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
347 studies on the registry are indexed under Cardiomyopathy, Hypertrophic; 110 are open to participants now.
This study's enrollment of 38 is below the median of 59 across 171 interventional studies indexed under Cardiomyopathy, Hypertrophic.
Browse Cardiomyopathy, Hypertrophic studies →Bristol-Myers Squibb is the lead sponsor of 1,538 studies on the registry; 116 are open to participants now.
Of its 429 completed or terminated interventional studies of FDA-regulated products, 223 (52%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria apply
Drug: Mavacamten
Specified dose on specified days
Also known as: BMS-986427, MYK-461
Change From Baseline in Post-exercise Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30
The post-exercise LVOT gradient was measured from echocardiograms obtained at baseline and week 30 following a study-specified exercise protocol and read by the doppler echocardiography. Baseline is defined as the last non-missing assessment prior to the first dose of the study treatment if both the time of the measurement and the time of first dose are available otherwise it is the last non-missing assessment on or prior to the first dose of the study treatment.
Time frame: At Baseline and Week 30
Change From Baseline in Kansas City Cardiomyopathy Questionnaire 23-item Version (KCCQ-23) Clinical Summary Score (CSS) at Week 30
The KCCQ-23 is a 23-item, self-administered questionnaire that measures the impact of a participant's cardiovascular disease or its treatment on 6 distinct domains using a 2-week recall period: symptoms/signs, physical limitation, quality of life (QoL), social limitations, self-efficacy, and symptom stability. The KCCQ 23 Clinical Summary Score (CSS) is derived from the Total Symptom Score (TSS) and the Physical Limitations (PL) score of the KCCQ 23. The CSS, TSS, and the PL score range from 0 to 100 with higher scores representing less severe symptoms and/or physical limitations. The CSS is a mean of the TSS and the PL score. Baseline is defined as the last non-missing assessment prior to the first dose of the study treatment if both the time of the measurement and the time of first dose are available otherwise it is the last non-missing assessment on or prior to the first dose of the study treatment.
Time frame: At Baseline and Week 30
Percentage of Participants With at Least 1 Class Improvement in New York Heart Association (NYHA) Functional Class From Baseline to Week 30
The NYHA Functional Classification of Heart Failure (HF) assigns participants to 1 of 4 categories based on the participant's symptoms. Class I (No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea); Class II (Slight limitation of physical activity, Comfortable at rest, Ordinary physical activity results in fatigue, palpitation, dyspnea); Class III (Marked limitation of physical activity, Comfortable at rest, Less-than ordinary-activity causes fatigue, palpitation, or dyspnea) and Class IV (Unable to carry on any physical activity without discomfort. Symptoms of heart failure at rest. If any physical activity is undertaken, discomfort increases). Improvement is defined as participant moving to lower class category from a higher one. Baseline is defined as last non-missing measurement prior to the first dose.
Time frame: Baseline and at Week 30
Change From Baseline in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) at Week 30
Blood samples were collected for assessing the concentration of NT-proBNP. Baseline is defined as last non-missing measurement prior to the first dose.
Time frame: At baseline and week 30
Change From Baseline in Cardiac Troponin I at Week 30
Blood samples were collected for assessing cardiac troponins. Baseline is defined as the last non-missing assessment prior to the first dose of the study treatment if both the time of the measurement and the time of first dose are available otherwise it is the last non-missing assessment on or prior to the first dose of the study treatment.
Time frame: At baseline and week 30
Change From Baseline in Cardiac Troponin T at Week 30
Blood samples were collected for assessing cardiac troponins. Baseline is defined as the last non-missing assessment prior to the first dose of the study treatment if both the time of the measurement and the time of first dose are available otherwise it is the last non-missing assessment on or prior to the first dose of the study treatment.
Time frame: At baseline and week 30
| Milestone | Mavacamten |
|---|---|
| Started | 38 |
| Intent to treat population | 38 |
| Safety population | 38 |
| Completed | 36 |
| Not completed | 2 |
| Withdrew: Withdrawal by subject | 1 |
| Withdrew: Other reasons | 1 |
The post-exercise LVOT gradient was measured from echocardiograms obtained at baseline and week 30 following a study-specified exercise protocol and read by the doppler echocardiography. Baseline is defined as the last non-missing assessment prior to the first dose of the study treatment if both the time of the measurement and the time of first dose are available otherwise it is the last non-missing assessment on or prior to the first dose of the study treatment.
| millimeters of mercury (mmHg) | Mavacamten |
|---|---|
| Change From Baseline in Post-exercise Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30 | -60.6963 ± 31.55674 |
The KCCQ-23 is a 23-item, self-administered questionnaire that measures the impact of a participant's cardiovascular disease or its treatment on 6 distinct domains using a 2-week recall period: symptoms/signs, physical limitation, quality of life (QoL), social limitations, self-efficacy, and symptom stability. The KCCQ 23 Clinical Summary Score (CSS) is derived from the Total Symptom Score (TSS) and the Physical Limitations (PL) score of the KCCQ 23. The CSS, TSS, and the PL score range from 0 to 100 with higher scores representing less severe symptoms and/or physical limitations. The CSS is a mean of the TSS and the PL score. Baseline is defined as the last non-missing assessment prior to the first dose of the study treatment if both the time of the measurement and the time of first dose are available otherwise it is the last non-missing assessment on or prior to the first dose of the study treatment.
| Score on a Scale | Mavacamten |
|---|---|
| Change From Baseline in Kansas City Cardiomyopathy Questionnaire 23-item Version (KCCQ-23) Clinical Summary Score (CSS) at Week 30 | 9.766 ± 16.8588 |
The NYHA Functional Classification of Heart Failure (HF) assigns participants to 1 of 4 categories based on the participant's symptoms. Class I (No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea); Class II (Slight limitation of physical activity, Comfortable at rest, Ordinary physical activity results in fatigue, palpitation, dyspnea); Class III (Marked limitation of physical activity, Comfortable at rest, Less-than ordinary-activity causes fatigue, palpitation, or dyspnea) and Class IV (Unable to carry on any physical activity without discomfort. Symptoms of heart failure at rest. If any physical activity is undertaken, discomfort increases). Improvement is defined as participant moving to lower class category from a higher one. Baseline is defined as last non-missing measurement prior to the first dose.
| Percentage of participants | Mavacamten |
|---|---|
| Percentage of Participants With at Least 1 Class Improvement in New York Heart Association (NYHA) Functional Class From Baseline to Week 30 | 63.2 |
Blood samples were collected for assessing the concentration of NT-proBNP. Baseline is defined as last non-missing measurement prior to the first dose.
| nanogram per liter (ng/L) | Mavacamten |
|---|---|
| Change From Baseline in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) at Week 30 | -738.0 (-3101 to 590) |
Blood samples were collected for assessing cardiac troponins. Baseline is defined as the last non-missing assessment prior to the first dose of the study treatment if both the time of the measurement and the time of first dose are available otherwise it is the last non-missing assessment on or prior to the first dose of the study treatment.
| ng/L | Mavacamten |
|---|---|
| Change From Baseline in Cardiac Troponin I at Week 30 | -10.920 (-226.42 to 0.80) |
Blood samples were collected for assessing cardiac troponins. Baseline is defined as the last non-missing assessment prior to the first dose of the study treatment if both the time of the measurement and the time of first dose are available otherwise it is the last non-missing assessment on or prior to the first dose of the study treatment.
| ng/L | Mavacamten |
|---|---|
| Change From Baseline in Cardiac Troponin T at Week 30 | -4.95 (-19.2 to 0.3) |
Collected over All-cause mortality was measured from first dose up until approximately 66 weeks post-first dose. SAEs and Other AEs were collected from first dose till last dose plus 140 days (up to approximately 50 weeks).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Mavacamten | 0/38 (0%) | 6/38 (15.8%) | 20/38 (52.6%) |
| Event | Mavacamten |
|---|---|
| Atrial fibrillationCardiac disorders | 1/38 |
| Macular oedemaEye disorders | 1/38 |
| CholangitisHepatobiliary disorders | 1/38 |
| Cholecystitis acuteHepatobiliary disorders | 1/38 |
| COVID-19Infections and infestations | 1/38 |
| Colonic abscessInfections and infestations | 1/38 |
| Rotator cuff syndromeMusculoskeletal and connective tissue disorders | 1/38 |
| Cerebral haemorrhageNervous system disorders | 1/38 |
| Event | Mavacamten |
|---|---|
| COVID-19Infections and infestations | 8/38 |
| NasopharyngitisInfections and infestations | 6/38 |
| Atrial fibrillationCardiac disorders | 3/38 |
| PyrexiaGeneral disorders | 3/38 |
| HypertensionVascular disorders | 3/38 |
| PalpitationsCardiac disorders | 2/38 |
| CataractEye disorders | 2/38 |
| Abdominal painGastrointestinal disorders | 2/38 |
| MalaiseGeneral disorders | 2/38 |
| BronchitisInfections and infestations | 2/38 |
| Age, Continuous(years) | Mavacamten |
|---|---|
| Mean | 64.8 ± 10.95 |
| Sex: Female, Male(Participants) | Mavacamten |
|---|---|
| Female | 25 |
| Male | 13 |
| Race/Ethnicity, Customized(participants) | Mavacamten |
|---|---|
| Japanese | 38 |
Documents are hosted by the registry — open the source record to download them.
This study is completed, as verified in Jan 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Bristol-Myers Squibb