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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Mavacamten | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Mavacamten | Drug | Specified dose on specified days |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | At Baseline and Week 30 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria apply
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| Name | Affiliation | Role |
|---|---|---|
| Bristol-Myers Squibb | Bristol-Myers Squibb | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Local Institution - 0020 | Uwajima | Ehime | 798-8510 | Japan | ||
| Local Institution - 0026 |
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| Label | URL |
|---|---|
| BMS Clinical Trial Information | View source |
| BMS Clinical Trial Patient Recruiting | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Mavacamten | Participants with symptomatic obstructive hypertrophic cardiomyopathy received oral capsule of mavacamten during the treatment period from Day 1 to Week 30. The starting dose of mavacamten was 2.5 milligram (mg) once daily. At Week 6, Week 8, Week 14, and Week 20, mavacamten dose were titrated based on the individual, transthoracic echocardiogram (TTE) response (Valsalva left ventricular outflow tract [LVOT] gradient and left ventricular ejection fraction LVEF). The permissible doses during the study were 1, 2.5, 5, 10, or 15 mg. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Mavacamten | Participants with symptomatic obstructive hypertrophic cardiomyopathy received oral capsule of mavacamten during the treatment period from Day 1 to Week 30. The starting dose of mavacamten was 2.5 milligram (mg) once daily. At Week 6, Week 8, Week 14, and Week 20, mavacamten dose were titrated based on the individual, transthoracic echocardiogram (TTE) response (Valsalva left ventricular outflow tract [LVOT] gradient and left ventricular ejection fraction LVEF). The permissible doses during the study were 1, 2.5, 5, 10, or 15 mg. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Intent-to-treat population. Only those participants with data available at the specified time points were analyzed. | Posted | Mean | Standard Deviation | millimeters of mercury (mmHg) | At Baseline and Week 30 |
|
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).
Safety analysis population included all participants who receive at least one dose of study drug.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Mavacamten | Participants with symptomatic obstructive hypertrophic cardiomyopathy received oral capsule of mavacamten during the treatment period from Day 1 to Week 30. The starting dose of mavacamten was 2.5 milligram (mg) once daily. At Week 6, Week 8, Week 14, and Week 20, mavacamten dose were titrated based on the individual, transthoracic echocardiogram (TTE) response (Valsalva left ventricular outflow tract [LVOT] gradient and left ventricular ejection fraction LVEF). The permissible doses during the study were 1, 2.5, 5, 10, or 15 mg. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial fibrillation | Cardiac disorders | 27.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial fibrillation | Cardiac disorders | 27.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Bristol-Myers Squibb Study Director | Bristol-Myers Squibb | Please email | Clinical.Trials@bms.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Oct 18, 2022 | Nov 5, 2024 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D002312 | Cardiomyopathy, Hypertrophic |
| ID | Term |
|---|---|
| D009202 | Cardiomyopathies |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D001020 | Aortic Stenosis, Subvalvular |
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| ID | Term |
|---|---|
| C000605992 | MYK-461 |
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| 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. | 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. | 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. | 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. | At baseline and week 30 |
| Himeji-Shi |
| Hyōgo |
| 672-8044 |
| Japan |
| Local Institution - 0016 | Kobe | Hyōgo | 650-0047 | Japan |
| Local Institution - 0017 | Tsukuba | Ibaraki | 305-0005 | Japan |
| Local Institution - 0023 | Kanazawa | Ishikawa-ken | 920-8641 | Japan |
| Local Institution - 0019 | Yokohama | Kanagawa | 227-8501 | Japan |
| Local Institution - 0015 | Nankoku-shi | Kochi | 783-8505 | Japan |
| Local Institution - 0028 | Tsu | Mie-ken | 514-8507 | Japan |
| Local Institution - 0027 | Sendai | Miyagi | 980-8574 | Japan |
| Local Institution - 0014 | Suita | Osaka | 564-8565 | Japan |
| Local Institution - 0011 | Suita-Shi | Osaka | 565-0871 | Japan |
| Local Institution - 0012 | Hamamatsu | Shizuoka | 431-3192 | Japan |
| Local Institution - 0010 | Bunkyo-Ku | Tokyo | 113-0033 | Japan |
| Local Institution - 0009 | Chuo-Ku | Tokyo | 104-0044 | Japan |
| Local Institution - 0003 | Fuchu-Shi | Tokyo | 1830003 | Japan |
| Local Institution - 0001 | Itabashi-Ku | Tokyo | 173-0003 | Japan |
| Local Institution - 0007 | Koto-Ku | Tokyo | 135-0061 | Japan |
| Local Institution - 0005 | Shinjuku-Ku | Tokyo | 160-8582 | Japan |
| Local Institution - 0013 | Shinjuku-ku | Tokyo | 162-8666 | Japan |
| Local Institution - 0018 | Osaka | 558-8558 | Japan |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Number | participants |
|
|
|
| Secondary | 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. | Intent to Treat Population. Only those participants with data available at the specified time points were analyzed. | Posted | Mean | Standard Deviation | Score on a Scale | At Baseline and Week 30 |
|
|
|
| Secondary | 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. | Intent To Treat Population. | Posted | Number | Percentage of participants | Baseline and at Week 30 |
|
|
|
| Secondary | 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. | Intent to Treat Population. Only those participants with data available at the specified time points were analyzed. | Posted | Median | Full Range | nanogram per liter (ng/L) | At baseline and week 30 |
|
|
|
| Secondary | 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. | Intent to Treat population. Only those participants with data available at the specified time points were analyzed. | Posted | Median | Full Range | ng/L | At baseline and week 30 |
|
|
|
| Secondary | 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. | Intent to Treat Population. Only those participants with data available at the specified time points were analyzed. | Posted | Median | Full Range | ng/L | At baseline and week 30 |
|
|
|
| 0 |
| 38 |
| 6 |
| 38 |
| 20 |
| 38 |
| Macular oedema | Eye disorders | 27.0 | Systematic Assessment |
|
| Cholangitis | Hepatobiliary disorders | 27.0 | Systematic Assessment |
|
| Cholecystitis acute | Hepatobiliary disorders | 27.0 | Systematic Assessment |
|
| COVID-19 | Infections and infestations | 27.0 | Systematic Assessment |
|
| Colonic abscess | Infections and infestations | 27.0 | Systematic Assessment |
|
| Rotator cuff syndrome | Musculoskeletal and connective tissue disorders | 27.0 | Systematic Assessment |
|
| Cerebral haemorrhage | Nervous system disorders | 27.0 | Systematic Assessment |
|
| Palpitations | Cardiac disorders | 27.0 | Systematic Assessment |
|
| Cataract | Eye disorders | 27.0 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | 27.0 | Systematic Assessment |
|
| Malaise | General disorders | 27.0 | Systematic Assessment |
|
| Pyrexia | General disorders | 27.0 | Systematic Assessment |
|
| Bronchitis | Infections and infestations | 27.0 | Systematic Assessment |
|
| COVID-19 | Infections and infestations | 27.0 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | 27.0 | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | 27.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | 27.0 | Systematic Assessment |
|
| Dermatitis contact | Skin and subcutaneous tissue disorders | 27.0 | Systematic Assessment |
|
| Hypertension | Vascular disorders | 27.0 | Systematic Assessment |
|
Bristol-Myers Squibb Co. agreements with investigators vary; constant is our right to embargo communications regarding trial results prior to public release for a period ≤60 days from submittal for review. We will not prohibit investigators from publishing but will prohibit the disclosure of previously undisclosed confidential information other than study results, and request postponement of single-center publications until after disclosure of the clinical trial's primary publication.
| D001024 |
| Aortic Valve Stenosis |
| D000082862 | Aortic Valve Disease |
| D006349 | Heart Valve Diseases |