A Study to Evaluate Mavacamten in Adults With Symptomatic... | NCT04349072

Status Module

Label	Type	Description	Intervention Names
Drug: Mavacamten	Experimental	Mavacamten Capsules Other names: MYK-461	Drug: Mavacamten
Drug: Placebo	Placebo Comparator	Matching Placebo Capsules	Drug: Placebo

Name	Type	Description	Arm Group Labels	Other Names
Mavacamten	Drug	Mavacamten Capsules Other names: MYK-461	Drug: Mavacamten

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Composite of Decision to Proceed With Septal Reduction Therapy (SRT) and SRT Guideline Eligible at Week 16	Participants who decided to proceed with SRT or were eligible for SRT at week 16. Participants with missing assessments were classified as meeting the primary endpoint (did not improve). SRT eligibility using the New York Heart Association Functional Class (NYHA) and left ventricular outflow tract (LVOT) assessments per the 2011 ACCF/AHA guideline clinical and hemodynamic criterion are below: NYHA Class III or IV/ NYHA Class II with exertion-induced syncope/near syncope, AND Dynamic LVOT gradient at rest or with provocation >= 50 mmHg. NYHA Class II at week 16, the following rules will be applied: NYHA Class II with history of exertional syncope/ syncope at baseline and at W16 is still NYHA Class II, they remain SRT eligible IF their maximal LVOT gradient is ≥ 50mmHg NYHA Class III/IV at baseline and at W16 has improved to Class II, they are no longer SRT eligible UNLESS they have AE of exertional syncope or pre-syncope during the 16 weeks.	Week 16

Secondary Outcomes

Measure	Description	Time Frame
Number of Participants With at Least One Class Improvement From Baseline in New York Heart Association (NYHA) Class at Week 16	The NYHA functional classification of heart failure assigns participants to 1 of 4 categories based on the participants symptoms. Baseline values are defined generally as the last available value before the first administration of study drug of analysis interest. Participants with missing NYHA class assessments are treated as no improvement. Class 1: No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea (shortness of breath). Class 2: Slight limitation of physical activity. Comfortable at rest. Ordinary physical activity results in fatigue, palpitation, dyspnea (shortness of breath). Class 3: Marked limitation of physical activity. Comfortable at rest. Less-than ordinary-activity causes fatigue, palpitation, or dyspnea. Class 4: Unable to carry on any physical activity without discomfort. Symptoms of heart failure at rest. If any physical activity is undertaken, discomfort increases.

Eligibility Module

Name	Affiliation	Role
Bristol-Myers Squibb	Bristol-Myers Squibb	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Local Institution - 0009		Los Angeles	California	90027	United States
Local Institution - 0011

PubMed Identifier	Type	Citation	Retractions
39556124	Derived	Desai MY, Wolski K, Owens A, Geske JB, Saberi S, Wang A, Sherrid M, Cremer PC, Lakdawala NK, Tower-Rader A, Fermin D, Naidu SS, Smedira NG, Schaff H, Gong Z, Mudarris L, Lampl K, Sehnert AJ, Nissen SE; VALOR-HCM Investigators. Mavacamten in Patients With Hypertrophic Cardiomyopathy Referred for Septal Reduction: Week 128 Results From VALOR-HCM. Circulation. 2025 May 13;151(19):1378-1390. doi: 10.1161/CIRCULATIONAHA.124.072445. Epub 2024 Nov 18.
39254622

Label	URL
BMS Clinical Trial Information	View source

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Mavacamten	Participants randomized to the mavacamten group started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 4 and up-titration at Weeks 8 and 12 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

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	Jun 30, 2022

Arm/Group Information

ID	Title	Description
BG000	Mavacamten	Participants randomized to the mavacamten group started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 4 and up-titration at Weeks 8 and 12 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).
BG001	Placebo to Mavacamten	One placebo-to-match mavacamten capsule once daily for 16 weeks and then switch to mavacamten. After Week 16 through Week 32, participants started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 20 and up-titration at Weeks 24 and 28 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).
BG002	Total	Total of all reporting groups

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

Composite of Decision to Proceed With Septal Reduction Therapy (SRT) and SRT Guideline Eligible at Week 16

Participants who decided to proceed with SRT or were eligible for SRT at week 16. Participants with missing assessments were classified as meeting the primary endpoint (did not improve).

SRT eligibility using the New York Heart Association Functional Class (NYHA) and left ventricular outflow tract (LVOT) assessments per the 2011 ACCF/AHA guideline clinical and hemodynamic criterion are below:

NYHA Class III or IV/ NYHA Class II with exertion-induced syncope/near syncope, AND
Dynamic LVOT gradient at rest or with provocation >= 50 mmHg.

NYHA Class II at week 16, the following rules will be applied:

NYHA Class II with history of exertional syncope/ syncope at baseline and at W16 is still NYHA Class II, they remain SRT eligible IF their maximal LVOT gradient is ≥ 50mmHg
NYHA Class III/IV at baseline and at W16 has improved to Class II, they are no longer SRT eligible UNLESS they have AE of exertional syncope or pre-syncope during the 16 weeks.

All randomized participants

Posted

Count of Participants

Participants

Week 16

ID	Title	Description
OG000	Mavacamten	Participants randomized to the mavacamten group started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 4 and up-titration at Weeks 8 and 12 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).
OG001	Placebo to Mavacamten	One placebo-to-match mavacamten capsule once daily for 16 weeks and then switch to mavacamten. After Week 16 through Week 32, participants started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 20 and up-titration at Weeks 24 and 28 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).

Units	Counts
Participants	OG00056 OG00156

Title

Denominators

Categories

Title	Measurements
	OG00010 OG00143

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG001		Cochran-Mantel-Haenszel		<0.0001		Proportion Difference	58.93			2-Sided	95	43.989	73.868					Superiority

Secondary

Number of Participants With at Least One Class Improvement From Baseline in New York Heart Association (NYHA) Class at Week 16

The NYHA functional classification of heart failure assigns participants to 1 of 4 categories based on the participants symptoms. Baseline values are defined generally as the last available value before the first administration of study drug of analysis interest. Participants with missing NYHA class assessments are treated as no improvement.

Class 1: No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea (shortness of breath).

Class 2: Slight limitation of physical activity. Comfortable at rest. Ordinary physical activity results in fatigue, palpitation, dyspnea (shortness of breath).

Class 3: Marked limitation of physical activity. Comfortable at rest. Less-than ordinary-activity causes fatigue, palpitation, or dyspnea. Class 4: Unable to carry on any physical activity without discomfort. Symptoms of heart failure at rest. If any physical activity is undertaken, discomfort increases.

All randomized participants

Posted

Count of Participants

Participants

Baseline and week 16

ID	Title	Description
OG000	Mavacamten	Participants randomized to the mavacamten group started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 4 and up-titration at Weeks 8 and 12 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).

Secondary

Change From Baseline to Week 16 in Kansas City Cardiomyopathy Questionnaire 23-item Version, Clinical Summary Score (KCCQ-23, CSS)

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.

All randomized participants with baseline and week 16 clinical summary scores

Posted

Mean

Standard Deviation

Score on a scale

Baseline and week 16

ID	Title	Description
OG000	Mavacamten	Participants randomized to the mavacamten group started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 4 and up-titration at Weeks 8 and 12 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).
OG001	Placebo to Mavacamten

Secondary

Change From Baseline to Week 16 in N-Terminal Pro-b-Type Natriuretic Peptide (NT-proBNP)

A geometric mean ratio was used to assess the change from baseline to Week 16 in N-Terminal Pro-b-Type Natriuretic Peptide (NT-proBNP). Baseline values are defined as the last non-missing value prior to the first dose of study drug unless specified otherwise.

All randomized participants with baseline and week 16 serum concentration data

Posted

Geometric Mean

Geometric Coefficient of Variation

ng/L

Baseline and week 16

ID	Title	Description
OG000	Mavacamten	Participants randomized to the mavacamten group started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 4 and up-titration at Weeks 8 and 12 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).
OG001	Placebo to Mavacamten	One placebo-to-match mavacamten capsule once daily for 16 weeks and then switch to mavacamten. After Week 16 through Week 32, participants started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 20 and up-titration at Weeks 24 and 28 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).

Secondary

Change From Baseline to Week 16 in Cardiac Troponin

A geometric mean ratio was used to assess the change from baseline to week 16 in cardiac troponin. Baseline values are defined as the last non-missing value prior to the first dose of study drug unless specified otherwise.

All randomized participants with baseline and week 16 serum concentration data

Posted

Geometric Mean

Geometric Coefficient of Variation

ng/L

Baseline and week 16

ID	Title	Description
OG000	Mavacamten	Participants randomized to the mavacamten group started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 4 and up-titration at Weeks 8 and 12 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).
OG001	Placebo to Mavacamten	One placebo-to-match mavacamten capsule once daily for 16 weeks and then switch to mavacamten. After Week 16 through Week 32, participants started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 20 and up-titration at Weeks 24 and 28 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).

Secondary

Change From Baseline to Week 16 in Post-Exercise Left Ventricular Outflow Tract (LVOT) Gradient

Change from baseline to week 16 in post-exercise left ventricular outflow tract (LVOT) gradient. Baseline values are defined as the last non-missing value prior to the first dose of study drug unless specified otherwise.

All randomized participants with baseline and week 16 measurements

Posted

Mean

Standard Deviation

mmHg

Baseline and week 16

ID	Title	Description
OG000	Mavacamten	Participants randomized to the mavacamten group started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 4 and up-titration at Weeks 8 and 12 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).
OG001	Placebo to Mavacamten	One placebo-to-match mavacamten capsule once daily for 16 weeks and then switch to mavacamten. After Week 16 through Week 32, participants started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration at Week 20 and up-titration at Weeks 24 and 28 (based on the LVEF and VLVOT gradient measured by TTE). Participants could be up- titrated at any scheduled visit after Week 32. Participants went through 16 weeks of placebo-controlled treatment (Day 1 to Week 16), 16 weeks of active-controlled treatment (Weeks 16 to 32), and 96 weeks of LTE mavacamten (Weeks 32 to 128).

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Acute left ventricular failure	Cardiac disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Acute myocardial infarction	Cardiac disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Arrhythmia	Cardiac disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Atrial fibrillation	Cardiac disorders	MedDRA 27.0	Systematic Assessment	EG0004 affected108 at risk EG0010 affected55 at risk
Bradycardia	Cardiac disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Cardiac failure acute	Cardiac disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Cardiac failure congestive	Cardiac disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Cardiogenic shock	Cardiac disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Ventricular arrhythmia	Cardiac disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Gastrooesophageal reflux disease	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Haematochezia	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Large intestine perforation	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Pneumatosis intestinalis	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Asthenia	General disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Sudden cardiac death	General disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Cholecystitis	Hepatobiliary disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Ischaemic hepatitis	Hepatobiliary disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
COVID-19	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0002 affected108 at risk EG0010 affected55 at risk
Clostridium difficile infection	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Pneumonia	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Alcohol poisoning	Injury, poisoning and procedural complications	MedDRA 27.0	Systematic Assessment	EG0000 affected108 at risk EG0011 affected55 at risk
Fall	Injury, poisoning and procedural complications	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Ejection fraction decreased	Investigations	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Hypokalaemia	Metabolism and nutrition disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Muscular weakness	Musculoskeletal and connective tissue disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Hormone receptor positive breast cancer	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Dyskinesia	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Embolic stroke	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Syncope	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG0002 affected108 at risk EG0010 affected55 at risk
Device inappropriate shock delivery	Product Issues	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Device lead damage	Product Issues	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Mental status changes	Psychiatric disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Acute kidney injury	Renal and urinary disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Nephrolithiasis	Renal and urinary disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Ureterolithiasis	Renal and urinary disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Acute respiratory failure	Respiratory, thoracic and mediastinal disorders	MedDRA 27.0	Systematic Assessment	EG0002 affected108 at risk EG0010 affected55 at risk
Hypoxia	Respiratory, thoracic and mediastinal disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Pulmonary embolism	Respiratory, thoracic and mediastinal disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Pulmonary oedema	Respiratory, thoracic and mediastinal disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Hypovolaemic shock	Vascular disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Peripheral venous disease	Vascular disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk
Shock haemorrhagic	Vascular disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0010 affected55 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Atrial fibrillation	Cardiac disorders	MedDRA 27.0	Systematic Assessment	EG00013 affected108 at risk EG0010 affected55 at risk
Palpitations	Cardiac disorders	MedDRA 27.0	Systematic Assessment	EG00014 affected108 at risk EG0012 affected55 at risk
Ventricular tachycardia	Cardiac disorders	MedDRA 27.0	Systematic Assessment	EG0001 affected108 at risk EG0015 affected55 at risk
Constipation	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment	EG0009 affected108 at risk EG0012 affected55 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment	EG0007 affected108 at risk EG0012 affected55 at risk
Nausea	Gastrointestinal disorders	MedDRA 27.0	Systematic Assessment	EG0009 affected108 at risk EG0011 affected55 at risk
Chest discomfort	General disorders	MedDRA 27.0	Systematic Assessment	EG0006 affected108 at risk EG0012 affected55 at risk
Chest pain	General disorders	MedDRA 27.0	Systematic Assessment	EG0007 affected108 at risk EG0011 affected55 at risk
Fatigue	General disorders	MedDRA 27.0	Systematic Assessment	EG00018 affected108 at risk EG0012 affected55 at risk
Bronchitis	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG0007 affected108 at risk EG0011 affected55 at risk
COVID-19	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG00039 affected108 at risk EG0012 affected55 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG00015 affected108 at risk EG0010 affected55 at risk
Urinary tract infection	Infections and infestations	MedDRA 27.0	Systematic Assessment	EG00015 affected108 at risk EG0011 affected55 at risk
Ejection fraction decreased	Investigations	MedDRA 27.0	Systematic Assessment	EG00012 affected108 at risk EG0010 affected55 at risk
SARS-CoV-2 test positive	Investigations	MedDRA 27.0	Systematic Assessment	EG0009 affected108 at risk EG0011 affected55 at risk
Gout	Metabolism and nutrition disorders	MedDRA 27.0	Systematic Assessment	EG0006 affected108 at risk EG0010 affected55 at risk
Hyperlipidaemia	Metabolism and nutrition disorders	MedDRA 27.0	Systematic Assessment	EG0006 affected108 at risk EG0010 affected55 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 27.0	Systematic Assessment	EG00013 affected108 at risk EG0010 affected55 at risk
Muscular weakness	Musculoskeletal and connective tissue disorders	MedDRA 27.0	Systematic Assessment	EG0006 affected108 at risk EG0011 affected55 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA 27.0	Systematic Assessment	EG0006 affected108 at risk EG0011 affected55 at risk
Dizziness	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG00016 affected108 at risk EG0013 affected55 at risk
Headache	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG00011 affected108 at risk EG0015 affected55 at risk
Syncope	Nervous system disorders	MedDRA 27.0	Systematic Assessment	EG0006 affected108 at risk EG0010 affected55 at risk
Anxiety	Psychiatric disorders	MedDRA 27.0	Systematic Assessment	EG0008 affected108 at risk EG0010 affected55 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 27.0	Systematic Assessment	EG0007 affected108 at risk EG0010 affected55 at risk
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA 27.0	Systematic Assessment	EG00011 affected108 at risk EG0013 affected55 at risk
Dyspnoea exertional	Respiratory, thoracic and mediastinal disorders	MedDRA 27.0	Systematic Assessment	EG00010 affected108 at risk EG0012 affected55 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA 27.0	Systematic Assessment	EG0007 affected108 at risk EG0010 affected55 at risk
Hypertension	Vascular disorders	MedDRA 27.0	Systematic Assessment	EG00010 affected108 at risk EG0012 affected55 at risk

ID	Term
D009202	Cardiomyopathies
D006331	Heart Diseases
D002318	Cardiovascular Diseases
D001020	Aortic Stenosis, Subvalvular
D001024	Aortic Valve Stenosis
D000082862	Aortic Valve Disease
D006349	Heart Valve Diseases

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 started on 5 mg once daily and were titrated to receive 2.5, 5, 10, or 15 mg capsule orally once daily based on possible down-titration and up-titration (based on the LVEF and VLVOT gradient measured by TTE).	1	108	21	108	96	108
EG001	Placebo	One placebo-to-match mavacamten capsule once daily for 16 weeks.	0	56	1	55	20	55