Trial · NCT04349072
A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal Reduction Therapy
NCT04349072, Phase 3, Completed, Bristol-Myers Squibb; results first posted 2023-03-07
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal Reduction Therapy. Trials, record NCT04349072. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04349072
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal Reduction Therapy
- NCT number
- NCT04349072
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 112 participants, actual
- Lead sponsor
- Bristol-Myers Squibb
- Conditions
- HOCM, Hypertrophic Obstructive Cardiomyopathy
- Interventions
- Mavacamten, Placebo
- First posted
- 2020-04-16
- Last updated
- 2025-05-23
- Results first posted
- 2023-03-07
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Composite of Decision to Proceed With Septal Reduction Therapy (SRT) and SRT Guideline Eligible at Week 16 | 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. |
| Secondary | Number of Participants With at Least One Class Improvement From Baseline in New York Heart Association (NYHA) Class at Week 16 | Baseline and 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. |
| Secondary | Change From Baseline to Week 16 in Kansas City Cardiomyopathy Questionnaire 23-item Version, Clinical Summary Score (KCCQ-23, CSS) | Baseline and week 16 | 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. |
| Secondary | Change From Baseline to Week 16 in N-Terminal Pro-b-Type Natriuretic Peptide (NT-proBNP) | Baseline and week 16 | 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. |
| Secondary | Change From Baseline to Week 16 in Cardiac Troponin | Baseline and week 16 | 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. |
| Secondary | Change From Baseline to Week 16 in Post-Exercise Left Ventricular Outflow Tract (LVOT) Gradient | Baseline and week 16 | 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. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Composite of Decision to Proceed With Septal Reduction Therapy (SRT) and SRT Guideline Eligible at Week 16 | Week 16 | All randomized participants | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With at Least One Class Improvement From Baseline in New York Heart Association (NYHA) Class at Week 16 | Baseline and week 16 | All randomized participants | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline to Week 16 in Kansas City Cardiomyopathy Questionnaire 23-item Version, Clinical Summary Score (KCCQ-23, CSS) | Baseline and week 16 | All randomized participants with baseline and week 16 clinical summary scores | Score on a scale | MEAN |
| SECONDARY | Change From Baseline to Week 16 in N-Terminal Pro-b-Type Natriuretic Peptide (NT-proBNP) | Baseline and week 16 | All randomized participants with baseline and week 16 serum concentration data | ng/L | GEOMETRIC_MEAN |
| SECONDARY | Change From Baseline to Week 16 in Cardiac Troponin | Baseline and week 16 | All randomized participants with baseline and week 16 serum concentration data | ng/L | GEOMETRIC_MEAN |
| SECONDARY | Change From Baseline to Week 16 in Post-Exercise Left Ventricular Outflow Tract (LVOT) Gradient | Baseline and week 16 | All randomized participants with baseline and week 16 measurements | mmHg | MEAN |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Composite of Decision to Proceed With Septal Reduction Therapy (SRT) and SRT Guideline Eligible at Week 16 | Mavacamten | 10 | Participants | ||
| Composite of Decision to Proceed With Septal Reduction Therapy (SRT) and SRT Guideline Eligible at Week 16 | Placebo to Mavacamten | 43 | Participants | ||
| Number of Participants With at Least One Class Improvement From Baseline in New York Heart Association (NYHA) Class at Week 16 | Mavacamten | 35 | Participants | ||
| Number of Participants With at Least One Class Improvement From Baseline in New York Heart Association (NYHA) Class at Week 16 | Placebo to Mavacamten | 12 | Participants | ||
| Change From Baseline to Week 16 in Kansas City Cardiomyopathy Questionnaire 23-item Version, Clinical Summary Score (KCCQ-23, CSS) | Mavacamten | 10.4 | Standard Deviation 16.06 | Score on a scale | |
| Change From Baseline to Week 16 in Kansas City Cardiomyopathy Questionnaire 23-item Version, Clinical Summary Score (KCCQ-23, CSS) | Placebo to Mavacamten | 1.8 | Standard Deviation 12.01 | Score on a scale | |
| Change From Baseline to Week 16 in N-Terminal Pro-b-Type Natriuretic Peptide (NT-proBNP) | Mavacamten | 0.35 | Geometric Coefficient of Variation NA | ng/L | |
| Change From Baseline to Week 16 in N-Terminal Pro-b-Type Natriuretic Peptide (NT-proBNP) | Placebo to Mavacamten | 1.13 | Geometric Coefficient of Variation NA | ng/L | |
| Change From Baseline to Week 16 in Cardiac Troponin | Mavacamten | 0.50 | Geometric Coefficient of Variation NA | ng/L | |
| Change From Baseline to Week 16 in Cardiac Troponin | Placebo to Mavacamten | 1.03 | Geometric Coefficient of Variation NA | ng/L |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Randomization Period | STARTED | Mavacamten | 56 | |
| Randomization Period | STARTED | Placebo to Mavacamten | 56 | |
| Randomization Period | COMPLETED | Mavacamten | 56 | |
| Randomization Period | COMPLETED | Placebo to Mavacamten | 55 | |
| Randomization Period | NOT COMPLETED | Mavacamten | 0 | |
| Randomization Period | NOT COMPLETED | Placebo to Mavacamten | 1 | |
| Double Blind Treatment Period | STARTED | Mavacamten | 56 | |
| Double Blind Treatment Period | STARTED | Placebo to Mavacamten | 55 | |
| Double Blind Treatment Period | COMPLETED | Mavacamten | 52 | |
| Double Blind Treatment Period | COMPLETED | Placebo to Mavacamten | 45 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events142
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Atrial fibrillation | Cardiac disorders | Mavacamten | 13 | 108 | |
| other | Atrial fibrillation | Cardiac disorders | Placebo | 0 | 55 | |
| other | Palpitations | Cardiac disorders | Mavacamten | 14 | 108 | |
| other | Palpitations | Cardiac disorders | Placebo | 2 | 55 | |
| other | Ventricular tachycardia | Cardiac disorders | Mavacamten | 1 | 108 | |
| other | Ventricular tachycardia | Cardiac disorders | Placebo | 5 | 55 | |
| other | Constipation | Gastrointestinal disorders | Mavacamten | 9 | 108 | |
| other | Constipation | Gastrointestinal disorders | Placebo | 2 | 55 | |
| other | Diarrhoea | Gastrointestinal disorders | Mavacamten | 7 | 108 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 2 | 55 |
1 to 10 of 142Source ClinicalTrials.gov
See all 142 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Mavacamten | 1 | 108 |
| Total, all-cause mortality | Placebo | 0 | 56 |
| Total, other adverse events | Mavacamten | 96 | 108 |
| Total, other adverse events | Placebo | 20 | 55 |
| Total, serious adverse events | Mavacamten | 21 | 108 |
| Total, serious adverse events | Placebo | 1 | 55 |
Source ClinicalTrials.gov
Papers10
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Mavacamten vs Placebo to Mavacamten: Number of Participants With at Least One Class Improvement From Baseline in New York Heart Association (NYHA) Class at Week 16 | Trial comparisons |
| Mavacamten vs Placebo to Mavacamten: Composite of Decision to Proceed With Septal Reduction Therapy (SRT) and SRT Guideline Eligible at Week 16 | Trial comparisons |
| Mavacamten vs Placebo: deaths from any cause | Trial comparisons |
| Mavacamten vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Source
- Release
- trials-2026-11-12-0.4.0
- Release manifest SHA-256
- 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
- Registry capture
- 2026-11-12
- Results copy, AACT
- 2026-03-23
- NIH clinical studies file
- 2026-11-13
Source ClinicalTrials.gov
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNone stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04349072
GET https://conductscience.org/api/trials/v1/NCT04349072/events?page=2