A Phase 3 interventional study of mavacamten and Placebo in Obstructive Hypertrophic Cardiomyopathy, sponsored by MyoKardia, Inc.. Completed at 71 sites in 13 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-10-04.
Sponsored by MyoKardia, Inc. (part of Bristol-Myers Squibb) · Phase 3, Interventional, and Treatment
Among 21 studies testing MYK-461 in Cardiomyopathy, Hypertrophic — 3 open to participants now, 7 under way, 1 of them phase 3. MYK-461 · Cardiomyopathy, Hypertrophic.
Among the 21 studies testing MYK-461 in Cardiomyopathy, Hypertrophic, this one is the 3rd largest by enrollment, is one of 4 that started in 2018, is one of 6 in phase 3, and was the 5th registered.
363 studies on the registry are indexed under Cardiomyopathy, Hypertrophic; 116 are open to participants now.
Browse Cardiomyopathy, Hypertrophic studies →3 of the 21 studies testing MYK-461 in Cardiomyopathy, Hypertrophic are open now, in 2 countries. Recruiting first, the newest first:
University of Manchester · Sites in United Kingdom
Ages 18+
Second Affiliated Hospital, Zhejiang University, School of Medicine · Sites in China
Ages 18–75
Second Affiliated Hospital of Nanchang University
Ages 18+
By country, each count listing its studies:
MYK-461: first FDA approval 2022 (CAMZYOS), first EMA approval 2023. What it is approved for, and how studies give it, is on its page →
Of the 81 Cardiomyopathy, Hypertrophic studies recruiting now, 55 (68%) set no upper age limit and 8 (10%) stop at 75 or younger; 21 take children under 18; 13 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
21 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 5 · Phase 2 4 · Phase 3 7 · Phase 4 4
First in 2014; this study’s year is marked. 4 started in 2018.
This study's enrollment of 251 is above the median of 60 across 180 interventional studies indexed under Cardiomyopathy, Hypertrophic.
Median enrollment 75 across the 21 interventional studies. Of the 14 that completed or were terminated, 9 (64%) have results posted.
This tab as a report to print or save as PDF → With an account.
17 of 21 have an industry lead sponsor. The 7 most frequent:
This study is registered under MyoKardia, Inc., one of the names counted as Bristol-Myers Squibb. Under that name alone:
MyoKardia, Inc. is the lead sponsor of 7 studies on the registry; none are open to participants now.
31 studies in Cardiomyopathy, Hypertrophic by Bristol-Myers Squibb: 9 open now, 14 under way, 1 of them phase 3. Of 17 finished, 8 (47%) have results posted.
Among the 31 studies in Cardiomyopathy, Hypertrophic by Bristol-Myers Squibb, this one is the 3rd largest by enrollment of the 16 interventional ones that give a count, is one of 4 that started in 2018, is one of 5 in phase 3, and was the 5th registered.
Bristol-Myers Squibb’s Cardiomyopathy, Hypertrophic studies by the year they started, since 2014.
None of the studies under way reaches primary completion within a year.
Among the 21 studies testing MYK-461 in Cardiomyopathy, Hypertrophic, from their registry updates:
Among Bristol-Myers Squibb’s 31 in Cardiomyopathy, Hypertrophic:
This tab as a report to print or save as PDF → With an account.
This is a multicenter, international, double-blind study of the administration of mavacamten in participants with symptomatic obstructive HCM (oHCM). Approximately 220 participants will be randomized to receive placebo or mavacamten.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: mavacamten
Drug: Placebo
mavacamten capsules
Also known as: MYK-461
placebo oral capsule
Percentage of Participants Achieving A Clinical Response
A positive clinical response (value="YES") is defined as having achieved either an improvement of at least 1.5 mL/kg/min in peak oxygen consumption (pVO2) as determined by cardiopulmonary exercise testing (CPET) and a reduction of one or more class in New York Heart Association (NYHA) functional classification (e.g.I, II, III, or IV) -OR- an improvement of 3.0 mL/kg/min or more in pVO2 with no worsening in NYHA Functional Class.
Time frame: 30 weeks
Changes From Baseline to Week 30 in Post Exercise in LVOT Peak Gradient.
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 Cardiovascular Imaging Core Laboratory (CICL, Boston MA). Change from baseline was determined as per the study statistical analysis plan and compared between treatment arms.
Time frame: 30 weeks
Change From Baseline to Week 30 in pVO2 as Assessed by CPET
Cardiopulmonary exercise testing (CPET) was performed at baseline and week 30 following a study-specified protocol and peak oxygen consumption (pVO2) was determined by the Cardiovascular Metabolic Disease Research Institute (CMDRI, Palo Alto, CA). Change from baseline was determined as per the study statistical analysis plan and compared between treatment arms.
Time frame: 30 weeks
Proportion of Participants With at Least 1 Class Improvement in NYHA Functional Class From Baseline to Week 30
New York Heart Association (NYHA) functional classification was determined by the principal investigator at baseline and at specified timepoints in the study. At baseline, all subjects were NYHA Class II or III. For the secondary outcome, NYHA class at Week 30 was compared to baseline and the proportion of subjects with an improvement of at least one class was determined, and the difference between treatment groups was analyzed. The proportion was also multiplied by 100 to provide the result as a percent.
Time frame: 30 weeks
Change From Baseline to Week 30 in Participant-reported Health-related Quality of Life as Assessed by the KCCQ Score
The Kansas City Cardiomyopathy Questionnaire (KCCQ) is a patient reported outcome instrument with minimum score = 0 and maximum score = 100 where higher score indicates better health status. There are no units to the score. The instrument utilizes a recall period of 2 weeks over which patients describe the frequency and severity of their symptoms, their physical and social limitations, and how they perceive their heart failure symptoms to affect their quality of life. The KCCQ clinical summary (KCCQ-CS) score, a prespecified secondary outcome of EXPLORER-HCM, combines the physical limitation and total symptom scores.
Time frame: 30 weeks
Change From Baseline to Week 30 in Participant-reported Severity of HCM Symptoms as Assessed by the HCMSQ Score
The Hypertrophic Cardiomyopathy Symptom Questionnaire (HCMSQ) is a patient reported outcome instrument that is a daily self-administered 11-item questionnaire. The HCMSQ assesses the core symptoms of HCM (tiredness/fatigue, heart palpitations, chest pain, dizziness, and shortness of breath). The Shortness of Breath domain score, a pre-specified secondary outcome of EXPLORER-HCM, assesses the frequency and severity of shortness of breath. The minimum score = 0 and maximum score = 18 where lower score indicates better health status. There are no units to the score.
Time frame: 30 weeks
Subjects who met all of the inclusion criteria and none of the exclusion criteria were enrolled into the study and were randomized 1:1 to receive mavacamten (2.5, 5, 10, or 15 mg capsule) or placebo once daily for 30 weeks, followed by an 8 week post-treatment period. In total, 429 participants were assessed for eligibility and 251 were enrolled from 68 sites in the United States and EMEA.
| Milestone | Mavacamten (MYK-461) | Placebo |
|---|---|---|
| Started | 123 | 128 |
| Completed | 119 | 125 |
| Not completed | 4 | 3 |
| Withdrew: Death | 0 | 1 |
| Withdrew: Did not complete week 30 assessments due to scheduling conflict | 1 | 0 |
| Withdrew: Adverse event | 2 | 0 |
| Withdrew: Withdrawal by subject | 1 | 1 |
| Withdrew: Week 30 not completed due to circumstances surrounding the covid-19 pandemic | 0 | 1 |
A positive clinical response (value="YES") is defined as having achieved either an improvement of at least 1.5 mL/kg/min in peak oxygen consumption (pVO2) as determined by cardiopulmonary exercise testing (CPET) and a reduction of one or more class in New York Heart Association (NYHA) functional classification (e.g.I, II, III, or IV) -OR- an improvement of 3.0 mL/kg/min or more in pVO2 with no worsening in NYHA Functional Class.
| Participants | Mavacamten (MYK-461) | Placebo |
|---|---|---|
| Percentage of Participants Achieving A Clinical Response | 45 | 22 |
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 Cardiovascular Imaging Core Laboratory (CICL, Boston MA). Change from baseline was determined as per the study statistical analysis plan and compared between treatment arms.
| mmHg | Mavacamten (MYK-461) | Placebo |
|---|---|---|
| Changes From Baseline to Week 30 in Post Exercise in LVOT Peak Gradient. | -47 ± 40.3 | -10 ± 29.6 |
Cardiopulmonary exercise testing (CPET) was performed at baseline and week 30 following a study-specified protocol and peak oxygen consumption (pVO2) was determined by the Cardiovascular Metabolic Disease Research Institute (CMDRI, Palo Alto, CA). Change from baseline was determined as per the study statistical analysis plan and compared between treatment arms.
| mL/kg/min | Mavacamten (MYK-461) | Placebo |
|---|---|---|
| Change From Baseline to Week 30 in pVO2 as Assessed by CPET | 1.4 ± 3.12 | -0.05 ± 3.02 |
New York Heart Association (NYHA) functional classification was determined by the principal investigator at baseline and at specified timepoints in the study. At baseline, all subjects were NYHA Class II or III. For the secondary outcome, NYHA class at Week 30 was compared to baseline and the proportion of subjects with an improvement of at least one class was determined, and the difference between treatment groups was analyzed. The proportion was also multiplied by 100 to provide the result as a percent.
| Participants | Mavacamten (MYK-461) | Placebo |
|---|---|---|
| Proportion of Participants With at Least 1 Class Improvement in NYHA Functional Class From Baseline to Week 30 | 80 | 40 |
The Kansas City Cardiomyopathy Questionnaire (KCCQ) is a patient reported outcome instrument with minimum score = 0 and maximum score = 100 where higher score indicates better health status. There are no units to the score. The instrument utilizes a recall period of 2 weeks over which patients describe the frequency and severity of their symptoms, their physical and social limitations, and how they perceive their heart failure symptoms to affect their quality of life. The KCCQ clinical summary (KCCQ-CS) score, a prespecified secondary outcome of EXPLORER-HCM, combines the physical limitation and total symptom scores.
| Scores on Scale | Mavacamten (MYK-461) | Placebo |
|---|---|---|
| Change From Baseline to Week 30 in Participant-reported Health-related Quality of Life as Assessed by the KCCQ Score | 13.6 ± 14.42 | 4.2 ± 13.68 |
The Hypertrophic Cardiomyopathy Symptom Questionnaire (HCMSQ) is a patient reported outcome instrument that is a daily self-administered 11-item questionnaire. The HCMSQ assesses the core symptoms of HCM (tiredness/fatigue, heart palpitations, chest pain, dizziness, and shortness of breath). The Shortness of Breath domain score, a pre-specified secondary outcome of EXPLORER-HCM, assesses the frequency and severity of shortness of breath. The minimum score = 0 and maximum score = 18 where lower score indicates better health status. There are no units to the score.
| Scores on Scale | Mavacamten (MYK-461) | Placebo |
|---|---|---|
| Change From Baseline to Week 30 in Participant-reported Severity of HCM Symptoms as Assessed by the HCMSQ Score | -2.8 ± 2.68 | -0.9 ± 2.41 |
Collected over Treatment-Emergent Adverse Events (TEAEs) were summarized for the on-treatment period (Day 1 to Week 30) and for the treatment-emergent period (Day 1 to Week 38).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Mavacamten (MYK-461) | 0/123 (0%) | 14/123 (11.4%) | 108/123 (87.8%) |
| Placebo | 1/128 (0.8%) | 12/128 (9.4%) | 104/128 (81.3%) |
| Event | Mavacamten (MYK-461) | Placebo |
|---|---|---|
| Atrial FibrillationCardiac disorders | 3/123 | 5/128 |
| SyncopeNervous system disorders | 3/123 | 1/128 |
| Stress cardiomyopathyCardiac disorders | 2/123 | 0/128 |
| Urinary Tract InfectionInfections and infestations | 0/123 | 2/128 |
| Cardiac FailureCardiac disorders | 1/123 | 0/128 |
| Cardiogenic shockCardiac disorders | 1/123 | 0/128 |
| Pericardial effusionCardiac disorders | 1/123 | 0/128 |
| Systolic dysfunctionCardiac disorders | 1/123 | 0/128 |
| Atrial septal defectCongenital, familial and genetic disorders | 1/123 | 0/128 |
| Abdominal PainGastrointestinal disorders | 1/123 | 0/128 |
| Event | Mavacamten (MYK-461) | Placebo |
|---|---|---|
| DizzinessNervous system disorders | 26/123 | 17/128 |
| NasopharyngitisInfections and infestations | 15/123 | 19/128 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 18/123 | 13/128 |
| HeadacheNervous system disorders | 15/123 | 10/128 |
| Atrial FibrillationCardiac disorders | 10/123 | 10/128 |
| CoughRespiratory, thoracic and mediastinal disorders | 10/123 | 4/128 |
| Back painMusculoskeletal and connective tissue disorders | 10/123 | 8/128 |
| Upper respiratory tract infectionInfections and infestations | 10/123 | 6/128 |
| PalpitationsCardiac disorders | 7/123 | 10/128 |
| SyncopeNervous system disorders | 7/123 | 2/128 |
| Age, Categorical(Participants) | Mavacamten (MYK-461) | Placebo | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 78 | 88 | 166 |
| >=65 years | 45 | 40 | 85 |
| Age, Continuous(years) | Mavacamten (MYK-461) | Placebo | Total |
|---|---|---|---|
| Median | 60 (26 to 82) | 60 (18 to 81) | 60 (18 to 82) |
| Sex: Female, Male(Participants) | Mavacamten (MYK-461) | Placebo | Total |
|---|---|---|---|
| Female | 57 | 45 | 102 |
| Male | 66 | 83 | 149 |
| Ethnicity (NIH/OMB)(Participants) | Mavacamten (MYK-461) | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 8 | 4 | 12 |
| Not Hispanic or Latino | 114 | 119 | 233 |
| Unknown or Not Reported | 1 | 5 | 6 |
| Race (NIH/OMB)(Participants) | Mavacamten (MYK-461) | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 1 | 1 |
| Asian | 4 | 2 | 6 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 1 | 5 | 6 |
| White | 115 | 114 | 229 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 3 | 6 | 9 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from May 2018 to primary completion in Mar 2020, 21 months.
Results were first posted in Oct 2021, 1 year 6 months after primary completion.
Of 15 phase 3 interventional studies under Cardiomyopathy, Hypertrophic, half reached primary completion within 21 months of starting (the median), counting those still running; a quarter within 19 and three quarters within 36. This study's 21 months is close to that.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in May 2020. 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.
Studies of the drug tested here: MYK-461
Cardiomyopathy, Hypertrophic →