Cardiomyopathy, Hypertrophic ObstructivePhase 3HORIZON-HCMNCT05414175
A Study of Mavacamten in Obstructive Hypertrophic Cardiomyopathy
A Phase 3 study testing Mavacamten in people with Cardiomyopathy, Hypertrophic Obstructive, run by Bristol-Myers Squibb at 20 sites in Japan.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cardiomyopathy, Hypertrophic Obstructive
- What is being tested?
- Mavacamten
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 20 sitesJapan
- Study dates
- 19 Aug 2022to 11 Dec 2025
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Age 18 and greater, body weight ≥ 35kg
- Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)
- Diagnosed with obstructive hypertrophic cardiomyopathy consistent with current American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Japanese Circulation Society guidelines
- Has documented left ventricular ejection fraction (LVEF) ≥60% NYHA Class II or III
Who cannot join
- Known infiltrative or storage disorder causing cardiac hypertrophy that mimics oHCM, such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy
- History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to Screening
- History of resuscitated sudden cardiac arrest (at any time) or known history of appropriate implantable cardioverter defibrillator (ICD) discharge for life-threatening ventricular arrhythmia within 6 months prior to Screening
- Paroxysmal atrial fibrillation with atrial fibrillation present at the time of Screening.
- Persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate controlled within 6 months prior to Screening
- Treatment (within 14 days prior to Screening) or planned treatment during the study with cibenzoline, disopyramide or ranolazine
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- Treatment (within 14 days prior to Screening) or planned treatment during the study with a combination of beta blockers and verapamil or a combination of beta blockers and diltiazem
- Has been successfully treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening or plans to have either of these treatments during the study
- ICD placement within 2 months prior to Screening or planned ICD placement during the study
- Has a history or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to participant safety or interfere with the study evaluation procedures, or completion
- Prior treatment with cardiotoxic agents such as doxorubicin or similar
- Other protocol-defined inclusion/exclusion criteria apply
Trial locations and contacts
20 sites in 1 country.
20 sites in 1 country
Japan20 sites
Local Institution - 0010
Bunkyo-Ku, Tokyo, 113-0033
Local Institution - 0009
Chuo-Ku, Tokyo, 104-0044
Local Institution - 0003
Fuchu-Shi, Tokyo, 1830003
Local Institution - 0012
Hamamatsu, Shizuoka, 431-3192
Local Institution - 0026
Himeji-Shi, Hyōgo, 672-8044
Local Institution - 0001
Itabashi-Ku, Tokyo, 173-0003
Local Institution - 0023
Kanazawa, Ishikawa-ken, 920-8641
Local Institution - 0016
Kobe, Hyōgo, 650-0047
Local Institution - 0007
Koto-Ku, Tokyo, 135-0061
Local Institution - 0015
Nankoku-shi, Kochi, 783-8505
Local Institution - 0018
Osaka, 558-8558
Local Institution - 0027
Sendai, Miyagi, 980-8574
Local Institution - 0013
Shinjuku-ku, Tokyo, 162-8666
Local Institution - 0005
Shinjuku-Ku, Tokyo, 160-8582
Local Institution - 0014
Suita, Osaka, 564-8565
Local Institution - 0011
Suita-Shi, Osaka, 565-0871
Local Institution - 0028
Tsu, Mie-ken, 514-8507
Local Institution - 0017
Tsukuba, Ibaraki, 305-0005
Local Institution - 0020
Uwajima, Ehime, 798-8510
Local Institution - 0019
Yokohama, Kanagawa, 227-8501
About this study
Official title: A Phase 3, Open-label, Single Arm, Clinical Study to Evaluate Efficacy, Safety and Tolerability of Mavacamten in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy
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).
- Conditions
- Cardiomyopathy, Hypertrophic Obstructive
- Being tested
- Mavacamten
- Keywords
- Mavacamten, Obstructive Hypertrophic Cardiomyopathy (HCM)
- Medical subject headings
- Cardiomyopathy, Hypertrophic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MavacamtenDrug
Also called BMS-986427, MYK-461
Specified dose on specified days
| Study group | Role | What they receive |
|---|---|---|
| Mavacamten | Experimental | Not described Receives: Drug: Mavacamten |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: At Baseline and Week 30
Secondary outcomes (5)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: At baseline and week 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2022
- Study start19 Aug 2022
- Primary completion27 Nov 2023
- Study completion11 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cardiomyopathy, Hypertrophic ObstructiveStudies of the same condition, open and under way.
- More trials run by Bristol-Myers SquibbThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bristol-Myers Squibb
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bristol-Myers Squibb, Study director, Bristol-Myers Squibb
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05414175
- Sponsor's protocol ID
- CV027-004
- Acronym
- HORIZON-HCM
- Status last verified
- Jan 2026
- First submitted
- 7 Jun 2022
- Last updated
- 27 Jan 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No