Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Mavacamtenis a novel, small molecule, selective allosteric inhibitor of cardiac-specific myosin, for the treatment of patients with symptomatic oHCM. This study will assess the efficacy and safety of mavacamten in Chinese adults with symptomatic oHCM.
This is a randomized, double-blinded, placebo-controlled clinical study witha long-term extension to evaluate the efficacy and safety of mavacamten in Chinese adults with symptomatic oHCM. Approximately 81eligible participants will be enrolled and randomized in a 2:1 ratio (mavacamten:placebo). Participants will receive mavacamten or matching placebofor 30 weeks indouble-blinded manner. After 30-week double-blinded placebo-controlled treatment, eligible participants will receive mavacamten for additional 48 weeks (placebogroup: switch from placebo to mavacamten, mavacamten group: maintain on mavacamten).
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Mavacamten | Experimental | Mavacamten Capsules |
|
| placebo | Placebo Comparator | Matching Placebo Capsules |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Mavacamten | Drug | Mavacamten Capsules |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Week 30 in Valsalva Left Ventricular Outflow Tract (LVOT) Peak Gradient | To compare the effect of a 30-week course of mavacamten with placebo on Valsalva LVOT peak gradient as determined by Doppler echocardiography | 30 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Week 30 in Resting LVOT Peak Gradient | To compare the effect of a 30-week course of mavacamten with placebo on LVOT obstruction. | 30 weeks |
| Proportion of Participants Achieving a Valsalva LVOT Peak Gradient < 30 mmHg at Week 30 |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Shuyang Zhang, M.D., Ph.D. | Peking Union Medical College Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | Beijing | Beijing Municipality | 100730 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42179278 | Derived | Tian Z, Li X, Li L, Zhang Q, Wang J, Shi Y, Peng D, Ma W, Yang P, Cheng X, Jin W, Wang F, Xie Y, Pan B, Florea V, Zhang S. Long-Term Efficacy and Safety of Mavacamten in Chinese Patients With Obstructive Hypertrophic Cardiomyopathy: Week 78 Results From the EXPLORER-CN Study. J Am Heart Assoc. 2026 Jun 2;15(11):e046251. doi: 10.1161/JAHA.125.046251. Epub 2026 May 25. | |
| 42165935 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
A total of 81 eligible participants were randomly assigned to one of 2 treatment groups, mavacamten or placebo in a ratio of 2:1 (2 mavacamten and 1 placebo). Randomization was stratified according to current treatment at enrollment with a beta-blocker (yes or no)
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Mavacamten | Mavacamten Capsules Mavacamten: Mavacamten Capsules |
| FG001 | Placebo | Matching Placebo Capsules Placebo: Matching PBO capsules during placebo controlled period,and mavacamten capsules during long term extension period |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Mavacamten | Mavacamten Capsules Mavacamten: Mavacamten Capsules |
| BG001 | Placebo | Matching Placebo Capsules Placebo: Matching PBO capsules during placebo controlled period,and mavacamten capsules during long term extension period |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| 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 to Week 30 in Valsalva Left Ventricular Outflow Tract (LVOT) Peak Gradient | To compare the effect of a 30-week course of mavacamten with placebo on Valsalva LVOT peak gradient as determined by Doppler echocardiography | Posted | Mean | Standard Deviation | mmHg | 30 weeks |
|
Adverse events were assessed from the first dose date of study medication up to 30 weeks.
Consistent with clinicaltrials.gov definitions.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Matching Placebo Capsules Placebo: Matching PBO capsules during placebo controlled period,and mavacamten capsules during long term extension period |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Haemorrhoids | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| COVID-19 | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Prof. Shuyang Zhang | Peking Union Medical College Hospital | +86 13911667211 | shuyangzhang103@163.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 25, 2023 | Jul 22, 2024 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 21, 2020 | Jul 22, 2024 | SAP_001.pdf |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D002312 | Cardiomyopathy, Hypertrophic |
| ID | Term |
|---|---|
| D009202 | Cardiomyopathies |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D001020 | Aortic Stenosis, Subvalvular |
Not provided
Not provided
| ID | Term |
|---|---|
| C000605992 | MYK-461 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo | Drug | Matching PBO capsules during placebo controlled period,and mavacamten capsules during long term extension period |
|
To compare the effect of a 30-week course of mavacamten with placebo on LVOT obstruction. |
| 30 weeks |
| Proportion of Participants Achieving a Valsalva LVOT Peak Gradient < 50 mmHg at Week 30. | To compare the effect of a 30-week course of mavacamten with placebo on LVOT obstruction. | 30 weeks |
| Proportion of Participants With at Least 1 Class Improvement in New York Heart Association (NYHA) Functional Classification From Baseline to Week 30 | To compare the effect of a 30-week course of mavacamten with placebo on clinical symptoms | 30 weeks |
| Change From Baseline to Week 30 in Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score (CSS) | To compare the effect of a 30-week course of Mavacamten with placebo on Participant-Reported health status individually The KCCQ (23-item version) is a patient-reported questionnaire that measures the impact of patients' CV disease or its treatment on 6 distinct domains using a 2-week recall: symptoms/signs, physical limitations, quality of life, social limitations, self-efficacy, and symptom stability (Green et al., 2000). In addition to the individual domains, 2 summary scores can be calculated from the KCCQ: the overall summary score (includes the total symptom, physical limitation, social limitations and quality of life scores) and the clinical summary score (combines the total symptom and physical limitation scales). Scores range from 0 to 100, with higher scores reflecting better health status. The KCCQ will be administered to participants as indicated. | 30 weeks |
| Change From Baseline to Week 30 in N-terminal Pro-B-type Natriuretic Peptide (NT-proBNP) | To compare the effect of a 30-week course of mavacamten on cardiac biomarkers | 30 weeks |
| Change From Baseline to Week 30 in Cardiac Troponin | To compare the effect of a 30-week course of mavacamten on cardiac biomarkers | 30 weeks |
| Change From Baseline to Week 30 in Left Ventricular (LV) Mass Index | To compare the effect of a 30-week course of mavacamten with placebo on LV mass evaluated by cardiac magnetic resonance (CMR) imaging. | 30 weeks |
| Tian Z, Li X, Li L, Zhang Q, Wang J, Shi Y, Peng D, Ma W, Yang P, Cheng X, Jin W, Wang F, Memaj A, Xie Y, Pan B, Florea V, Zheng J, Zhong Y, Zhang S. Mavacamten in Chinese Patients with Obstructive Hypertrophic Cardiomyopathy: Patient-Reported Health Status Analysis up to 78 Weeks in the EXPLORER-CN Study. Cardiol Ther. 2026 Jun;15(2):185-200. doi: 10.1007/s40119-026-00449-5. Epub 2026 May 21. |
| 40632050 | Derived | Tian Z, Li L, Li X, Zhang Q, Peng D, Ma W, Yang P, Wang F, Cheng X, Fu Y, Sun J, Wang J, Zhang S. Effects of Mavacamten on Cardiac Magnetic Resonance Features in Chinese Patients With Obstructive Hypertrophic Cardiomyopathy. JACC Asia. 2025 Aug;5(8):1064-1074. doi: 10.1016/j.jacasi.2025.05.015. Epub 2025 Jul 8. |
| 40299193 | Derived | Tian Z, Li X, Li L, Zhang Q, Wang J, Shi Y, Peng D, Yang P, Ma W, Wang F, Jin W, Cheng X, Chen YM, Zhong Y, Barrett YC, Zheng J, Zhang S. Effect of Mavacamten on Echocardiographic Features in Chinese Patients with Obstructive Hypertrophic Cardiomyopathy: Results from the EXPLORER-CN Study. Cardiol Ther. 2025 Jun;14(2):267-282. doi: 10.1007/s40119-025-00409-5. Epub 2025 Apr 29. |
| 37639259 | Derived | Tian Z, Li L, Li X, Wang J, Zhang Q, Li Z, Peng D, Yang P, Ma W, Wang F, Jin W, Cheng X, Sun J, Fu Y, Lyu C, Zhang S. Effect of Mavacamten on Chinese Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy: The EXPLORER-CN Randomized Clinical Trial. JAMA Cardiol. 2023 Oct 1;8(10):957-965. doi: 10.1001/jamacardio.2023.3030. |
| 37336533 | Derived | Tian Z, Wang F, Jin W, Zhang Q, Zhou J, Yang P, Wang G, Hsu P, Sun J, Zhang S, Han Y. Study design and rationale of EXPLORER-CN: a phase III, randomised, double-blind, placebo-controlled clinical study to evaluate the efficacy and safety of mavacamten in Chinese adults with symptomatic obstructive hypertrophic cardiomyopathy. BMJ Open. 2023 Jun 19;13(6):e071473. doi: 10.1136/bmjopen-2022-071473. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Valsalva LVOT peak gradient | Mean | Standard Deviation | mmHg |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Secondary | Change From Baseline to Week 30 in Resting LVOT Peak Gradient | To compare the effect of a 30-week course of mavacamten with placebo on LVOT obstruction. | Posted | Mean | Standard Deviation | mmHg | 30 weeks |
|
|
|
| Secondary | Proportion of Participants Achieving a Valsalva LVOT Peak Gradient < 30 mmHg at Week 30 | To compare the effect of a 30-week course of mavacamten with placebo on LVOT obstruction. | Posted | Count of Participants | Participants | 30 weeks |
|
|
|
| Secondary | Proportion of Participants Achieving a Valsalva LVOT Peak Gradient < 50 mmHg at Week 30. | To compare the effect of a 30-week course of mavacamten with placebo on LVOT obstruction. | Posted | Count of Participants | Participants | 30 weeks |
|
|
|
| Secondary | Proportion of Participants With at Least 1 Class Improvement in New York Heart Association (NYHA) Functional Classification From Baseline to Week 30 | To compare the effect of a 30-week course of mavacamten with placebo on clinical symptoms | Posted | Count of Participants | Participants | 30 weeks |
|
|
|
| Secondary | Change From Baseline to Week 30 in Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score (CSS) | To compare the effect of a 30-week course of Mavacamten with placebo on Participant-Reported health status individually The KCCQ (23-item version) is a patient-reported questionnaire that measures the impact of patients' CV disease or its treatment on 6 distinct domains using a 2-week recall: symptoms/signs, physical limitations, quality of life, social limitations, self-efficacy, and symptom stability (Green et al., 2000). In addition to the individual domains, 2 summary scores can be calculated from the KCCQ: the overall summary score (includes the total symptom, physical limitation, social limitations and quality of life scores) and the clinical summary score (combines the total symptom and physical limitation scales). Scores range from 0 to 100, with higher scores reflecting better health status. The KCCQ will be administered to participants as indicated. | Posted | Mean | Standard Deviation | score on a scale | 30 weeks |
|
|
|
| Secondary | Change From Baseline to Week 30 in N-terminal Pro-B-type Natriuretic Peptide (NT-proBNP) | To compare the effect of a 30-week course of mavacamten on cardiac biomarkers | Posted | Mean | Standard Deviation | ng/L | 30 weeks |
|
|
|
| Secondary | Change From Baseline to Week 30 in Cardiac Troponin | To compare the effect of a 30-week course of mavacamten on cardiac biomarkers | Posted | Mean | Standard Deviation | ng/L | 30 weeks |
|
|
|
| Secondary | Change From Baseline to Week 30 in Left Ventricular (LV) Mass Index | To compare the effect of a 30-week course of mavacamten with placebo on LV mass evaluated by cardiac magnetic resonance (CMR) imaging. | Posted | Median | Standard Deviation | g/m^2 | 30 weeks |
|
|
|
| 0 |
| 27 |
| 0 |
| 27 |
| 24 |
| 27 |
| EG001 | Mavacamten | Mavacamten Capsules Mavacamten: Mavacamten Capsules | 0 | 54 | 4 | 54 | 45 | 54 |
| Atrial flutter | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
|
| fibrillation | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
|
| Hypotension | Vascular disorders | MedDRA (25.1) | Systematic Assessment |
|
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA (25.1) | Systematic Assessment |
|
| Sinus arrest | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
|
| Atrial fibrillation | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
|
| Defect conduction intraventricular | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
|
| Palpitations | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
|
| Ventricular extrasystoles | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
|
| Cardiac discomfort | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
|
| Ventricular tachycardia | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
|
| Hyperuricemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
|
| Hyperlipidaemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
|
| Hypoaesthesia | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
|
| Hepatic enzyme increased | Investigations | MedDRA (25.1) | Systematic Assessment |
|
| Electrocardiogram QT prolonged | Investigations | MedDRA (25.1) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
|
| Hepatic function abnormal | Hepatobiliary disorders | MedDRA (25.1) | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA (25.1) | Systematic Assessment |
|
Not provided
Not provided
| D001024 |
| Aortic Valve Stenosis |
| D000082862 | Aortic Valve Disease |
| D006349 | Heart Valve Diseases |