Hypertrophic Cardiomyopathy (HCM)MAVA-HCMNCT07529938
Mavacamten in Adult Patients With Obstructive Hypertrophic Cardiomyopathy
An observational study of Hypertrophic Cardiomyopathy (HCM) and Obstructive Hypertrophic Cardiomyopathy (oHCM), run by Chinese Academy of Medical Sciences, Fuwai Hospital at 1 site in China.
Trial summary
This retrospective cohort study aims to evaluate the clinical efficacy and safety of mavacamten in adult patients with obstructive hypertrophic cardiomyopathy (oHCM). A total of 222 patients were included and categorized based on treatments received in routine clinical practice into a mavacamten group and a standard… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hypertrophic Cardiomyopathy (HCM), Obstructive Hypertrophic Cardiomyopathy (oHCM)
- What is being tested?
- mavacamten
- How many people
- 222 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2024to 30 Jun 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
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
- No healthy volunteers
- Adult, Older adult
Study population: Adult patients diagnosed with obstructive hypertrophic cardiomyopathy who received treatment at Fuwai Central China Cardiovascular Hospital between January 2024 and May 2025 in routine clinical practice.
Who may be able to join
- Age ≥18 years
- Diagnosis of hypertrophic cardiomyopathy
- NYHA functional class II-III
- First-time treatment with mavacamten
Who cannot join
- Known hypersensitivity to mavacamten or its components
- Left ventricular ejection fraction (LVEF) <55%
- Septal reduction therapy within 6 months prior to enrollment
- Presence of other conditions requiring long-term anticoagulation
- Pregnancy or breastfeeding
- Any condition deemed unsuitable by the investigator
Trial locations and contacts
1 site in 1 country.
China1 site
Fuwai Central China Cardiovascular Hospital
Zhengzhou, Henan, 450000
About this study
Official title: Mavacamten for Improving Cardiac Function and Clinical Outcomes in Adult Patients With Obstructive Hypertrophic Cardiomyopathy: A Retrospective Cohort Study
This retrospective cohort study aims to evaluate the clinical efficacy and safety of mavacamten in adult patients with obstructive hypertrophic cardiomyopathy (oHCM). A total of 222 patients were included and categorized based on treatments received in routine clinical practice into a mavacamten group and a standard therapy group.
The primary outcome is the change in resting left ventricular outflow tract (LVOT) gradient at Week 30. Secondary outcomes include changes in Valsalva LVOT gradient, New York Heart Association (NYHA) functional class, cardiac biomarkers, and echocardiographic parameters. Safety outcomes include adverse events and left ventricular systolic dysfunction.
This study provides real-world evidence on the effectiveness and safety of mavacamten in Chinese patients with oHCM.
Read the detailed description
Hypertrophic cardiomyopathy (HCM) is a genetic cardiac disorder characterized by left ventricular hypertrophy, with approximately two-thirds of patients presenting with obstructive physiology. Obstructive hypertrophic cardiomyopathy (oHCM) is associated with significant morbidity due to left ventricular outflow tract (LVOT) obstruction.
Mavacamten, a first-in-class cardiac myosin inhibitor, directly targets the underlying pathophysiology of oHCM by reducing excessive myosin-actin cross-bridge formation. Although previous randomized clinical trials, such as EXPLORER-CN, have demonstrated its efficacy, real-world data in Chinese populations remain limited.
This study is a retrospective cohort study conducted at Fuwai Central China Cardiovascular Hospital. Patients were not assigned to interventions. Instead, they were categorized based on treatments received in routine clinical practice. Eligible adult patients with oHCM who initiated mavacamten therapy were included in the treatment group, while patients receiving standard pharmacological therapy served as the control group.
Clinical data were collected from January 2024 to May 2025, with final database lock completed in June 2025. Baseline characteristics, echocardiographic parameters, and laboratory biomarkers were obtained from medical records.
The primary endpoint was the change in resting LVOT peak gradient at Week 30 compared with baseline. Secondary endpoints included changes in Valsalva LVOT gradient, NYHA functional class improvement, NT-proBNP, high-sensitivity cardiac troponin I (hs-cTnI), left ventricular ejection fraction (LVEF), and tricuspid annular plane systolic excursion (TAPSE). Safety endpoints included adverse events, serious adverse events, treatment discontinuation, and reduction of LVEF below 50%.
This study aims to provide real-world evidence regarding the clinical effectiveness and safety of mavacamten in adult patients with oHCM in China.
- Being tested
- mavacamten
- Keywords
- Mavacamten, Hypertrophic Cardiomyopathy, LVOT obstruction, Cardiac function, Real-world study, Retrospective cohort, Echocardiography
- Medical subject headings
- Cardiomyopathy, Hypertrophic, Ventricular Outflow Obstruction, Left
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mavacamtenDrug
Also called Camzyos, Mai Fan Tuo
Mavacamten was administered as part of routine clinical care for patients with obstructive hypertrophic cardiomyopathy. This observational study did not assign interventions; instead, patients were categorized based on treatments received in real-world clinical practice. The initial dose was 2.5 mg once daily, with dose adjustments based on left ventricular ejection fraction and LVOT gradient as clinically indicated.
| Study group | Role | What they receive |
|---|---|---|
| Mavacamten Group | Adult patients with obstructive hypertrophic cardiomyopathy (oHCM) who received mavacamten as part of routine clinical practice. Patients were not assigned to interventions but were categorized based on treatment received. Receives: Drug: mavacamten | |
| Standard Therapy Group | Adult patients with obstructive hypertrophic cardiomyopathy (oHCM) who received standard pharmacological therapy, including beta-blockers or non-dihydropyridine calcium channel blockers, in routine clinical practice. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Resting Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30
Change from baseline in resting left ventricular outflow tract (LVOT) peak gradient measured by Doppler echocardiography (unit: mmHg). Higher values indicate greater obstruction.
Measured: Baseline to Week 30
Secondary outcomes (10)
Change in Valsalva-Induced Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30
Change from baseline in LVOT peak gradient induced by the Valsalva maneuver measured by Doppler echocardiography (unit: mmHg). Higher values indicate greater obstruction.
Measured: Baseline to Week 30
Proportion of Participants with Improvement in New York Heart Association (NYHA) Functional Classification at Week 30
Proportion of participants achieving at least one class improvement in New York Heart Association (NYHA) functional classification compared with baseline. NYHA class ranges from I to IV, where higher classes indicate worse functional status.
Measured: Baseline to Week 30
Change in N-terminal pro-B-type Natriuretic Peptide (NT-proBNP) Levels at Week 30
Change from baseline in N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels (unit: pg/mL). Higher values indicate worse cardiac function.
Measured: Baseline to Week 30
Change in High-Sensitivity Cardiac Troponin I (hs-cTnI) Levels at Week 30
Change from baseline in high-sensitivity cardiac troponin I (hs-cTnI) levels (unit: ng/L). Higher values indicate greater myocardial injury.
Measured: Baseline to Week 30
Change in Left Ventricular Ejection Fraction (LVEF) at Week 30
Change from baseline in left ventricular ejection fraction (LVEF) measured by echocardiography (unit: %). Higher values indicate better cardiac systolic function.
Measured: Baseline to Week 30
Change in Tricuspid Annular Plane Systolic Excursion (TAPSE) at Week 30
Change from baseline in tricuspid annular plane systolic excursion (TAPSE) measured by echocardiography (unit: mm). Higher values indicate better right ventricular function.
Measured: Baseline to Week 30
Incidence of Adverse Events
Number and proportion of participants experiencing any adverse events.
Measured: Up to Week 30
Incidence of Serious Adverse Events
Number and proportion of participants experiencing serious adverse events.
Measured: Up to Week 30
Incidence of Treatment Discontinuation Due to Adverse Events
Number and proportion of participants who discontinued treatment due to adverse events.
Measured: Up to Week 30
Incidence of Left Ventricular Ejection Fraction Reduction Below 50%
Number and proportion of participants with LVEF <50% during treatment.
Measured: Up to Week 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2026
- Study start1 Jan 2024
- Primary completion30 May 2025
- Study completion30 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypertrophic Cardiomyopathy (HCM)Studies of the same condition, open and under way.
- More trials run by Chinese Academy of Medical Sciences, Fuwai HospitalThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 222 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Yanan Shi, Chinese Academy of Medical Sciences, Fuwai Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07529938
- Sponsor's protocol ID
- FW-CHC-HCM-2024-01
- Acronym
- MAVA-HCM
- Status last verified
- Apr 2026
- First submitted
- 8 Apr 2026
- Last updated
- 17 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Individual participant data (IPD) sharing has not yet been determined. Due to the retrospective nature of this study and the use of de-identified clinical data, any potential data sharing will be considered in accordance with institutional policies, ethical approvals, and applicable data protection regulations.
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mavacamten for Improving Cardiac Function and Clinical Outcomes in Adult Patients With Obstructive Hypertrophic Cardiomyopathy: A Retrospective Cohort Study
- ClinicalTrials.gov ID
- NCT07529938
- Acronym / sponsor ID
- MAVA-HCM / FW-CHC-HCM-2024-01
- Keywords
- Mavacamten, Hypertrophic Cardiomyopathy, LVOT obstruction, Cardiac function, Real-world study, Retrospective cohort, Echocardiography
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 222 participants (actual)
- Lead sponsor
- Chinese Academy of Medical Sciences, Fuwai HospitalUniversity, hospital or other
- Study start
- 1 Jan 2024 (actual)
- Primary completion
- 30 May 2025 (actual)
- Study completion
- 30 Jun 2025 (actual)
- First posted
- 14 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This retrospective cohort study aims to evaluate the clinical efficacy and safety of mavacamten in adult patients with obstructive hypertrophic cardiomyopathy (oHCM). A total of 222 patients were included and categorized based on treatments received in routine clinical practice into a mavacamten group and a standard therapy group.
The primary outcome is the change in resting left ventricular outflow tract (LVOT) gradient at Week 30. Secondary outcomes include changes in Valsalva LVOT gradient, New York Heart Association (NYHA) functional class, cardiac biomarkers, and echocardiographic parameters. Safety outcomes include adverse events and left ventricular systolic dysfunction.
This study provides real-world evidence on the effectiveness and safety of mavacamten in Chinese patients with oHCM.
Detailed description
Hypertrophic cardiomyopathy (HCM) is a genetic cardiac disorder characterized by left ventricular hypertrophy, with approximately two-thirds of patients presenting with obstructive physiology. Obstructive hypertrophic cardiomyopathy (oHCM) is associated with significant morbidity due to left ventricular outflow tract (LVOT) obstruction.
Mavacamten, a first-in-class cardiac myosin inhibitor, directly targets the underlying pathophysiology of oHCM by reducing excessive myosin-actin cross-bridge formation. Although previous randomized clinical trials, such as EXPLORER-CN, have demonstrated its efficacy, real-world data in Chinese populations remain limited.
This study is a retrospective cohort study conducted at Fuwai Central China Cardiovascular Hospital. Patients were not assigned to interventions. Instead, they were categorized based on treatments received in routine clinical practice. Eligible adult patients with oHCM who initiated mavacamten therapy were included in the treatment group, while patients receiving standard pharmacological therapy served as the control group.
Clinical data were collected from January 2024 to May 2025, with final database lock completed in June 2025. Baseline characteristics, echocardiographic parameters, and laboratory biomarkers were obtained from medical records.
The primary endpoint was the change in resting LVOT peak gradient at Week 30 compared with baseline. Secondary endpoints included changes in Valsalva LVOT gradient, NYHA functional class improvement, NT-proBNP, high-sensitivity cardiac troponin I (hs-cTnI), left ventricular ejection fraction (LVEF), and tricuspid annular plane systolic excursion (TAPSE). Safety endpoints included adverse events, serious adverse events, treatment discontinuation, and reduction of LVEF below 50%.
This study aims to provide real-world evidence regarding the clinical effectiveness and safety of mavacamten in adult patients with oHCM in China.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mavacamten Group | – | Drug: mavacamtenAdult patients with obstructive hypertrophic cardiomyopathy (oHCM) who received mavacamten as part of routine clinical practice. Patients were not assigned to interventions but were categorized based on treatment received. |
| Standard Therapy Group | – | –Adult patients with obstructive hypertrophic cardiomyopathy (oHCM) who received standard pharmacological therapy, including beta-blockers or non-dihydropyridine calcium channel blockers, in routine clinical practice. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult patients diagnosed with obstructive hypertrophic cardiomyopathy who received treatment at Fuwai Central China Cardiovascular Hospital between January 2024 and May 2025 in routine clinical practice.
Key inclusion criteria (4 listed)
- Age ≥18 years
- Diagnosis of hypertrophic cardiomyopathy
- NYHA functional class II-III
- First-time treatment with mavacamten
Key exclusion criteria (6 listed)
- Known hypersensitivity to mavacamten or its components
- Left ventricular ejection fraction (LVEF) <55%
- Septal reduction therapy within 6 months prior to enrollment
- Presence of other conditions requiring long-term anticoagulation
- Pregnancy or breastfeeding
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| mavacamtenAlso: Camzyos, Mai Fan TuoMavacamten was administered as part of routine clinical care for patients with obstructive hypertrophic cardiomyopathy. This observational study did not assign interventions; instead, patients were categorized based on treatments received in real-world clinical practice. The initial dose was 2.5 mg once daily, with dose adjustments based on left ventricular ejection fraction and LVOT gradient as clinically indicated. | Drug | Mavacamten Group |
Primary outcome
Change in Resting Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30
Time frame: Baseline to Week 30
Change from baseline in resting left ventricular outflow tract (LVOT) peak gradient measured by Doppler echocardiography (unit: mmHg). Higher values indicate greater obstruction.
10 secondary outcomes
Change in Valsalva-Induced Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30
Time frame: Baseline to Week 30
Proportion of Participants with Improvement in New York Heart Association (NYHA) Functional Classification at Week 30
Time frame: Baseline to Week 30
Change in N-terminal pro-B-type Natriuretic Peptide (NT-proBNP) Levels at Week 30
Time frame: Baseline to Week 30
Change in High-Sensitivity Cardiac Troponin I (hs-cTnI) Levels at Week 30
Time frame: Baseline to Week 30
Change in Left Ventricular Ejection Fraction (LVEF) at Week 30
Time frame: Baseline to Week 30
Change in Tricuspid Annular Plane Systolic Excursion (TAPSE) at Week 30
Time frame: Baseline to Week 30
Incidence of Adverse Events
Time frame: Up to Week 30
Incidence of Serious Adverse Events
Time frame: Up to Week 30
Incidence of Treatment Discontinuation Due to Adverse Events
Time frame: Up to Week 30
Incidence of Left Ventricular Ejection Fraction Reduction Below 50%
Time frame: Up to Week 30
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Apr 2026.
- Individual participant dataSharing planned: Undecided. Individual participant data (IPD) sharing has not yet been determined. Due to the retrospective nature of this study and the use of de-identified clinical data, any potential data sharing will be considered in accordance with institutional policies, ethical approvals, and applicable data protection regulations.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hypertrophic Cardiomyopathy (HCM) and Obstructive Hypertrophic Cardiomyopathy (oHCM), run by Chinese Academy of Medical Sciences, Fuwai Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Resting Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30 (time frame: Baseline to Week 30). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Apr 2026.