HCM - Hypertrophic CardiomyopathyNCT06947590
Efficacy of Mavacamten in Patients With Symptomatic Latent Obstructive Hypertrophic Cardiomyopathy
A study testing mavacamten in people with HCM - Hypertrophic Cardiomyopathy and Mavacamten, run by Xu Liu at 1 site in China.
Trial summary
This study aimed to evaluate the efficacy and safety of Mavacamten compared to no treatment in patients with symptomatic latent obstructive hypertrophic cardiomyopathy. The trial was randomized into two groups: Mavacamten group and Non-Mavacamten group. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- HCM - Hypertrophic Cardiomyopathy, Mavacamten
- What is being tested?
- mavacamten
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 78 participants
- Where
- 1 siteChina
- Study dates
- 20 Apr 2025to 1 Feb 2026
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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
Who may be able to join
- Age ≥18 years.
- Weight greater than 45 kg.
- Adequate acoustic windows to allow for accurate transthoracic echocardiograms (TTEs).
- Diagnosis of latent obstructive hypertrophic cardiomyopathy, in accordance with the current guidelines of the American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Chinese Society of Cardiology.
- Left ventricular ejection fraction (LVEF) ≥55% at rest, confirmed by the echocardiography core laboratory.
- New York Heart Association (NYHA) Class II or III symptoms at the time of screening.
- Resting oxygen saturation ≥90% at the time of screening.
Who cannot join
- Any acute or severe comorbidities (e.g., severe infections or hematological, renal, metabolic, gastrointestinal, or endocrine dysfunction).
- Currently using or having used prohibited medications within 14 days prior to screening, such as cytochrome CYP2C19 inhibitors (e.g., omeprazole or esomeprazole) or strong CYP3A4 inhibitors.
- Life expectancy of less than 1 year.
- Pregnant or breastfeeding women.
- History of syncope or sustained ventricular tachyarrhythmia during exercise within the past 6 months.
- Atrial fibrillation (AF).
- Patients currently receiving or planning to receive treatment with disopyramide, cibenzoline, ranolazine, or a combination of beta-blockers with verapamil or diltiazem.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Efficacy of Mavacamten in Patients With Symptomatic Latent Obstructive Hypertrophic Cardiomyopathy: A Randomized Controlled Trial
This study aimed to evaluate the efficacy and safety of Mavacamten compared to no treatment in patients with symptomatic latent obstructive hypertrophic cardiomyopathy. The trial was randomized into two groups: Mavacamten group and Non-Mavacamten group. Over the 30-week treatment period, patients underwent a series of assessments at predefined time points, including transthoracic echocardiography, electrocardiogram (ECG), Holter monitoring, NYHA functional classification, Kansas City Cardiomyopathy Questionnaire (KCCQ), and cardiac biomarkers.
- Conditions
- HCM - Hypertrophic Cardiomyopathy, Mavacamten
- Being tested
- mavacamten
- Keywords
- HCM, Mavacamten
- 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
Patients in this group were treated with Mavacamten, starting at an initial dose of 2.5 mg, administered orally once daily. Subsequent doses were adjusted based on changes in pressure gradients and cardiac function observed during follow-up.
| Study group | Role | What they receive |
|---|---|---|
| Mavacamten Group | Experimental | Patients in this group were treated with Mavacamten, starting at an initial dose of 2.5 mg, administered orally once daily. Subsequent doses were adjusted based on changes in pressure gradients and cardiac function observed during follow-up. Receives: Drug: mavacamten |
| Control Group | No intervention | Patients in this group did not receive Mavacamten treatment and were instead managed with traditional therapies of their choice, such as beta-blockers or calcium channel blockers. |
What is being measured
The primary outcome is the main question the study is designed to answer.
The change in peak left ventricular outflow tract (LVOT) gradient
The change in peak left ventricular outflow tract (LVOT) gradient, determined by Doppler echocardiography, during exercise or pharmacologic provocation from baseline to week 30.
Measured: From baseline to week 30.
Secondary outcomes (7)
The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 30 mmHg.
The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 30 mmHg during exercise or pharmacologic provocation at week 30.
Measured: At week 30.
The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 50 mmHg.
The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 50 mmHg during exercise or pharmacologic provocation at week 30.
Measured: At week 30.
The proportion of patients with at least a one-class improvement in NYHA functional classification.
The proportion of patients with at least a one-class improvement in NYHA functional classification at week 30.
Measured: At week 30.
The change in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).
The change in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) from baseline to week 30.
Measured: From baseline to week 30.
The change in N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels.
The change in N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels from baseline to week 30.
Measured: From baseline to week 30.
The change in high-sensitivity cardiac troponin I (hs-cTnI) levels.
The change in high-sensitivity cardiac troponin I (hs-cTnI) levels from baseline to week 30.
Measured: From baseline to week 30.
Key safety endpoints
Key safety endpoints include the incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).
Measured: From baseline 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 posted27 Apr 2025
- Study start20 Apr 2025
- Primary completion1 Feb 2026 (estimated)
- Study completion1 Feb 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HCM - Hypertrophic CardiomyopathyStudies of the same condition, open and under way.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Investigator, Outcomes assessor
- Enrolment
- 78 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xu Liu
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Xu Liu, Shanghai Chest Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06947590
- Sponsor's protocol ID
- MVLOHCM
- Status last verified
- Apr 2025
- First submitted
- 20 Apr 2025
- Last updated
- 27 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Mavacamten in Patients With Symptomatic Latent Obstructive Hypertrophic Cardiomyopathy: A Randomized Controlled Trial
- ClinicalTrials.gov ID
- NCT06947590
- Acronym / sponsor ID
- MVLOHCM
- Conditions
- HCM - Hypertrophic Cardiomyopathy, Mavacamten
- Keywords
- HCM, Mavacamten
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 78 participants (estimated)
- Lead sponsor
- Xu LiuUniversity, hospital or other
- Study start
- 20 Apr 2025 (actual)
- Primary completion
- 1 Feb 2026 (estimated)
- Study completion
- 1 Feb 2026 (estimated)
- First posted
- 27 Apr 2025 (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 study aimed to evaluate the efficacy and safety of Mavacamten compared to no treatment in patients with symptomatic latent obstructive hypertrophic cardiomyopathy. The trial was randomized into two groups: Mavacamten group and Non-Mavacamten group. Over the 30-week treatment period, patients underwent a series of assessments at predefined time points, including transthoracic echocardiography, electrocardiogram (ECG), Holter monitoring, NYHA functional classification, Kansas City Cardiomyopathy Questionnaire (KCCQ), and cardiac biomarkers.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mavacamten Group | Experimental | Drug: mavacamtenPatients in this group were treated with Mavacamten, starting at an initial dose of 2.5 mg, administered orally once daily. Subsequent doses were adjusted based on changes in pressure gradients and cardiac function observed during follow-up. |
| Control Group | No intervention | –Patients in this group did not receive Mavacamten treatment and were instead managed with traditional therapies of their choice, such as beta-blockers or calcium channel blockers. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age ≥18 years.
- Weight greater than 45 kg.
- Adequate acoustic windows to allow for accurate transthoracic echocardiograms (TTEs).
- Diagnosis of latent obstructive hypertrophic cardiomyopathy, in accordance with the current guidelines of the American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Chinese Society of Cardiology.
- Left ventricular ejection fraction (LVEF) ≥55% at rest, confirmed by the echocardiography core laboratory.
Key exclusion criteria (7 listed)
- Any acute or severe comorbidities (e.g., severe infections or hematological, renal, metabolic, gastrointestinal, or endocrine dysfunction).
- Currently using or having used prohibited medications within 14 days prior to screening, such as cytochrome CYP2C19 inhibitors (e.g., omeprazole or esomeprazole) or strong CYP3A4 inhibitors.
- Life expectancy of less than 1 year.
- Pregnant or breastfeeding women.
- History of syncope or sustained ventricular tachyarrhythmia during exercise within the past 6 months.
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 |
|---|---|---|
| mavacamtenPatients in this group were treated with Mavacamten, starting at an initial dose of 2.5 mg, administered orally once daily. Subsequent doses were adjusted based on changes in pressure gradients and cardiac function observed during follow-up. | Drug | Mavacamten Group |
Primary outcome
The change in peak left ventricular outflow tract (LVOT) gradient
Time frame: From baseline to week 30.
The change in peak left ventricular outflow tract (LVOT) gradient, determined by Doppler echocardiography, during exercise or pharmacologic provocation from baseline to week 30.
7 secondary outcomes
The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 30 mmHg.
Time frame: At week 30.
The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 50 mmHg.
Time frame: At week 30.
The proportion of patients with at least a one-class improvement in NYHA functional classification.
Time frame: At week 30.
The change in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).
Time frame: From baseline to week 30.
The change in N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels.
Time frame: From baseline to week 30.
The change in high-sensitivity cardiac troponin I (hs-cTnI) levels.
Time frame: From baseline to week 30.
Key safety endpoints
Time frame: From baseline to week 30.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2025.
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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?
A study testing mavacamten in people with HCM - Hypertrophic Cardiomyopathy and Mavacamten, run by Xu Liu 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 7 inclusion and 7 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: under way, not looking for participants, as of the sponsor's last update on 27 Apr 2025. 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?
This study is still under way. Results are not available in the source record yet.
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?
The change in peak left ventricular outflow tract (LVOT) gradient (time frame: From 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 27 Apr 2025.