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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| 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. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| mavacamten | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | From baseline to week 30. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | At week 30. |
| The proportion of patients with a peak left ventricular outflow tract (LVOT) gradient of less than 50 mmHg. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Shanghai Chest Hospital | Shanghai | China |
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| ID | Term |
|---|---|
| D002312 | Cardiomyopathy, Hypertrophic |
| ID | Term |
|---|---|
| D009202 | Cardiomyopathies |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D001020 | Aortic Stenosis, Subvalvular |
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| ID | Term |
|---|---|
| C000605992 | MYK-461 |
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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. |
| 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. | 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. | 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. | 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. | 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). | From baseline to week 30. |
| D001024 |
| Aortic Valve Stenosis |
| D000082862 | Aortic Valve Disease |
| D006349 | Heart Valve Diseases |