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| ID | Type | Description | Link |
|---|---|---|---|
| CTR20233249 | Registry Identifier | China Center for Drug Evaluation(CDE) |
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This is an open-label extension study of China cohort in the phase 3 study (CY 6031) of aficamten for the treatment of obstructive HCM (oHCM) to collect long-term safety and tolerability data, including assessments of cardiac function and steady-state Pharmacokinetics (PK) during chronic dosing with aficamten.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Aficamten | Experimental | Patients in this arm take daily dose of Aficamten. Each patient will start at the lowest dose (5 mg) in the pre-specified dose range and undergo dose titration up to a maximum of 20 mg. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Aficamten | Drug | 5-20mg |
|
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| Measure | Description | Time Frame |
|---|---|---|
| Patient incidence of reported adverse events (AEs) | Baseline to End of study, up to 2 years | |
| Patient incidence of reported serious adverse events (SAEs ) | Baseline to End of study, up to 2 years | |
| Patient incidence of LVEF<50% & LVEF <40% | Baseline to End of study, up to 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Peak LVOT-G at rest and with Valsalva provocation | Change from baseline values to week 48 at 12-week intervals; | |
| Proportion of patients with resting LVOT-G <50 mmHg | Change from baseline values to week 48 at 12-week intervals; |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Yuhui Zhang, MD | Chinese Academy of Medical Sciences, Fuwai Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Beijing Anzhen Hospital | Beijing | China | ||||
| Beijing Chao-yang Hospital, Capital Medical University |
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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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| Proportion of patients with resting LVOT-G <30 mmHg | Change from baseline values to week 48 at 12-week intervals; |
| Proportion of patients with post-Valsalva LVOT-G <50 mmHg | Change from baseline values to week 48 at 12-week intervals; |
| Proportion of patients with post-Valsalva LVOT-G <30 mmHg | Change from baseline values to week 48 at 12-week intervals; |
| Proportion of patients with LVEF ≥50%, resting LVOT-G <30 mmHg, and post-Valsalva LVOT-G <50 mmHg | Change from baseline values to week 48 at 12-week intervals; |
| First resting LVOT-G <50 mmHg | Time to the following event through last follow-up, up to 2 years. |
| First resting LVOT-G <30 mmHg | Time to the following event through last follow-up, up to 2 years. |
| First post-Valsalva LVOT-G <50 mmHg | Time to the following event through last follow-up, up to 2 years. |
| First post-Valsalva LVOT-G <30 mmHg | Time to the following event through last follow-up, up to 2 years. |
| First LVEF ≥50%, resting LVOT-G <30 mmHg, and post-Valsalva LVOT-G <50 mmHg | Time to the following event through last follow-up, up to 2 years. |
| Beijing |
| China |
| Fuwai Hospital, CAMS & PUMC | Beijing | China |
| Peking University Third Hospital | Beijing | China |
| Sichuan Provincial People's Hospital | Chengdu | China |
| Nanfang Hospital | Guangzhou | China |
| The Second Affiliated Hospital of Nanchang University | Jiangxi | China |
| The First Hospital of Jilin University | Jilin City | China |
| Shengjing Hospital of China Medical University | Shenyang | China |
| Union Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology | Wuhan | China |
| The First Affiliated Hospital of Xi'an Jiaotong University | Xi'an | China |
| The First Affiliated Hospital of Zhengzhou University | Zhengzhou | China |
| D001024 |
| Aortic Valve Stenosis |
| D000082862 | Aortic Valve Disease |
| D006349 | Heart Valve Diseases |