| Primary | Part A: Number of Participants With Adverse Events and Serious Adverse Events | An Adverse Event (AE) is defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in a clinical investigation participant administered study treatment and that does not necessarily have a causal relationship with this treatment. Serious Adverse Event (SAE) is defined as any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or causes prolongation of existing hospitalization. | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. | Posted | | Count of Participants | | Participants | | From first dose (Day 1) until study end date in Part A or 30 days after early termination date (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
| | | Title | Denominators | Categories |
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| Participants with at least one adverse events in Part A | | | | Participants with at least one serious adverse events in Part A | | |
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| Primary | Part A: Number of Participants With at Least One Event of Arrhythmias | Arrhythmias included atrial fibrillation/flutter (new from screening and recurrent), ventricular tachyarrhythmias (ventricular tachycardia, ventricular fibrillation, and Torsades de Pointe). | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. | Posted | | Count of Participants | | Participants | | From first dose (Day 1) until study end date in Part A or 30 days after early termination date (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Primary | Part A: Number of Participants With at Least One Event of Appropriate Implantable Cardioverter Defibrillator Therapy and Resuscitated Cardiac Arrest | | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. | Posted | | Count of Participants | | Participants | | From first dose (Day 1) until study end date in Part A or 30 days after early termination date (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Primary | Part A: Change From Baseline in Vital Signs - Heart Rate | Baseline is defined as the last non-missing value in date/time recorded before the first dose of MYK-224. Heart rate was measured in rested state. | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. Only participants with data available at the specified timepoint are included in the analysis. | Posted | | Mean | Standard Deviation | beats per minute | | Baseline and until treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Primary | Part A: Change From Baseline in Vital Signs - Mean Systolic Blood Pressure | Baseline is defined as the last non-missing value in date/time recorded before the first dose of MYK-224. Systolic blood pressure was measured in rested state. | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. Only participants with data available at the specified timepoint are included in the analysis. | Posted | | Mean | Standard Deviation | mmHg | | Baseline and until treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Primary | Part A: Change From Baseline in Vital Signs - Mean Diastolic Blood Pressure | Baseline is defined as the last non-missing value in date/time recorded before the first dose of MYK-224. Diastolic blood pressure was measured in rested state. | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. Only participants with data available at the specified timepoint are included in the analysis. | Posted | | Mean | Standard Deviation | mmHg | | Baseline and until treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Primary | Part A: Change From Baseline in Vital Signs - Respiratory Rate | Baseline is defined as the last non-missing value in date/time recorded before the first dose of MYK-224. Respiratory rate was measured in rested state. | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. Only participants with data available at the specified timepoint are included in the analysis. | Posted | | Mean | Standard Deviation | breaths per minute | | Baseline and until treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Primary | Part A: Change From Baseline in Vital Signs - Temperature | Baseline is defined as the last non-missing value in date/time recorded before the first dose of MYK-224. | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. Only participants with data available at the specified timepoint are included in the analysis. | Posted | | Mean | Standard Deviation | Celsius | | Baseline and until treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Primary | Part A: Change From Baseline in Vital Signs - Weight | Baseline is defined as the last non-missing value in date/time recorded before the first dose of MYK-224. | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. Only participants with data available at the specified timepoint are included in the analysis. | Posted | | Mean | Standard Deviation | kilogram | | Baseline and until treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Primary | Part A: Number of Participants With Abnormal Physical Examination Results | The complete physical examination included weight and calculated BMI, a neurological examination (gross motor and deep tendon reflexes),and an assessment of the following: general appearance, skin, head and neck, mouth, lymph nodes, thyroid, abdomen, musculoskeletal, cardiovascular, neurological, and respiratory systems with other systems included, as directed by interval history. | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. | Posted | | Count of Participants | | Participants | | Baseline and until study end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Primary | Part A: Number of Participants With Abnormal Electrocardiogram (ECG) Results | Twelve-lead ECG evaluations will be performed in the supine position after 10 minutes of rest at Screening and at selected clinic visits prior to MYK-224 dosing and before any blood sample collection. QTc prolongation is defined by either the mean of QTcF > 499 or mean of QTcB > 499 according to the project requirement specification from Clario. | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. Only participants with data available at the specified timepoint are included in the analysis. | Posted | | Count of Participants | | Participants | | Baseline and until study end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Primary | Part A: Number of Participants With Adverse Events Related to Clinical Laboratory Parameters (Hematology, Chemistry and Urinalysis) | Blood samples were collected to assess the clinical laboratory parameters. | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. | Posted | | Count of Participants | | Participants | | Baseline and untill end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Primary | Part A:Percentage of Participants With Incidence of Symptomatic Left Ventricular Ejection Fraction (LVEF) < 50% | LVEF was assessed using transthoracic echocardiogram (TTE) in resting state. 90% CIs are calculated based on Exact Binomial proportion test. LVEF value was calculated by Simpson Biplane method. | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. | Posted | | Number | 90% Confidence Interval | percentage of participants | | From first dose (Day 1) until study end date in Part A or 30 days after early termination date (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Primary | Part A: Percentage of Participants With Incidence of Symptomatic Left Ventricular Ejection Fraction (LVEF) <= 30% | LVEF was assessed using transthoracic echocardiogram (TTE) in resting state. 90% CIs are calculated based on Exact Binomial proportion test. LVEF value was calculated by Simpson Biplane method. | Safety A population consist of all enrolled participants who received at least 1 dose of MYK-224. Pre-specified to be analyzed for Part A. | Posted | | Number | 90% Confidence Interval | percentage of participants | | From first dose (Day 1) until study end date in Part A or 30 days after early termination date (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Secondary | Part A: Aggregate Mean of Left Ventricular Ejection Fraction (LVEF) | | Efficacy/PD A population included All enrolled A participants who receive at least 1 dose of MYK-224 and have primary or secondary endpoint data, including a baseline value and at least 1 post-baseline value Pre-specified to be analyzed for Part A. | Posted | | Mean | Standard Deviation | percentage | | Treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Secondary | Part A: Change From Baseline in Left Ventricular Ejection Fraction (LVEF) | Baseline is defined as the last non-missing value in date/time recorded before the first dose of MYK-224. Participants with data available at the timepoint were included in the analysis. | Efficacy/PD A population included All enrolled A participants who receive at least 1 dose of MYK-224 and have primary or secondary endpoint data, including a baseline value and at least 1 post-baseline value Pre-specified to be analyzed for Part A. | Posted | | Mean | Standard Deviation | percentage | | Baseline and until treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Secondary | Part A: Aggregate Mean of Left Ventricular Outflow Tract (LVOT) Peak Gradient (Post-Exercise, Resting, and Valsalva) | | Efficacy/PD A population included All enrolled A participants who receive at least 1 dose of MYK-224 and have primary or secondary endpoint data, including a baseline value and at least 1 post-baseline value Pre-specified to be analyzed for Part A. | Posted | | Mean | Standard Deviation | mmHg | | Treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Secondary | Part A: Change From Baseline in Left Ventricular Outflow Tract (LVOT) Peak Gradient (Post-Exercise, Resting, and Valsalva) | Baseline is defined as the last non-missing value in date/time recorded before the first dose of MYK-224. Participants with data available at the timepoint were included in the analysis. | Efficacy/PD A population included All enrolled A participants who receive at least 1 dose of MYK-224 and have primary or secondary endpoint data, including a baseline value and at least 1 post-baseline value Pre-specified to be analyzed for Part A. | Posted | | Mean | Standard Deviation | mmHg | | Treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Secondary | Part A: Percentage of Participants With Resting Left Ventricular Outflow Tract (LVOT) Peak Gradient < 30 mmHg and Valsalva LVOT Peak Gradient < 50 mmHg | Baseline is defined as the last non-missing value in date/time recorded before the first dose of MYK-224. 90%CIs are calculated based on Normal approximation. | Efficacy/PD A population included All enrolled A participants who receive at least 1 dose of MYK-224 and have primary or secondary endpoint data, including a baseline value and at least 1 post-baseline value Pre-specified to be analyzed for Part A. | Posted | | Number | 90% Confidence Interval | percentage of participants | | Baseline and until treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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| Secondary | Part A: Plasma Concentration of MYK-224 | Blood samples were collected to assess plasma concentration of MYK-224. | Evaluable PK Analysis Population - Part A included All enrolled A participants who received at least 1 dose of MYK-224 and have at least 1 evaluable PK concentration. Pre-specified to be analyzed for Part A. | Posted | | Geometric Mean | Geometric Coefficient of Variation | ng/mL | | Pre-dose and 1 hour post-dose end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks) | | | | ID | Title | Description |
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| OG000 | Part A - Dose Treatment - MYK-224 | Participants with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction and who took or did not take Beta blockers at or before baseline received MYK-224. Dosing consisted of a once-daily oral dose in the form of tablets. All participants began at an initial dose of 5 mg of MYK-224, followed by up-or down-titration to doses between 2.5mg and 50mg. Dose titration was based on TTE measures taken at 3 weeks (at least 21 days) post-initiation of each new dose. Participants were to repeat this 4-week period as needed to identify their individual target dose. Once the individual dose was identified, participants were to continue dosing until the following 2 criteria were met: 6 weeks (at least 42 days) total dosing at their target dose (including the 4-week titration phase, if applicable) and an overall minimum total treatment period of 12 weeks (at least 84 days) at any dose or combination of doses. |
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