Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Medpace, Inc. | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
The ARCH study was an open-label, single-center, Phase 1b study of sevasemtem (EDG-5506) to assess the safety and pharmacokinetics (PK) of sevasemten in adults with Becker muscular dystrophy (BMD).
Sevasemten is an investigational product intended to protect and improve function of dystrophic muscle fibers.
This open-label study evaluated the safety, tolerability, and pharmacokinetics (PK) of sevasemten in participants with BMD who completed the first-in-human study, EDG-5506-001, as well as additional (treatment-naïve) participants from outside the EDG-5506-001 study to meet the target sample size.
All participants received sevasemten. This study had a 24 month treatment period, followed by an optional 4 week follow-up period. On-site visits occurred approximately monthly for the first 12 months, followed by every 3 months to assess safety and measures of function.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment | Experimental | Drug: Sevasemten |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sevasemten | Drug | Daily oral dose of 10 mg daily until Visit 8 (Day 57), followed by 15 mg daily until Visit 13 (Month 6), followed by 20 mg until Visit 21 (Month 15), followed by 10 mg daily to Visit 27 (Month 24). |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Treated With Sevasemten Experiencing AEs | An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The numerator of the percentage is the number of participants experiencing at least one AE after first dose of study drug up to 25 months. | From first dose of study drug to 25 months |
| Frequency of AEs in Those Treated With Sevasemten | An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The endpoint is the cumulative total number of AEs occurring after first dose of study drug up to 25 months among participants who received at least one dose of study drug. | From first dose of study drug to 25 months |
| Number of Participants Treated With Sevasemten With AEs by Maximum Severity | An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The severity of an AE is graded, according to the study protocol definitions of AE severity/intensity, as "mild", "moderate" or "severe". Participants who reported multiple AEs are counted only once at the highest severity reported. | From first dose of study drug to 25 months |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Experiencing Treatment-Emergent Abnormal Clinical Chemistry Test Results | Treatment-emergent defined as any event that occurs after the start of study drug or was present at baseline and worsened after taking study drug. The numerator of the percentage is the number of participants experiencing at least one treatment-emergent abnormal clinical chemistry test result after first dose of study drug up to 25 months. |
Not provided
Inclusion Criteria:
Participants who have completed Study EDG-5506-001.
Participants who were not from Study EDG-5506-001 must meet the following:
Female sexual partners of male participants must use highly effective contraception (<1% failure rate per year) through 6 months after last dose.
Capable of giving signed informed consent.
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Rare Disease Research | Atlanta | Georgia | 30329 | United States |
Not provided
| Label | URL |
|---|---|
| Sponsor Website | View source |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Treatment | Drug: Sevasemten Sevasemten: Daily oral dose of 10 mg daily until Visit 8 (Day 57), followed by 15 mg daily until Visit 13 (Month 6), followed by 20 mg until Visit 21 (Month 15), followed by 10 mg daily to Visit 27 (Month 24). |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Participants who received at least one dose of study drug.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Treatment | Drug: Sevasemten Sevasemten: Daily oral dose of 10 mg daily until Visit 8 (Day 57), followed by 15 mg daily until Visit 13 (Month 6), followed by 20 mg until Visit 21 (Month 15), followed by 10 mg daily to Visit 27 (Month 24). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants Treated With Sevasemten Experiencing AEs | An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The numerator of the percentage is the number of participants experiencing at least one AE after first dose of study drug up to 25 months. | Participants who received at least one dose of study drug. | Posted | Number | percentage of participants | From first dose of study drug to 25 months |
|
From first dose of study drug to 25 months.
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Treatment | Drug: Sevasemten Sevasemten: Daily oral dose of 10 mg daily until Visit 8 (Day 57), followed by 15 mg daily until Visit 13 (Month 6), followed by 20 mg until Visit 21 (Month 15), followed by 10 mg daily to Visit 27 (Month 24). |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| COVID-19 | Infections and infestations | MedDRA 24.1 | Non-systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Joanne Donovan MD PhD, Chief Medical Officer | Edgewise Therapeutics, Inc. | 720-262-7002 | studies@edgewisetx.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 8, 2023 | Mar 27, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 9, 2024 | Mar 27, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| From first dose of study drug to 25 months |
| Percentage of Participants Experiencing Treatment-Emergent Abnormal Hematology Test Results | Treatment-emergent defined as any event that occurs after the start of study drug or was present at baseline and worsened after taking study drug. The numerator of the percentage is the number of participants experiencing at least one treatment-emergent abnormal hematology test result after first dose of study drug up to 25 months. | From first dose of study drug to 25 months |
| Percentage of Participants Experiencing Treatment-Emergent Abnormal Coagulation Test Results | Treatment-emergent defined as any event that occurs after the start of study drug or was present at baseline and worsened after taking study drug. The numerator of the percentage is the number of participants experiencing at least one treatment-emergent abnormal coagulation test result after first dose of study drug up to 25 months. | From first dose of study drug to 25 months |
| Percentage of Participants Experiencing Treatment-Emergent Abnormal Urinalysis Test Results | Treatment-emergent defined as any event that occurs after the start of study drug or was present at baseline and worsened after taking study drug. The numerator of the percentage is the number of participants experiencing at least one treatment-emergent abnormal urinalysis test result after first dose of study drug up to 25 months. | From first dose of study drug to 25 months |
| Number of Participants With Clinically Significant Changes in Clinical Chemistry | Refer to Protocol for list of clinical chemistry tests that were performed. Clinically significant changes in clinical chemistry are defined as adverse events related to clinical chemistry tests or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in Hematology | Refer to Protocol for list of hematology tests that were performed. Clinically significant changes in hematology are defined as adverse events related to hematology tests or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in Coagulation | Refer to Protocol for list of coagulation tests that were performed. Clinically significant changes in coagulation are defined as adverse events related to coagulation tests or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in Urinalysis | Refer to Protocol for list of urinalysis tests that were performed. Clinically significant changes in urinalysis are defined as adverse events related to urinalysis tests or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in Vital Signs | Supine systolic and diastolic blood pressure, pulse rate, respiratory rate, and temperature were measured. Clinically significant changes in vital signs are defined as adverse events related to vital signs or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in Physical and Neurological Examinations | A physical examination included head, ears, eyes, nose, mouth, skin, heart and lung, lymph nodes, and gastrointestinal and musculoskeletal systems. A neurological examination was also conducted to include upper and lower limb tone, power, reflexes, and examination of cranial nerves II-XII (excluding ophthalmoscopy). Clinically significant changes in physical and neurological examinations are defined as adverse events related to physical and neurological examinations or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in ECG PR Interval | Triplicate 12-lead electrocardiogram (ECG) parameters were obtained using an ECG machine that automatically measured PR interval. Clinically significant changes in ECG PR interval are defined as adverse events related to ECG PR interval or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in ECG QRS Interval | Triplicate 12-lead electrocardiogram (ECG) parameters were obtained using an ECG machine that automatically measured QRS interval. Clinically significant changes in ECG QRS interval are defined as adverse events related to ECG QRS interval or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in ECG QT Interval | Triplicate 12-lead electrocardiogram (ECG) parameters were obtained using an ECG machine that automatically measured QT interval. Clinically significant changes in ECG QT interval are defined as adverse events related to ECG QT interval or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in ECG QTc Interval | Triplicate 12-lead electrocardiogram (ECG) parameters were obtained using an ECG machine that automatically measured QT corrected (QTc) Interval. QT corrected by Bazett's formula (QTcB) and QT corrected by Fridericia's formula (QTcF) were both recorded. Clinically significant changes in ECG QTc interval are defined as adverse events related to ECG QTc interval or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in Forced Vital Capacity (FVC) | Assessed by spirometry. Clinically significant changes in FVC are defined as adverse events related to FVC or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in Forced Expiratory Volume in 1 Second (FEV1) | Assessed by spirometry. Clinically significant changes in FEV1 are defined as adverse events related to FEV1 or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in Cardiac Function as Assessed by Echocardiogram | Echocardiographic examinations were performed by a qualified individual (sonographer) at the site to evaluate left-ventricular systolic and diastolic function, geometry, and mass, as well as left-atrial and right-ventricular function and geometry via two-dimensional, doppler, and/or speckle-tracking imaging techniques. Valvular competence, including presence or absence of regurgitation, was evaluated and quantified, while overall cardiac health was qualitatively evaluated (e.g., presence/absence of pericardiac effusion). Clinically significant changes in cardiac function are defined as adverse events related to cardiac function as assessed by echocardiogram or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | From first dose of study drug to 24 months |
| Number of Participants With Clinically Significant Changes in Columbia Suicide Severity Rating Scale (C-SSRS) | The C-SSRS is a questionnaire used for suicide assessment. The assessment includes "yes" or "no" responses for 6 questions. Questions 1-5 are related to suicidal ideation, including: 1=Wish to be Dead, 2=Non-specific Active Suicidal Thoughts, 3=Active Suicidal Ideation with Any Methods (Not Plan) without Intent to Act, 4=Active Suicidal Ideation with Some Intent to Act, without Specific Plan, 5=Active Suicidal Ideation with Specific Plan and Intent. Question 6 is related to suicidal behavior and asks about actual attempts. Numeric ratings were provided for severity of ideation (if present), from 1 to 5, with 5 being the most severe. For this study, clinically significant changes in C-SSRS are defined as responses of "yes" to suicidal ideation or suicidal behavior item as measured by C-SSRS or investigator identified results reported from first dose of study drug to 24 months. | From first dose of study drug to 24 months |
| Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints | Values below the limit of quantitation (BLQ) of 0.500 ng/mL were treated as 0 before the first quantifiable concentration and as missing elsewhere. Single PK concentration was collected at each visit, except for Day 1. For treatment-naive participants, Day 1 was a serial collection at pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, and 24 hours post-dose. The last timepoint was on Day 2. The dose on Day 2 was administered only after the 24 hour timepoint. For treatment experienced participants, Day 1 was pre-dose only. Days 29 and 57 have window of +/- 3 days; Months 3-24 have window of +/- 5 days. | Pre-dose; 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, and 24 hours post-dose; 29 and 57 days post-dose; 3, 4, 6, 7, 8, 10, 12, 15, 18, 21, and 24 months post-dose. |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Height | Mean | Standard Deviation | cm |
|
| Weight | Mean | Standard Deviation | kg |
|
|
|
| Primary | Frequency of AEs in Those Treated With Sevasemten | An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The endpoint is the cumulative total number of AEs occurring after first dose of study drug up to 25 months among participants who received at least one dose of study drug. | Participants who received at least one dose of study drug. | Posted | Number | Count of Events | From first dose of study drug to 25 months |
|
|
|
| Primary | Number of Participants Treated With Sevasemten With AEs by Maximum Severity | An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The severity of an AE is graded, according to the study protocol definitions of AE severity/intensity, as "mild", "moderate" or "severe". Participants who reported multiple AEs are counted only once at the highest severity reported. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 25 months |
|
|
|
| Secondary | Percentage of Participants Experiencing Treatment-Emergent Abnormal Clinical Chemistry Test Results | Treatment-emergent defined as any event that occurs after the start of study drug or was present at baseline and worsened after taking study drug. The numerator of the percentage is the number of participants experiencing at least one treatment-emergent abnormal clinical chemistry test result after first dose of study drug up to 25 months. | Participants who received at least one dose of study drug. | Posted | Number | percentage of participants | From first dose of study drug to 25 months |
|
|
|
| Secondary | Percentage of Participants Experiencing Treatment-Emergent Abnormal Hematology Test Results | Treatment-emergent defined as any event that occurs after the start of study drug or was present at baseline and worsened after taking study drug. The numerator of the percentage is the number of participants experiencing at least one treatment-emergent abnormal hematology test result after first dose of study drug up to 25 months. | Participants who received at least one dose of study drug. | Posted | Number | percentage of participants | From first dose of study drug to 25 months |
|
|
|
| Secondary | Percentage of Participants Experiencing Treatment-Emergent Abnormal Coagulation Test Results | Treatment-emergent defined as any event that occurs after the start of study drug or was present at baseline and worsened after taking study drug. The numerator of the percentage is the number of participants experiencing at least one treatment-emergent abnormal coagulation test result after first dose of study drug up to 25 months. | Participants who received at least one dose of study drug. | Posted | Number | percentage of participants | From first dose of study drug to 25 months |
|
|
|
| Secondary | Percentage of Participants Experiencing Treatment-Emergent Abnormal Urinalysis Test Results | Treatment-emergent defined as any event that occurs after the start of study drug or was present at baseline and worsened after taking study drug. The numerator of the percentage is the number of participants experiencing at least one treatment-emergent abnormal urinalysis test result after first dose of study drug up to 25 months. | Participants who received at least one dose of study drug. | Posted | Number | percentage of participants | From first dose of study drug to 25 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Clinical Chemistry | Refer to Protocol for list of clinical chemistry tests that were performed. Clinically significant changes in clinical chemistry are defined as adverse events related to clinical chemistry tests or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Hematology | Refer to Protocol for list of hematology tests that were performed. Clinically significant changes in hematology are defined as adverse events related to hematology tests or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Coagulation | Refer to Protocol for list of coagulation tests that were performed. Clinically significant changes in coagulation are defined as adverse events related to coagulation tests or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Urinalysis | Refer to Protocol for list of urinalysis tests that were performed. Clinically significant changes in urinalysis are defined as adverse events related to urinalysis tests or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Vital Signs | Supine systolic and diastolic blood pressure, pulse rate, respiratory rate, and temperature were measured. Clinically significant changes in vital signs are defined as adverse events related to vital signs or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Physical and Neurological Examinations | A physical examination included head, ears, eyes, nose, mouth, skin, heart and lung, lymph nodes, and gastrointestinal and musculoskeletal systems. A neurological examination was also conducted to include upper and lower limb tone, power, reflexes, and examination of cranial nerves II-XII (excluding ophthalmoscopy). Clinically significant changes in physical and neurological examinations are defined as adverse events related to physical and neurological examinations or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in ECG PR Interval | Triplicate 12-lead electrocardiogram (ECG) parameters were obtained using an ECG machine that automatically measured PR interval. Clinically significant changes in ECG PR interval are defined as adverse events related to ECG PR interval or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in ECG QRS Interval | Triplicate 12-lead electrocardiogram (ECG) parameters were obtained using an ECG machine that automatically measured QRS interval. Clinically significant changes in ECG QRS interval are defined as adverse events related to ECG QRS interval or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in ECG QT Interval | Triplicate 12-lead electrocardiogram (ECG) parameters were obtained using an ECG machine that automatically measured QT interval. Clinically significant changes in ECG QT interval are defined as adverse events related to ECG QT interval or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in ECG QTc Interval | Triplicate 12-lead electrocardiogram (ECG) parameters were obtained using an ECG machine that automatically measured QT corrected (QTc) Interval. QT corrected by Bazett's formula (QTcB) and QT corrected by Fridericia's formula (QTcF) were both recorded. Clinically significant changes in ECG QTc interval are defined as adverse events related to ECG QTc interval or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Forced Vital Capacity (FVC) | Assessed by spirometry. Clinically significant changes in FVC are defined as adverse events related to FVC or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Forced Expiratory Volume in 1 Second (FEV1) | Assessed by spirometry. Clinically significant changes in FEV1 are defined as adverse events related to FEV1 or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Cardiac Function as Assessed by Echocardiogram | Echocardiographic examinations were performed by a qualified individual (sonographer) at the site to evaluate left-ventricular systolic and diastolic function, geometry, and mass, as well as left-atrial and right-ventricular function and geometry via two-dimensional, doppler, and/or speckle-tracking imaging techniques. Valvular competence, including presence or absence of regurgitation, was evaluated and quantified, while overall cardiac health was qualitatively evaluated (e.g., presence/absence of pericardiac effusion). Clinically significant changes in cardiac function are defined as adverse events related to cardiac function as assessed by echocardiogram or investigator identified results reported from first dose of study drug up to 24 months. Only clinically significant changes were counted. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Columbia Suicide Severity Rating Scale (C-SSRS) | The C-SSRS is a questionnaire used for suicide assessment. The assessment includes "yes" or "no" responses for 6 questions. Questions 1-5 are related to suicidal ideation, including: 1=Wish to be Dead, 2=Non-specific Active Suicidal Thoughts, 3=Active Suicidal Ideation with Any Methods (Not Plan) without Intent to Act, 4=Active Suicidal Ideation with Some Intent to Act, without Specific Plan, 5=Active Suicidal Ideation with Specific Plan and Intent. Question 6 is related to suicidal behavior and asks about actual attempts. Numeric ratings were provided for severity of ideation (if present), from 1 to 5, with 5 being the most severe. For this study, clinically significant changes in C-SSRS are defined as responses of "yes" to suicidal ideation or suicidal behavior item as measured by C-SSRS or investigator identified results reported from first dose of study drug to 24 months. | Participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug to 24 months |
|
|
|
| Secondary | Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints | Values below the limit of quantitation (BLQ) of 0.500 ng/mL were treated as 0 before the first quantifiable concentration and as missing elsewhere. Single PK concentration was collected at each visit, except for Day 1. For treatment-naive participants, Day 1 was a serial collection at pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, and 24 hours post-dose. The last timepoint was on Day 2. The dose on Day 2 was administered only after the 24 hour timepoint. For treatment experienced participants, Day 1 was pre-dose only. Days 29 and 57 have window of +/- 3 days; Months 3-24 have window of +/- 5 days. | Participants who received at least one dose of study drug and have a sufficient PK profile to derive at least one PK parameter. | Posted | Mean | Standard Deviation | ng/mL | Pre-dose; 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, and 24 hours post-dose; 29 and 57 days post-dose; 3, 4, 6, 7, 8, 10, 12, 15, 18, 21, and 24 months post-dose. |
|
|
|
| 0 |
| 12 |
| 0 |
| 12 |
| 12 |
| 12 |
| Influenza | Infections and infestations | MedDRA 24.1 | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 24.1 | Non-systematic Assessment |
|
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 24.1 | Non-systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA 24.1 | Non-systematic Assessment |
|
| Gastroenteritis viral | Infections and infestations | MedDRA 24.1 | Non-systematic Assessment |
|
| Herpes zoster | Infections and infestations | MedDRA 24.1 | Non-systematic Assessment |
|
| Pharyngitis streptococcal | Infections and infestations | MedDRA 24.1 | Non-systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 24.1 | Non-systematic Assessment |
|
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 24.1 | Non-systematic Assessment |
|
| Arthropod sting | Injury, poisoning and procedural complications | MedDRA 24.1 | Non-systematic Assessment |
|
| Concussion | Injury, poisoning and procedural complications | MedDRA 24.1 | Non-systematic Assessment |
|
| Hand fracture | Injury, poisoning and procedural complications | MedDRA 24.1 | Non-systematic Assessment |
|
| Meniscus injury | Injury, poisoning and procedural complications | MedDRA 24.1 | Non-systematic Assessment |
|
| Post-traumatic pain | Injury, poisoning and procedural complications | MedDRA 24.1 | Non-systematic Assessment |
|
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA 24.1 | Non-systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Somnolence | Nervous system disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Temporomandibular joint syndrome | Musculoskeletal and connective tissue disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Epiploic appendagitis | Gastrointestinal disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Gait inability | General disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Non-cardiac chest pain | General disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Euphoric mood | Psychiatric disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Panic attack | Psychiatric disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Hyperthyroidism | Endocrine disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Seasonal allergy | Immune system disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Testicle adenoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.1 | Non-systematic Assessment |
|
| Hiccups | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Snoring | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Non-systematic Assessment |
|
| Flushing | Vascular disorders | MedDRA 24.1 | Non-systematic Assessment |
|
Not provided
Not provided
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| Title | Measurements |
|---|
|
|
| 0.5 hours |
|
|
| 1 hour |
|
|
| 1.5 hours |
|
|
| 2 hours |
|
|
| 3 hours |
|
|
| 4 hours |
|
|
| 6 hours |
|
|
| 24 hours |
|
|
| 29 days |
|
|
| 57 days |
|
|
| 3 months |
|
|
| 4 months |
|
|
| 6 months |
|
|
| 7 months |
|
|
| 8 months |
|
|
| 10 months |
|
|
| 12 months |
|
|
| 15 months |
|
|
| 18 months |
|
|
| 21 months |
|
|
| 24 months |
|
|