Trial Outcomes & Findings for A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy (NCT NCT05160415)
NCT ID: NCT05160415
Last Updated: 2025-06-24
Results Overview
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.
COMPLETED
PHASE1
12 participants
From first dose of study drug to 25 months
2025-06-24
Participant Flow
Participant milestones
| Measure |
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).
|
|---|---|
|
Overall Study
STARTED
|
12
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
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).
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
3
|
Baseline Characteristics
A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy
Baseline characteristics by cohort
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Age, Continuous
|
32.9 years
STANDARD_DEVIATION 7.98 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=5 Participants
|
|
Height
|
175.37 cm
STANDARD_DEVIATION 6.747 • n=5 Participants
|
|
Weight
|
77.77 kg
STANDARD_DEVIATION 10.563 • n=5 Participants
|
PRIMARY outcome
Timeframe: From first dose of study drug to 25 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Percentage of Participants Treated With Sevasemten Experiencing AEs
|
100 percentage of participants
|
PRIMARY outcome
Timeframe: From first dose of study drug to 25 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Frequency of AEs in Those Treated With Sevasemten
|
95 Count of Events
|
PRIMARY outcome
Timeframe: From first dose of study drug to 25 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants Treated With Sevasemten With AEs by Maximum Severity
Moderate
|
2 Participants
|
|
Number of Participants Treated With Sevasemten With AEs by Maximum Severity
Severe
|
0 Participants
|
|
Number of Participants Treated With Sevasemten With AEs by Maximum Severity
Mild
|
10 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 25 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Percentage of Participants Experiencing Treatment-Emergent Abnormal Clinical Chemistry Test Results
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 25 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Percentage of Participants Experiencing Treatment-Emergent Abnormal Hematology Test Results
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 25 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Percentage of Participants Experiencing Treatment-Emergent Abnormal Coagulation Test Results
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 25 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Percentage of Participants Experiencing Treatment-Emergent Abnormal Urinalysis Test Results
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in Clinical Chemistry
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in Hematology
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in Coagulation
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in Urinalysis
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in Vital Signs
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in Physical and Neurological Examinations
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in ECG PR Interval
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in ECG QRS Interval
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in ECG QT Interval
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in ECG QTc Interval
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in Forced Vital Capacity (FVC)
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in Forced Expiratory Volume in 1 Second (FEV1)
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in Cardiac Function as Assessed by Echocardiogram
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose of study drug to 24 monthsPopulation: Participants who received at least one dose of study drug.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Number of Participants With Clinically Significant Changes in Columbia Suicide Severity Rating Scale (C-SSRS)
|
0 Participants
|
SECONDARY outcome
Timeframe: 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.Population: Participants who received at least one dose of study drug and have a sufficient PK profile to derive at least one PK parameter.
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.
Outcome measures
| Measure |
Treatment
n=12 Participants
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).
|
|---|---|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
Pre-dose
|
0 ng/mL
Standard Deviation 0
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
2 hours
|
21.60 ng/mL
Standard Deviation 18.013
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
3 hours
|
12.63 ng/mL
Standard Deviation 6.5033
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
4 hours
|
8.899 ng/mL
Standard Deviation 2.3685
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
6 hours
|
6.536 ng/mL
Standard Deviation 2.5716
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
8 months
|
137.7 ng/mL
Standard Deviation 55.542
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
12 months
|
141.7 ng/mL
Standard Deviation 60.399
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
18 months
|
78.20 ng/mL
Standard Deviation 34.885
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
21 months
|
69.33 ng/mL
Standard Deviation 26.315
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
0.25 hours
|
3.671 ng/mL
Standard Deviation 4.1099
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
0.5 hours
|
23.19 ng/mL
Standard Deviation 30.408
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
1 hour
|
21.68 ng/mL
Standard Deviation 15.091
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
1.5 hours
|
22.57 ng/mL
Standard Deviation 10.959
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
24 hours
|
4.000 ng/mL
Standard Deviation 1.5452
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
29 days
|
53.27 ng/mL
Standard Deviation 20.607
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
57 days
|
60.57 ng/mL
Standard Deviation 26.389
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
3 months
|
93.63 ng/mL
Standard Deviation 38.939
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
4 months
|
92.34 ng/mL
Standard Deviation 34.940
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
6 months
|
105.3 ng/mL
Standard Deviation 40.764
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
7 months
|
137.4 ng/mL
Standard Deviation 52.673
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
10 months
|
149.3 ng/mL
Standard Deviation 69.139
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
15 months
|
175.2 ng/mL
Standard Deviation 58.905
|
|
Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints
24 months
|
55.38 ng/mL
Standard Deviation 29.536
|
Adverse Events
Treatment
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Treatment
n=12 participants at risk
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).
|
|---|---|
|
Infections and infestations
COVID-19
|
41.7%
5/12 • From first dose of study drug to 25 months.
|
|
Infections and infestations
Influenza
|
25.0%
3/12 • From first dose of study drug to 25 months.
|
|
Infections and infestations
Nasopharyngitis
|
25.0%
3/12 • From first dose of study drug to 25 months.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
25.0%
3/12 • From first dose of study drug to 25 months.
|
|
Infections and infestations
Sinusitis
|
16.7%
2/12 • From first dose of study drug to 25 months.
|
|
Infections and infestations
Gastroenteritis viral
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Infections and infestations
Herpes zoster
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Infections and infestations
Pharyngitis streptococcal
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Injury, poisoning and procedural complications
Fall
|
33.3%
4/12 • From first dose of study drug to 25 months.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
25.0%
3/12 • From first dose of study drug to 25 months.
|
|
Injury, poisoning and procedural complications
Arthropod sting
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Injury, poisoning and procedural complications
Concussion
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Injury, poisoning and procedural complications
Hand fracture
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Injury, poisoning and procedural complications
Meniscus injury
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Injury, poisoning and procedural complications
Post-traumatic pain
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Nervous system disorders
Dizziness
|
33.3%
4/12 • From first dose of study drug to 25 months.
|
|
Nervous system disorders
Headache
|
25.0%
3/12 • From first dose of study drug to 25 months.
|
|
Nervous system disorders
Somnolence
|
25.0%
3/12 • From first dose of study drug to 25 months.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
33.3%
4/12 • From first dose of study drug to 25 months.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Musculoskeletal and connective tissue disorders
Temporomandibular joint syndrome
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
25.0%
3/12 • From first dose of study drug to 25 months.
|
|
Gastrointestinal disorders
Diarrhoea
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Gastrointestinal disorders
Dyspepsia
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Gastrointestinal disorders
Epiploic appendagitis
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Gastrointestinal disorders
Toothache
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
General disorders
Gait inability
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
General disorders
Non-cardiac chest pain
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Psychiatric disorders
Euphoric mood
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Psychiatric disorders
Panic attack
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Endocrine disorders
Hyperthyroidism
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Immune system disorders
Seasonal allergy
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Metabolism and nutrition disorders
Dehydration
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Testicle adenoma
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Respiratory, thoracic and mediastinal disorders
Snoring
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
|
Vascular disorders
Flushing
|
8.3%
1/12 • From first dose of study drug to 25 months.
|
Additional Information
Joanne Donovan MD PhD, Chief Medical Officer
Edgewise Therapeutics, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place