Phase 1
N=12
A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy
Becker Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT05160415 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Jun 2025
Primary outcome: Primary: Percentage of Participants Treated With Sevasemten Experiencing AEs — 100 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Sevasemten (Drug)
- Age
- Adult · 18+ yrs
- Sex
- Male
- Sponsor
- Edgewise Therapeutics, Inc.
- Primary completion
- Mar 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants Treated With Sevasemten Experiencing AEs |
100 | — |
| PRIMARY Frequency of AEs in Those Treated With Sevasemten |
95 | — |
| PRIMARY Number of Participants Treated With Sevasemten With AEs by Maximum Severity |
10; 2; 0 | — |
| SECONDARY Percentage of Participants Experiencing Treatment-Emergent Abnormal Clinical Chemistry Test Results |
— | — |
| SECONDARY Percentage of Participants Experiencing Treatment-Emergent Abnormal Hematology Test Results |
— | — |
| SECONDARY Percentage of Participants Experiencing Treatment-Emergent Abnormal Coagulation Test Results |
— | — |
| SECONDARY Percentage of Participants Experiencing Treatment-Emergent Abnormal Urinalysis Test Results |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Clinical Chemistry |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Hematology |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Coagulation |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Urinalysis |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Vital Signs |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Physical and Neurological Examinations |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in ECG PR Interval |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in ECG QRS Interval |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in ECG QT Interval |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in ECG QTc Interval |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Forced Vital Capacity (FVC) |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Forced Expiratory Volume in 1 Second (FEV1) |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Cardiac Function as Assessed by Echocardiogram |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Columbia Suicide Severity Rating Scale (C-SSRS) |
— | — |
| SECONDARY Plasma Sevasemten (EDG-5506) Concentrations at Sample Timepoints |
0; 3.671; 23.19; 21.68; 22.57; 21.60 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- Participants who have completed Study EDG-5506-001.
- Participants who were not from Study EDG-5506-001 must meet the following:
- Male sex at birth and aged 18 to 55 years inclusive at time of consent.
- Documented dystrophin mutation with phenotype consistent with BMD.
- Ambulatory at Screening (defined as ability to complete 100 meter [m] timed test, with or without assistance).
- Body weight ≥ 50 kg at the Screening visit.
- Body mass index (BMI) between 20 and 34 kg/m2 inclusive.
- Female sexual partners of male participants must use highly effective contraception ( 5 days in the previous 6 months at a dose of >5 mg equivalent per day. Lower oral doses or inhaled/intranasal steroids are permitted.
- Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers.
- Participation in any other investigational drug study or use of use of an investigational drug within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.
- Participants who are unlikely to comply with the study protocol or, in the opinion of the Investigator, would not be a suitable candidate for participation in the study.
- Medical history or other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory result or abnormality that may increase the risk of study participation or, in the Investigator's judgment, make the participant inappropriate for the study. Includes venous access that would be too difficult to facilitate repeated blood sampling.
Data sourced from ClinicalTrials.gov (NCT05160415). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.