A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy

Phase 1

Completed

• Conditions: Becker Muscular Dystrophy
• Interventions: Drug: Sevasemten

• Registration Number: NCT05160415
• Lead Sponsor: Edgewise Therapeutics, Inc.

Brief Summary

The ARCH study is 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.

Detailed Description

This open-label study will evaluate 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 will receive sevasemten. On-site visits will occur approximately monthly for the first 12 months, followed by every 3 months to assess safety and measures of function. This study will have a 24 month treatment period, followed by a 4 week follow-up period.

Study Timeline

• Study First Submitted: 2021-12-02
• Study First Submitted QC: 2021-12-15
• Study First Posted: 2021-12-16
• Study Start: 2021-12-28
• Primary Completion: 2024-03-01
• Study Completion: 2024-03-01
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-02
• Last Update Posted: 2024-12-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 12

Inclusion Criteria

1. Participants who have completed Study EDG-5506-001.

2. Participants who were not from Study EDG-5506-001 must meet the following:

   1. Male sex at birth and aged 18 to 55 years inclusive at time of consent.
   2. Documented dystrophin mutation with phenotype consistent with BMD.
   3. Ambulatory at Screening (defined as ability to complete 100 meter [m] timed test, with or without assistance).
   4. Body weight ≥ 50 kg at the Screening visit.
   5. Body mass index (BMI) between 20 and 34 kg/m2 inclusive.

Exclusion Criteria

1. Receipt of oral corticosteroids for >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.
2. Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers.
3. 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.
4. 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment	Sevasemten	Drug: Sevasemten

Primary Outcome Measures

Name	Time	Method
Incidence of AEs in those treated with sevasemten	25 Months
Frequency of AEs in those treated with sevasemten	25 Months
Severity of AEs in those treated with sevasemten	25 Months

Secondary Outcome Measures

Name	Time	Method
Number of participants with changes in vital signs	24 Months
Number of participants with changes in FVC	24 Months	Assessed by spirometry
Number of participants with changes in hematology	24 Months
Number of participants with changes in ECG QT Interval	24 Months
Number of participants with changes in urinalysis	24 Months
Number of participants with changes in physical examination	24 Months
Incidence of treatment-emergent abnormal clinical chemistry test results	24 Months
Incidence of treatment-emergent abnormal coagulation test results	24 Months
Number of participants with changes in FEV1	24 Months	As assessed by spirometry
Incidence of treatment-emergent abnormal urinalysis test results	24 Months
Number of participants with changes in clinical chemistry	24 Months
Number of participants with changes in ECG QTc Interval	24 Months
Incidence of treatment-emergent abnormal hematology test results	24 Months
Number of participants with changes in coagulation	24 Months
Number of participants with changes in ECG PR Interval	24 Months
Number of participants with changes in ECG QRS Interval	24 Months

Trial Locations

Locations (1)

Rare Disease Research; 🇺🇸 Atlanta, Georgia, United States