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

NCT05160415 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: EDG-5506-002
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Becker Muscular Dystrophy

Keywords

Becker Muscular Dystrophy

Outcome Measures

Primary Outcomes (3)

• 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

Secondary Outcomes (19)

• Percentage of Participants Experiencing Treatment-Emergent Abnormal Clinical Chemistry Test Results

  From first dose of study drug to 25 months

• Percentage of Participants Experiencing Treatment-Emergent Abnormal Hematology Test Results

  From first dose of study drug to 25 months

• Percentage of Participants Experiencing Treatment-Emergent Abnormal Coagulation Test Results

  From first dose of study drug to 25 months

• Percentage of Participants Experiencing Treatment-Emergent Abnormal Urinalysis Test Results

  From first dose of study drug to 25 months

• Number of Participants With Clinically Significant Changes in Clinical Chemistry

  From first dose of study drug to 24 months

Study Arms (1)

Treatment

EXPERIMENTAL

Drug: Sevasemten

Drug: Sevasemten

Interventions

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).

Also known as: EDG-5506

Treatment

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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 (\<1% failure rate per year) through 6 months after last dose.
• Capable of giving signed informed consent.

You may not qualify if:

• Any clinically significant changes during or following the completion of Study EDG 5506-001 that would affect the potential safety of the participant to receive EDG 5506.
• Cardiac echocardiogram ejection fraction \<45% or New York Heart Association (NYHA) Class III or Class IV.
• Baseline 12-lead electrocardiogram (ECG) that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results.
• Forced vital capacity (FVC) predicted \<65% or using daytime (mechanical or noninvasive) ventilatory support.
• Moderate or severe renal or hepatic impairment (eGFR \<60 mL/min/1.73 m2).
• Positive test for hepatitis C antibody (unless negative HCV PCR), hepatitis B surface antigen, or human immunodeficiency virus (HIV) antibody at screening.
• History of substance abuse or dependency.
• 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.
• 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.

Sponsors & Collaborators

• Edgewise Therapeutics, Inc.: lead
• Medpace, Inc.: collaborator

Study Sites (1)

Rare Disease Research

Atlanta, Georgia, 30329, United States

Location

Related Links

• Sponsor Website

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Joanne Donovan MD PhD, Chief Medical Officer
• Organization: Edgewise Therapeutics, Inc.

studies@edgewisetx.com

720-262-7002

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 2, 2021
• First Posted: December 16, 2021
• Study Start: December 28, 2021
• Primary Completion: March 1, 2024
• Study Completion: March 1, 2024
• Last Updated: June 24, 2025
• Results First Posted: June 24, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)