Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy

NCT06066580 | Enrolling By Invitation Phase 2 DMC FDA Drug

• 1 other identifier: EDG-5506-203
• Study Type: interventional
• Target: 260
• Locations: 10 countries
• Sites: 46

Brief Summary

EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy

Conditions

Becker Muscular Dystrophy

Keywords

Becker Muscular Dystrophy

Outcome Measures

Primary Outcomes (2)

• Number of adverse events in those treated with sevasemten

  55 Months

• Severity of adverse events in those treated with sevasemten

  55 Months

Secondary Outcomes (2)

• Incidence of treatment-emergent abnormal clinical chemistry laboratory test results

  54 Months

• Incidence of treatment-emergent abnormal hematology laboratory test results

  54 Months

Study Arms (1)

Treatment

EXPERIMENTAL

Drug: Sevasemten

Drug: Sevasemten

Interventions

Sevasemten DRUG

Sevasemten is administered orally once per day

Treatment

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• \. Males with a diagnosis of BMD and participation in EDG-5506-002 ARCH, EDG-5506-201 CANYON and GRAND CANYON, or EDG-5506-202 DUNE. Participants are eligible if they complete the respective prior study visits as follows:
• EDG-5506-002 ARCH: Complete the final study Visit 27 \[Month 24\]; or, completion of the ET visit prior to Visit 27 \[Month 24\]
• EDG-5506-201 CANYON and GRAND CANYON: Complete the final study visit (Cohorts 1, 2, 4, and 5: Visit 12 \[Month 12\]; Cohort 6: Visit 11 \[Month 18\])
• EDG-5506-202 DUNE: Complete at least 36 weeks of open-label treatment (Visit 14 \[Week 52\])

You may not qualify if:

• Any clinically significant changes during or following participation in EDG-5506-002, EDG-5506-201, or EDG-5506-202 that would affect the potential safety of the participant to receive sevasemten.
• Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers.
• Receipt of an investigational drug other than sevasemten within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.
• \. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.

Sponsors & Collaborators

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

Study Sites (46)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

UC San Diego

La Jolla, California, 92037, United States

Location

UCLA Medical Center

Los Angeles, California, 90095, United States

Location

UC Irvine Medical Center

Orange, California, 92868, United States

Location

Stanford Neuroscience Health Center

Palo Alto, California, 94304, United States

Location

UC Davis Medical Center

Sacramento, California, 95817, United States

Location

UC Denver

Aurora, Colorado, 80045, United States

Location

University of Florida

Gainesville, Florida, 32611, United States

Location

Rare Disease Research

Atlanta, Georgia, 30329, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

University of Massachusetts Memorial Medical Center

Worcester, Massachusetts, 01605, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Rare Disease Research, LLC NC

Hillsborough, North Carolina, 27278, United States

Location

University of Cincinnati Gardner Neuroscience Institute

Cincinnati, Ohio, 45219, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

National Neuromuscular Research Institute

Austin, Texas, 78759, United States

Location

Neurology Rare Disease Center

Denton, Texas, 76208, United States

Location

Virginia Commonwealth University Health

Richmond, Virginia, 23298, United States

Location

University Hospital Gent

Ghent, Belgium, 9000, Belgium

Location

Universitaire Ziekenhuizen Leuven

Leuven, Belgium, 3000, Belgium

Location

Centre Hospitalier Régional de la Citadelle

Liége, Belgium, 4000, Belgium

Location

Rigshospitalet

Copenhagen, 2100, Denmark

Location

Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone

Marseille, France, 13005, France

Location

CHU de Nantes

Nantes, France, 44093, France

Location

CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires

Nice, France, 06001, France

Location

AP-HP Hopital Pitie-Salpetriere

Paris, France, 75013, France

Location

Klinikum der Ludwig-Maximilians-Universitaet Muenchen

Munich, Germany, 80336, Germany

Location

Hadassah University Hospital

Jerusalem, 91240, Israel

Location

Fondazione IRCCS Ca'Granda Ospedale Maggiore

Milan, Italy, 20122, Italy

Location

Azienda Ospedale - Università Padova

Padova, Italy, 35128, Italy

Location

Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

Rome, Italy, 00168, Italy

Location

Leids Universitair Medisch Centrum

Leiden, 2333 ZA, Netherlands

Location

Hospital Universitario Vall d'Hebron

Barcelona, Spain, 08035, Spain

Location

Hospital Universitario de Bellvitge

Barcelona, Spain, 08907, Spain

Location

Hospital Universitario Donostia

Donostia / San Sebastian, Spain, 20014, Spain

Location

Hospital Universitari i Politecnic La Fe

Valencia, Spain, 46026, Spain

Location

Salford Royal Hospital

Salford, United Kingdom, M68HD, United Kingdom

Location

University College London Hospital

London, NW1 2PG, United Kingdom

Location

St. George's University Hospitals NHS Foundation Trust

London, SW17 0QT, United Kingdom

Location

Newcastle Freeman Hospital

Newcastle, NE7 7DN, United Kingdom

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

Study Officials

• Joanne M. Donovan, MD, PhD

  Edgewise Therapeutics, Inc.

  STUDY CHAIR

• Roxana D. Dreghici, MD

  Edgewise Therapeutics, Inc.

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 27, 2023
• First Posted: October 4, 2023
• Study Start: November 2, 2023
• Primary Completion (Estimated): February 1, 2031
• Study Completion (Estimated): February 1, 2031
• Last Updated: May 6, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

IL (1); GB (4); BE (3); FR (4); IT (3); US (24); ES (4); DE (1); NL (1); DK (1)