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

May 2, 2026 updated by: Edgewise Therapeutics, Inc.

An Open-Label Extension Study to Assess the Long-term Effect of EDG-5506 on Safety, Biomarkers, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy

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

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Detailed Description

This is an open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of sevasemten. EDG-5506-203 MESA will provide continued access to sevasemten treatment to participants with Becker muscular dystrophy who were previously enrolled in EDG-5506-002 ARCH, completed EDG-5506-201 CANYON and GRAND CANYON, or completed EDG-5506-202 DUNE.

Study Type

Interventional

Enrollment (Estimated)

260

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
    • Belgium
      • Ghent, Belgium, Belgium, 9000
        • University Hospital Gent
      • Leuven, Belgium, Belgium, 3000
        • Universitaire Ziekenhuizen Leuven
      • Liége, Belgium, Belgium, 4000
        • Centre Hospitalier Regional de la Citadelle
  • Denmark
      • Copenhagen, Denmark, 2100
        • Rigshospitalet
  • France
    • France
      • Marseille, France, France, 13005
        • Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
      • Nantes, France, France, 44093
        • CHU de NANTES
      • Nice, France, France, 06001
        • CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
      • Paris, France, France, 75013
        • AP-HP Hopital Pitie-Salpetriere
  • Germany
    • Germany
      • Munich, Germany, Germany, 80336
        • Klinikum der Ludwig-Maximilians-Universitaet Muenchen
  • Israel
      • Jerusalem, Israel, 91240
        • Hadassah University Hospital
  • Italy
    • Italy
      • Milan, Italy, Italy, 20122
        • Fondazione IRCCS Ca'Granda Ospedale Maggiore
      • Padova, Italy, Italy, 35128
        • Azienda Ospedale - Università Padova
      • Rome, Italy, Italy, 00168
        • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
  • Netherlands
      • Leiden, Netherlands, 2333 ZA
        • Leids Universitair Medisch Centrum
  • Spain
    • Spain
      • Barcelona, Spain, Spain, 08035
        • Hospital Universitario Vall d'Hebron
      • Barcelona, Spain, Spain, 08907
        • Hospital Universitario de Bellvitge
      • Donostia / San Sebastian, Spain, Spain, 20014
        • Hospital Universitario Donostia
      • Valencia, Spain, Spain, 46026
        • Hospital Universitari i Politècnic La Fe
  • United Kingdom
      • London, United Kingdom, SW17 0QT
        • St. George's University Hospitals NHS Foundation Trust
      • London, United Kingdom, NW1 2PG
        • University College London Hospital
      • Newcastle, United Kingdom, NE7 7DN
        • Newcastle Freeman Hospital
    • United Kingdom
      • Salford, United Kingdom, United Kingdom, M68HD
        • Salford Royal Hospital
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • La Jolla, California, United States, 92037
        • UC San Diego
      • Los Angeles, California, United States, 90095
        • UCLA Medical Center
      • Orange, California, United States, 92868
        • UC Irvine Medical Center
      • Palo Alto, California, United States, 94304
        • Stanford Neuroscience Health Center
      • Sacramento, California, United States, 95817
        • UC Davis Medical Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • UC Denver
    • Florida
      • Gainesville, Florida, United States, 32611
        • University of Florida
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Rare Disease Research
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University School of Medicine
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Kennedy Krieger Institute
    • Massachusetts
      • Worcester, Massachusetts, United States, 01605
        • University of Massachusetts Memorial Medical Center
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • New York
      • Rochester, New York, United States, 14642
        • University of Rochester Medical Center
    • North Carolina
      • Hillsborough, North Carolina, United States, 27278
        • Rare Disease Research, LLC NC
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • University of Cincinnati Gardner Neuroscience Institute
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania
    • Texas
      • Austin, Texas, United States, 78759
        • National Neuromuscular Research Institute
      • Denton, Texas, United States, 76208
        • Neurology Rare Disease Center
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth University Health

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

1. 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])

Key Exclusion Criteria:

  1. 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.
  2. Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers.
  3. Receipt of an investigational drug other than sevasemten within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.

3. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Treatment
Drug: Sevasemten
Drug: Sevasemten
Sevasemten is administered orally once per day

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of adverse events in those treated with sevasemten
Time Frame: 55 Months
55 Months
Severity of adverse events in those treated with sevasemten
Time Frame: 55 Months
55 Months

Secondary Outcome Measures

Outcome Measure
Time Frame
Incidence of treatment-emergent abnormal clinical chemistry laboratory test results
Time Frame: 54 Months
54 Months
Incidence of treatment-emergent abnormal hematology laboratory test results
Time Frame: 54 Months
54 Months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Edgewise Therapeutics, Inc.

Collaborators

Medpace, Inc.

Investigators

  • Study Chair: Joanne M. Donovan, MD, PhD, Edgewise Therapeutics, Inc.
  • Study Chair: Roxana D. Dreghici, MD, Edgewise Therapeutics, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 2, 2023

Primary Completion (Estimated)

February 1, 2031

Study Completion (Estimated)

February 1, 2031

Study Registration Dates

First Submitted

September 27, 2023

First Submitted That Met QC Criteria

September 27, 2023

First Posted (Actual)

October 4, 2023

Study Record Updates

Last Update Posted (Actual)

May 6, 2026

Last Update Submitted That Met QC Criteria

May 2, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EDG-5506-203

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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