CClinicalTrials.gg
Enrolling by invitationNCT06066580MESAUpdated Sep 17, 2026

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

A Phase 2 interventional study of Sevasemten in Becker Muscular Dystrophy, sponsored by Edgewise Therapeutics, Inc.. Enrolling by invitation at 48 sites in 12 countries. Open to male participants. Per ClinicalTrials.gov, last updated 2026-09-17.

Sponsored by Edgewise Therapeutics, Inc. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
260
Allocation
Not applicable
Sex
Male
01

Study 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

Read the 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.

02

Conditions studied

  • Becker Muscular Dystrophy

Keywords

  • Becker Muscular Dystrophy
03

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
Male
Accepts healthy volunteers
No

Eligibility criteria

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.
  4. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
260 participants (estimated)

Study arms

  • Experimental
    Treatment

    Drug: Sevasemten

    Drug: Sevasemten

Interventions

  • DrugSevasemten

    Sevasemten is administered orally once per day

05

What researchers measure

Primary outcomes

  1. Number of adverse events in those treated with sevasemten

    Time frame: 55 Months

  2. Severity of adverse events in those treated with sevasemten

    Time frame: 55 Months

Secondary outcomes

  1. Incidence of treatment-emergent abnormal clinical chemistry laboratory test results

    Time frame: 54 Months

  2. Incidence of treatment-emergent abnormal hematology laboratory test results

    Time frame: 54 Months

06

Study locations

48 sites
  • Arkansas Children's Hospital
    Little Rock, Arkansas 72202, United States
  • UC San Diego
    La Jolla, California 92037, United States
  • UCLA Medical Center
    Los Angeles, California 90095, United States
  • UC Irvine Medical Center
    Orange, California 92868, United States
  • Stanford Neuroscience Health Center
    Palo Alto, California 94304, United States
  • UC Davis Medical Center
    Sacramento, California 95817, United States
  • UC Denver
    Aurora, Colorado 80045, United States
  • University of Florida
    Gainesville, Florida 32611, United States
  • Rare Disease Research
    Atlanta, Georgia 30329, United States
  • Northwestern University
    Chicago, Illinois 60611, United States
  • Indiana University School of Medicine
    Indianapolis, Indiana 46202, United States
  • University of Iowa
    Iowa City, Iowa 52242, United States
  • University of Kansas Medical Center
    Kansas City, Kansas 66160, United States
  • Kennedy Krieger Institute
    Baltimore, Maryland 21205, United States
  • University of Massachusetts Memorial Medical Center
    Worcester, Massachusetts 01605, United States
  • Washington University School of Medicine
    St Louis, Missouri 63110, United States
  • University of Rochester Medical Center
    Rochester, New York 14642, United States
  • Rare Disease Research, LLC NC
    Hillsborough, North Carolina 27278, United States
  • University of Cincinnati Gardner Neuroscience Institute
    Cincinnati, Ohio 45219, United States
  • Nationwide Children's Hospital
    Columbus, Ohio 43205, United States
  • University of Pennsylvania
    Philadelphia, Pennsylvania 19104, United States
  • National Neuromuscular Research Institute
    Austin, Texas 78759, United States
  • Neurology Rare Disease Center
    Denton, Texas 76208, United States
  • Virginia Commonwealth University Health
    Richmond, Virginia 23298, United States
  • St Vincent's Hospital
    Melbourne, Victoria 3065, Australia
  • University Hospital Gent
    Ghent, Belgium 9000, Belgium
  • Universitaire Ziekenhuizen Leuven
    Leuven, Belgium 3000, Belgium
  • Centre Hospitalier Régional de la Citadelle
    Liége, Belgium 4000, Belgium
  • Rigshospitalet
    Copenhagen, 2100, Denmark
  • Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
    Marseille, France 13005, France
  • CHU de Nantes
    Nantes, France 44093, France
  • CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
    Nice, France 06001, France
  • AP-HP Hopital Pitie-Salpetriere
    Paris, France 75013, France
  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen
    Munich, Germany 80336, Germany
  • Hadassah University Hospital
    Jerusalem, 91240, Israel
  • Fondazione IRCCS Ca'Granda Ospedale Maggiore
    Milan, Italy 20122, Italy
  • Azienda Ospedale - Università Padova
    Padova, Italy 35128, Italy
  • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
    Rome, Italy 00168, Italy
  • Leids Universitair Medisch Centrum
    Leiden, 2333 ZA, Netherlands
  • Optimal Clinical Trials
    Auckland, 1023, New Zealand
  • Hospital Universitario Vall d'Hebron
    Barcelona, Spain 08035, Spain
  • Hospital Universitario de Bellvitge
    Barcelona, Spain 08907, Spain
  • Hospital Universitario Donostia
    Donostia / San Sebastian, Spain 20014, Spain
  • Hospital Universitari i Politecnic La Fe
    Valencia, Spain 46026, Spain
  • Salford Royal Hospital
    Salford, United Kingdom M68HD, United Kingdom
  • University College London Hospital
    London, NW1 2PG, United Kingdom
  • St. George's University Hospitals NHS Foundation Trust
    London, SW17 0QT, United Kingdom
  • Newcastle Freeman Hospital
    Newcastle, NE7 7DN, United Kingdom
07

References and documents

Related links

Individual participant data

Plan to share: No

08

Registry details

Key details

Study ID
NCT06066580
Lead sponsor
Edgewise Therapeutics, Inc.
Collaborators
Medpace, Inc.
Responsible party
Sponsor
First posted
Oct 4, 2023
Start date
Nov 2, 2023
Primary completion
Feb 2031 (estimated)
Completion
Feb 2031 (estimated)
Last update
Sep 17, 2026

Study contacts

Joanne M. Donovan, MD, PhD
study chair · Edgewise Therapeutics, Inc.

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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