Becker Muscular DystrophyPhase 2MESANCT06066580
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
A Phase 2 study testing Sevasemten in people with Becker Muscular Dystrophy, run by Edgewise Therapeutics, Inc. at 48 sites in 12 countries.
Trial 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 more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Becker Muscular Dystrophy
- What is being tested?
- Sevasemten
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 260 participants
- Where
- 48 sitesin 12 countries
- Study dates
- 2 Nov 2023to Feb 2031
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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])
Who cannot join
- 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.
Trial locations and contacts
48 sites in 12 countries. Contact the study team or a site near you to ask about taking part.
48 sites in 12 countries
United States24 sites
Rare Disease Research
Atlanta, Georgia, 30329
UC Denver
Aurora, Colorado, 80045
National Neuromuscular Research Institute
Austin, Texas, 78759
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Northwestern University
Chicago, Illinois, 60611
University of Cincinnati Gardner Neuroscience Institute
Cincinnati, Ohio, 45219
Nationwide Children's Hospital
Columbus, Ohio, 43205
Neurology Rare Disease Center
Denton, Texas, 76208
University of Florida
Gainesville, Florida, 32611
Rare Disease Research, LLC NC
Hillsborough, North Carolina, 27278
Indiana University School of Medicine
Indianapolis, Indiana, 46202
University of Iowa
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
UC San Diego
La Jolla, California, 92037
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
UCLA Medical Center
Los Angeles, California, 90095
UC Irvine Medical Center
Orange, California, 92868
Stanford Neuroscience Health Center
Palo Alto, California, 94304
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Virginia Commonwealth University Health
Richmond, Virginia, 23298
University of Rochester Medical Center
Rochester, New York, 14642
UC Davis Medical Center
Sacramento, California, 95817
Washington University School of Medicine
St Louis, Missouri, 63110
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, 01605
France4 sites
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
Marseille, France, 13005
CHU de Nantes
Nantes, France, 44093
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
Nice, France, 06001
AP-HP Hopital Pitie-Salpetriere
Paris, France, 75013
Spain4 sites
Hospital Universitario Vall d'Hebron
Barcelona, Spain, 08035
Hospital Universitario de Bellvitge
Barcelona, Spain, 08907
Hospital Universitario Donostia
Donostia / San Sebastian, Spain, 20014
Hospital Universitari i Politecnic La Fe
Valencia, Spain, 46026
United Kingdom4 sites
University College London Hospital
London, NW1 2PG
St. George's University Hospitals NHS Foundation Trust
London, SW17 0QT
Newcastle Freeman Hospital
Newcastle, NE7 7DN
Salford Royal Hospital
Salford, United Kingdom, M68HD
Belgium3 sites
University Hospital Gent
Ghent, Belgium, 9000
Universitaire Ziekenhuizen Leuven
Leuven, Belgium, 3000
Centre Hospitalier Régional de la Citadelle
Liége, Belgium, 4000
Italy3 sites
Fondazione IRCCS Ca'Granda Ospedale Maggiore
Milan, Italy, 20122
Azienda Ospedale - Università Padova
Padova, Italy, 35128
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, Italy, 00168
Germany1 site
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
Munich, Germany, 80336
About this study
Official title: 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
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.
- Conditions
- Becker Muscular Dystrophy
- Being tested
- Sevasemten
- Keywords
- Becker Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SevasemtenDrug
Sevasemten is administered orally once per day
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Drug: Sevasemten Receives: Drug: Sevasemten |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse events in those treated with sevasemten
Measured: 55 Months
Severity of adverse events in those treated with sevasemten
Measured: 55 Months
Secondary outcomes (2)
Incidence of treatment-emergent abnormal clinical chemistry laboratory test results
Measured: 54 Months
Incidence of treatment-emergent abnormal hematology laboratory test results
Measured: 54 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2023
- Study start2 Nov 2023
- Primary completionFeb 2031 (estimated)
- Study completionFeb 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Becker Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 260 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Edgewise Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Medpace, Inc.
- Responsible party
- Sponsor
- Investigators
- Joanne M. Donovan, MD, PhD, Study chair, Edgewise Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06066580
- Sponsor's protocol ID
- EDG-5506-203
- Acronym
- MESA
- Status last verified
- Sep 2026
- First submitted
- 27 Sep 2023
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06066580
- Acronym / sponsor ID
- MESA / EDG-5506-203
- Conditions
- Becker Muscular Dystrophy
- Keywords
- Becker Muscular Dystrophy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 260 participants (estimated)
- Lead sponsor
- Edgewise Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Medpace, Inc.
- Study start
- 2 Nov 2023 (actual)
- Primary completion
- Feb 2031 (estimated)
- Study completion
- Feb 2031 (estimated)
- First posted
- 4 Oct 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: SevasemtenDrug: Sevasemten |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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 (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SevasemtenSevasemten is administered orally once per day | Drug | Treatment |
Primary outcome
Number of adverse events in those treated with sevasemten
Time frame: 55 Months
Severity of adverse events in those treated with sevasemten
Time frame: 55 Months
2 secondary outcomes
Incidence of treatment-emergent abnormal clinical chemistry laboratory test results
Time frame: 54 Months
Incidence of treatment-emergent abnormal hematology laboratory test results
Time frame: 54 Months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2026.
- Sponsor WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Joanne M. Donovan, MD, PhD study chair
Edgewise Therapeutics, Inc.
48 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sevasemten in people with Becker Muscular Dystrophy, run by Edgewise Therapeutics, Inc. at 48 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 48 sites in United States, France, Spain, United Kingdom, Belgium and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of adverse events in those treated with sevasemten (time frame: 55 Months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2026.