A Phase 2 interventional study of Sevasemten 10 mg and Sevasemten 5 mg in Becker Muscular Dystrophy, sponsored by Edgewise Therapeutics, Inc.. Completed at 51 sites in 12 countries. Open to male participants aged 12 Years to 50 Years. Per ClinicalTrials.gov, last updated 2026-09-21.
Sponsored by Edgewise Therapeutics, Inc. · Phase 2, Interventional, and Treatment
Among 232 studies in Muscular Dystrophy, Duchenne with a site in United States — 36 open to participants now, 60 under way, 8 of them phase 3. Muscular Dystrophy, Duchenne · United States.
Among the 232 studies in Muscular Dystrophy, Duchenne with a site in United States, this one is the 7th largest by enrollment of the 171 interventional ones that give a count, is one of 15 that started in 2022, is one of 57 in phase 2, and was the 181st registered.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 108 are open to participants now.
Browse Muscular Dystrophy, Duchenne studies →36 of the 232 studies in Muscular Dystrophy, Duchenne with a site in United States are open now. Recruiting first, the newest first:
Sarepta Therapeutics, Inc. · Sites in United States
University of Florida · Sites in United States
Vanderbilt University Medical Center · Sites in United States
University of Rochester · Sites in United States
Dyne Therapeutics · Sites in United States
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 66 Muscular Dystrophy, Duchenne studies recruiting now, 30 (45%) set no upper age limit and 34 (52%) stop at 75 or younger; 60 take children under 18; 12 take healthy volunteers.
This study: 12 Years to 50 Years · male · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
232 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 57 · Phase 2 90 · Phase 3 40 · Phase 4 5
First in 2000; this study’s year is marked. 15 started in 2022.
This study's enrollment of 244 is above the median of 26 across 332 interventional studies indexed under Muscular Dystrophy, Duchenne.
Median enrollment 25 across the 171 interventional studies, 75 across the 55 observational. Of the 160 that completed or were terminated, 77 (48%) have results posted.
This tab as a report to print or save as PDF → With an account.
130 of 232 have an industry lead sponsor. The 10 most frequent:
Edgewise Therapeutics, Inc. is the lead sponsor of 17 studies on the registry; 2 are open to participants now.
Of its 12 completed or terminated interventional studies of FDA-regulated products, 1 (8%) have results posted.
Edgewise Therapeutics, Inc. has 6 studies filed under Muscular Dystrophy, Duchenne — fewer than ten, so there is no comparison to draw.
19 of the studies in Muscular Dystrophy, Duchenne with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05881408Phase 3Sarepta Therapeutics, Inc.A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) | 2027-05-31 |
| NCT06290713University of FloridaVasodilator and Exercise Study for DMD (VASO-REx) | 2026-11 |
| NCT06450639Hoffmann-La RocheA Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) | 2026-11-18 |
| NCT03368742Solid Biosciences Inc.Microdystrophin Gene Transfer Study in Adolescents and Children With DMD | 2026-11-30 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
Among the 232 studies in Muscular Dystrophy, Duchenne with a site in United States, from their registry updates:
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A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 175 adult participants in a cohort called GRAND CANYON, that is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of sevasemten in adults with Becker.
The EDG-5506-201 protocol was amended to include an additional cohort thus consists of two parts.
Part 1: CANYON is a double-blind, randomized, placebo-controlled design to investigate the effect of sevasemten on the safety, pharmacokinetics, biomarkers, and functional measures. Approximately 32 adults and 18 adolescents with Becker muscular dystrophy are planned to enroll in this study. This study will have up to a 4-week Screening period, a 12-month Treatment period, followed by a 4-week follow-up period.
Approximately 32 adult participants will randomize to Cohort 1 or Cohort 2 in a 1:1 ratio then each cohort will further randomize to sevasemten or placebo in a 3:1 ratio.
Approximately 9 adolescent participants will enroll in Cohort 4 and randomize in a 2:1 ratio to sevasemten or placebo. Cohort 5 will randomize an additional 9 participants in a 2:1 ratio to either sevasemten or placebo after Cohort 4.
Part 2: GRAND CANYON or Cohort 6 is a double-blind, randomized, placebo-controlled design to investigate the safety and efficacy of sevasemten in adults with Becker muscular dystrophy after 18 months of treatment. Approximately 175 adults with Becker muscular dystrophy are planned to enroll in this study. This study will have up to a 4-week Screening period, an 18-month Treatment period, followed by a 4-week follow-up period.
Approximately 175 adult participants will be randomized in Cohort 6 in a 2:1 ratio either to sevasemten or placebo.
Enrollment for CANYON and GRAND CANYON has been completed.
GRAND CANYON eligibility is listed below.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Sevasemten Drug: Placebo
Drug: Sevasemten 10 mg · Drug: Placebo
Drug: Sevasemten Drug: Placebo
Drug: Sevasemten 10 mg · Drug: Placebo
Drug: Sevasemten Drug: Placebo
Drug: Sevasemten 10 mg · Drug: Placebo
Drug: Sevasemten Drug: Placebo
Drug: Sevasemten 5 mg · Drug: Placebo
Drug: Sevasemten Drug: Placebo
Drug: Sevasemten 12.5 mg · Drug: Placebo
Sevasemten is administered orally once per day
Sevasemten is administered orally once per day
Sevasemten is administered orally once per day
Placebo is administered orally once per day
Number of treatment emergent adverse events in those treated with sevasemten or placebo
All participants
Time frame: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Severity of treatment emergent adverse events in those treated with sevasemten or placebo
All participants
Time frame: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum Creatine Kinase
Adult participants
Time frame: Averaged across Months 6, 9, 12 Months (CANYON Cohorts 1, 2)
Change from Baseline in total North Star Ambulatory Assessment score
Adult participants
Time frame: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in total North Star Ambulatory Assessment score
Adult participants
Time frame: 12 Months (CANYON Cohorts 1, 2)
Pharmacokinetics as measured by steady state plasma concentration
All participants
Time frame: 12 Months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Change from Baseline in growth as assessed by height centile on World Health Organization growth charts
Adolescent participants
Time frame: 12 months (CANYON Cohorts 4, 5)
Incidence of laboratory test-related treatment emergent adverse events
Adult participants
Time frame: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum Creatine Kinase
Adult participants
Time frame: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in plasma fast skeletal muscle Troponin I
Adult participants
Time frame: Averaged across Months 6 and 12 (CANYON Cohorts 1, 2); 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum myoglobin
Adult participants
Time frame: Averaged across Months 6, 9, and 12 (CANYON Cohorts 1,2); 18 months (GRAND CANYON Cohort 6)
Change from Baseline in total NSAD score (CANYON Cohorts 1,2)
Adult participants
Time frame: 12 Months (CANYON Cohorts 1, 2)
Change from Baseline in 4-stair climb test
Adult participants
Time frame: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in the 10-meter walk/run test
Adult participants
Time frame: 12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Change from Baseline in 100-meter timed test
Adult participants
Time frame: 12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Change from Baseline in stride velocity (95th percentile)
Adult participants
Time frame: 18 Months (GRAND CANYON Cohort 6)
Month 18 change from Baseline in fat fraction of upper leg muscles as assessed by Magnetic Resonance Imaging
Adult participants
Time frame: 18 months (GRAND CANYON Cohort 6)
Plan to share: No
Ran from Nov 2022 to primary completion in Sep 2026, 45 months.
Of 40 industry-sponsored phase 2 interventional studies under Muscular Dystrophy, Duchenne, half reached primary completion within 31 months of starting (the median), counting those still running; a quarter within 20 and three quarters within 42. This study's 45 months is above that.
Counting every sponsor, half of 47 studies reached it within 31 months; a quarter within 20 and three quarters within 42.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Sevasemten
Muscular Dystrophy, Duchenne →
Edgewise Therapeutics, Inc.