Becker Muscular DystrophyPhase 2NCT05291091
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
A Phase 2 study testing Sevasemten 10 mg, Sevasemten 5 mg and Sevasemten 12.5 mg in people with Becker Muscular Dystrophy, run by Edgewise Therapeutics, Inc. at 51 sites in 12 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 12 to 50 years, men only
- Conditions the study is for
- Becker Muscular Dystrophy
- What is being tested?
- Sevasemten 10 mg, Sevasemten 5 mg and Sevasemten 12.5 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 244 participants
- Where
- 51 sitesin 12 countries
- Study dates
- 10 Nov 2022to 7 Sep 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 12 to 50 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Adults (aged 18 to 50 years, inclusive) with a documented dystrophin mutation and phenotype consistent with Becker muscular dystrophy, and history of being ambulatory beyond 16 years of age without steroids; history of being ambulatory beyond 18 years of age with steroids.
- Able to complete the 100-meter timed test in < 200 seconds with or without use of mobility aid devices.
- Able to perform the North Star Ambulatory Assessment scale and achieve a score of 5 to 32, inclusive.
Who cannot join
- Medical history or clinically significant physical examination/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes contraindications to magnetic resonance imaging such as non-compatible implanted medical devices or severe claustrophobia.
- Cardiac echocardiogram ejection fraction < 40%
- Forced vital capacity predicted <60% or using daytime ventilatory support
- Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
- Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.
Trial locations and contacts
51 sites in 12 countries.
51 sites in 12 countries
United States26 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
University of Minnesota
Minneapolis, Minnesota, 55455
UC Irvine Medical Center
Orange, California, 92868
Stanford Neuroscience Health Center
Palo Alto, California, 94304
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
University of Pittsburgh
Pittsburgh, Pennsylvania, 15224
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, 13005
CHU de Nantes
Nantes, 44093
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
Nice, 06001
AP-HP Hopital Pitie-Salpetriere
Paris, 75013
Spain4 sites
Hospital Universitario Vall d'Hebron
Barcelona, 08035
Hospital Universitari de Bellvitge
Barcelona, 08907
Hospital Universitario Donostia
Donostia / San Sebastian, 20014
Hospital Universitari i Politecnic La Fe
Valencia, 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, M68HD
Belgium3 sites
University Hospital Gent
Ghent, Belgium, 9000
Universitaire Ziekenhuizen Leuven
Leuven
Centre Hospitalier Régional de la Citadelle
Liège, 4000
Italy3 sites
Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico di Milano
Milan, 20122
Azienda Ospedale - Università Padova
Padova, 35128
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, 00168
Israel2 sites
Hadassah University Hospital
Jerusalem, 91240
Schneider Children's Hospital of Israel
Petah Tikva, 49202
Germany1 site
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
Munich, 80336
About this study
Official title: A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy
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.
Read the detailed description
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.
- Conditions
- Becker Muscular Dystrophy
- Being tested
- Sevasemten 10 mg, Sevasemten 5 mg, Sevasemten 12.5 mg, Placebo
- 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.
Sevasemten 10 mgDrug
Sevasemten is administered orally once per day
Sevasemten 5 mgDrug
Sevasemten is administered orally once per day
Sevasemten 12.5 mgDrug
Sevasemten is administered orally once per day
PlaceboDrug
Placebo is administered orally once per day
| Study group | Role | What they receive |
|---|---|---|
| Adult Cohort 1 | Experimental | Drug: Sevasemten Drug: Placebo Receives: Drug: Sevasemten 10 mg; Drug: Placebo |
| Adult Cohort 2 | Experimental | Drug: Sevasemten Drug: Placebo Receives: Drug: Sevasemten 10 mg; Drug: Placebo |
| Adult Cohort 6 | Experimental | Drug: Sevasemten Drug: Placebo Receives: Drug: Sevasemten 10 mg; Drug: Placebo |
| Adolescent Cohort 4 | Experimental | Drug: Sevasemten Drug: Placebo Receives: Drug: Sevasemten 5 mg; Drug: Placebo |
| Adolescent Cohort 5 | Experimental | Drug: Sevasemten Drug: Placebo Receives: Drug: Sevasemten 12.5 mg; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of treatment emergent adverse events in those treated with sevasemten or placebo
All participants
Measured: 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
Measured: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum Creatine Kinase
Adult participants
Measured: Averaged across Months 6, 9, 12 Months (CANYON Cohorts 1, 2)
Change from Baseline in total North Star Ambulatory Assessment score
Adult participants
Measured: 18 months (GRAND CANYON Cohort 6)
Secondary outcomes (13)
Change from Baseline in total North Star Ambulatory Assessment score
Adult participants
Measured: 12 Months (CANYON Cohorts 1, 2)
Pharmacokinetics as measured by steady state plasma concentration
All participants
Measured: 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
Measured: 12 months (CANYON Cohorts 4, 5)
Incidence of laboratory test-related treatment emergent adverse events
Adult participants
Measured: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum Creatine Kinase
Adult participants
Measured: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in plasma fast skeletal muscle Troponin I
Adult participants
Measured: Averaged across Months 6 and 12 (CANYON Cohorts 1, 2); 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum myoglobin
Adult participants
Measured: 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
Measured: 12 Months (CANYON Cohorts 1, 2)
Change from Baseline in 4-stair climb test
Adult participants
Measured: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in the 10-meter walk/run test
Adult participants
Measured: 12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Change from Baseline in 100-meter timed test
Adult participants
Measured: 12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Change from Baseline in stride velocity (95th percentile)
Adult participants
Measured: 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
Measured: 18 months (GRAND CANYON Cohort 6)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2022
- Study start10 Nov 2022
- Primary completion7 Sep 2026
- Study completion7 Sep 2026
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
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 244 participants (actual)
Sponsor and investigators
- Lead sponsor
- Edgewise Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Medpace, Inc., ImagingNMD, SYSNAV
- Responsible party
- Sponsor
- Investigators
- Joanne Donovan, MD, PhD, Study chair, Edgewise Therapeutics, Inc.Roxana D. Dreghici, Study chair, Edgewise Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05291091
- Sponsor's protocol ID
- EDG-5506-201
- Status last verified
- Sep 2026
- First submitted
- 14 Mar 2022
- Last updated
- 21 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 21 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT05291091
- Acronym / sponsor ID
- EDG-5506-201
- Conditions
- Becker Muscular Dystrophy
- Keywords
- Becker Muscular Dystrophy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 244 participants (actual)
- Lead sponsor
- Edgewise Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Medpace, Inc., ImagingNMD, SYSNAV
- Study start
- 10 Nov 2022 (actual)
- Primary completion
- 7 Sep 2026 (actual)
- Study completion
- 7 Sep 2026 (actual)
- First posted
- 22 Mar 2022 (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.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Adult Cohort 1 | Experimental | Drug: Sevasemten 10 mg, Drug: PlaceboDrug: Sevasemten Drug: Placebo |
| Adult Cohort 2 | Experimental | Drug: Sevasemten 10 mg, Drug: PlaceboDrug: Sevasemten Drug: Placebo |
| Adult Cohort 6 | Experimental | Drug: Sevasemten 10 mg, Drug: PlaceboDrug: Sevasemten Drug: Placebo |
| Adolescent Cohort 4 | Experimental | Drug: Sevasemten 5 mg, Drug: PlaceboDrug: Sevasemten Drug: Placebo |
| Adolescent Cohort 5 | Experimental | Drug: Sevasemten 12.5 mg, Drug: PlaceboDrug: Sevasemten Drug: Placebo |
Who may qualify
- Ages
- 12 to 50 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Adults (aged 18 to 50 years, inclusive) with a documented dystrophin mutation and phenotype consistent with Becker muscular dystrophy, and history of being ambulatory beyond 16 years of age without steroids; history of being ambulatory beyond 18 years of age with steroids.
- Able to complete the 100-meter timed test in < 200 seconds with or without use of mobility aid devices.
- Able to perform the North Star Ambulatory Assessment scale and achieve a score of 5 to 32, inclusive.
Key exclusion criteria (5 listed)
- Medical history or clinically significant physical examination/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes contraindications to magnetic resonance imaging such as non-compatible implanted medical devices or severe claustrophobia.
- Cardiac echocardiogram ejection fraction < 40%
- Forced vital capacity predicted <60% or using daytime ventilatory support
- Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
- Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.
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 |
|---|---|---|
| Sevasemten 10 mgSevasemten is administered orally once per day | Drug | Adult Cohort 1, Adult Cohort 2, Adult Cohort 6 |
| Sevasemten 5 mgSevasemten is administered orally once per day | Drug | Adolescent Cohort 4 |
| Sevasemten 12.5 mgSevasemten is administered orally once per day | Drug | Adolescent Cohort 5 |
| PlaceboPlacebo is administered orally once per day | Drug | Adolescent Cohort 4, Adolescent Cohort 5, Adult Cohort 1, Adult Cohort 2, Adult Cohort 6 |
Primary outcome
Number of treatment emergent adverse events in those treated with sevasemten or placebo
Time frame: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
All participants
Severity of treatment emergent adverse events in those treated with sevasemten or placebo
Time frame: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
All participants
Change from Baseline in serum Creatine Kinase
Time frame: Averaged across Months 6, 9, 12 Months (CANYON Cohorts 1, 2)
Adult participants
Change from Baseline in total North Star Ambulatory Assessment score
Time frame: 18 months (GRAND CANYON Cohort 6)
Adult participants
13 secondary outcomes
Change from Baseline in total North Star Ambulatory Assessment score
Time frame: 12 Months (CANYON Cohorts 1, 2)
Pharmacokinetics as measured by steady state plasma concentration
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
Time frame: 12 months (CANYON Cohorts 4, 5)
Incidence of laboratory test-related treatment emergent adverse events
Time frame: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum Creatine Kinase
Time frame: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in plasma fast skeletal muscle Troponin I
Time frame: Averaged across Months 6 and 12 (CANYON Cohorts 1, 2); 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum myoglobin
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)
Time frame: 12 Months (CANYON Cohorts 1, 2)
Change from Baseline in 4-stair climb test
Time frame: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in the 10-meter walk/run test
Time frame: 12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Change from Baseline in 100-meter timed test
Time frame: 12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Change from Baseline in stride velocity (95th percentile)
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
Time frame: 18 months (GRAND CANYON Cohort 6)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 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. This study is not recruiting at the moment.
Joanne Donovan, MD, PhD study chair
Edgewise Therapeutics, Inc.
Roxana D. Dreghici study chair
Edgewise Therapeutics, Inc.
51 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 10 mg, Sevasemten 5 mg and Sevasemten 12.5 mg in people with Becker Muscular Dystrophy, run by Edgewise Therapeutics, Inc. at 51 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 3 inclusion and 5 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: finished, as of the sponsor's last update on 21 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 51 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 treatment emergent adverse events in those treated with sevasemten or placebo (time frame: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)). The study lists 4 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 21 Sep 2026.