Duchenne Muscular DystrophyPhase 2NCT05540860
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
A Phase 2 study testing Sevasemten Dose 1, Sevasemten Dose 2 and Sevasemten Dose 3 in people with Duchenne Muscular Dystrophy, run by Edgewise Therapeutics, Inc. at 14 sites in United States.
Trial summary
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an open-label part B. Read more about this study
- Who is it for?
- Ages 4 to 9 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Sevasemten Dose 1, Sevasemten Dose 2 and Sevasemten Dose 3
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 76 participants
- Where
- 14 sitesUnited States
- Study dates
- 24 Oct 2022to Jan 2027
- 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 4 to 9 years
- Men only
- No healthy volunteers
- Child
Key Common Inclusion Criteria:
- A documented mutation on the DMD gene and phenotype consistent with Duchenne muscular dystrophy.
- Able to complete the stand from supine in ≤ 10 seconds and able to perform the 4-stair climb in < 10 seconds at the Screening visit.
- Body weight greater than or equal to 15 kg at the Screening visit.
For Cohorts 1, 2, 3, 4 and 5:
Aged 4-9 years on a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit.
For Cohort 2 Non-Steroid (Cohort 2NS):
Aged 4-7 years not on corticosteroids within 6 months prior to the Baseline visit.
Key Common Exclusion Criteria:
- Medical history or clinically significant physical exam/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes venous access that would be too difficult to facilitate repeated blood testing.
- A forced vital capacity < 60% predicted at the Screening visit for those participants who are > 8 years old at Screening.
- A cardiac echocardiography showing left ventricular ejection < 45% at the Screening visit.
- Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the Screening visit in the present study.
- Receipt of a stable dose of an approved exon-skipping therapy with a treatment duration of less than 1 year prior to the Screening visit.
For Cohort 2 Non-Steroid (Cohort 2NS):
Receipt of oral corticosteroids for the treatment of Duchenne muscular dystrophy in the previous 6 months. Participants will not be tapered off steroids for the purpose of this study and oral corticosteroids for the treatment of Duchenne muscular dystrophy may be initiated after the Week 16 visit.
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
Rare Disease Research
Atlanta, Georgia, 30329
Children's Hospital Colorado
Aurora, Colorado, 80045
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43205
Cook Children's Medical Center
Fort Worth, Texas, 76104
University of Florida
Gainesville, Florida, 32610
University of Iowa
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
UCLA Medical Center
Los Angeles, California, 90095
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
About this study
Official title: A 2-part Phase 2 Study of Safety, Pharmacokinetics and Biomarkers in Children With Duchenne Muscular Dystrophy Including a Randomized, Double-Blind, Placebo-Controlled Part A, Followed by an Open-Label Part B
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an open-label part B.
Read the detailed description
This is a 2-part, multi-center, Phase 2 study to evaluate the effect of sevasemten (EDG-5506) on safety, pharmacokinetics and biomarkers of muscle damage in approximately 72 children with DMD treated with oral, once-daily sevasemten for up to 48 months. This study will have up to a 4-week Screening period, a 12-week randomized, double-blind, placebo controlled treatment period (Part A), up to a 196-week open-label extension period (Part B), and a 2-week follow up period.
Approximately 72 participants aged 4 to 9 years inclusive will be randomized to sevasemten or placebo in a 2:1 ratio. Five dose cohorts (C1, C2, C3, C4 and C5) of approximately 9 participants each will be enrolled sequentially. Approximately 18 total additional participants may be added across Cohorts 2, 3, or 4.
An additional cohort, Cohort 2NS, to include participants (aged 4 to 7 years inclusive) not currently treated with corticosteroids, will enroll approximately 9 participants after Cohort 2 safety review and in parallel with the additional cohorts.
After review of emerging data, the protocol was amended so all dose cohorts receive the same dose in Part B.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3, Sevasemten Dose 4, Sevasemten Dose 5
- Keywords
- Duchenne 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 Dose 1Drug
Sevasemten is administered orally once per day
Sevasemten Dose 2Drug
Sevasemten is administered orally once per day
Sevasemten Dose 3Drug
Sevasemten is administered orally once per day
Sevasemten Dose 4Drug
Sevasemten is administered orally once per day
Sevasemten Dose 5Drug
Sevasemten is administered orally once per day
PlaceboDrug
Placebo is administered orally once per day
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Drug: Sevasemten Drug: Placebo Receives: Drug: Sevasemten Dose 1; Drug: Placebo |
| Cohort 2 | Experimental | Drug: Sevasemten Drug: Placebo Receives: Drug: Sevasemten Dose 2; Drug: Placebo |
| Cohort 3 | Experimental | Drug: Sevasemten Drug: Placebo Receives: Drug: Sevasemten Dose 3; Drug: Placebo |
| Cohort 4 | Experimental | Drug: Sevasemten Drug: Placebo Receives: Drug: Sevasemten Dose 4; Drug: Placebo |
| Cohort 5 | Experimental | Drug: Sevasemten Drug: Placebo Receives: Drug: Sevasemten Dose 5; Drug: Placebo |
| Cohort 2NS | Experimental | Drug: Sevasemten Drug: Placebo Receives: Drug: Sevasemten Dose 2; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse events during treatment with sevasemten or placebo
All participants
Measured: 48 months
Severity of adverse events during treatment with sevasemten or placebo
All participants
Measured: 48 months
Secondary outcomes (4)
Incidence of laboratory test-related treatment emergent adverse events
All participants
Measured: 48 months
Pharmacokinetics as measured by steady state plasma concentration
All participants
Measured: 48 months
Change from Baseline in serum creatinine kinase
All participants
Measured: 12 weeks
Change from Baseline in fast skeletal muscle troponin I
All participants
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2022
- Study start24 Oct 2022
- Primary completionJan 2027 (estimated)
- Study completionJan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne 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
- 76 participants (actual)
Sponsor and investigators
- Lead sponsor
- Edgewise Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05540860
- Sponsor's protocol ID
- EDG-5506-210
- Status last verified
- Nov 2025
- First submitted
- 6 Sep 2022
- Last updated
- 12 Nov 2025
- 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 12 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 2-part Phase 2 Study of Safety, Pharmacokinetics and Biomarkers in Children With Duchenne Muscular Dystrophy Including a Randomized, Double-Blind, Placebo-Controlled Part A, Followed by an Open-Label Part B
- ClinicalTrials.gov ID
- NCT05540860
- Acronym / sponsor ID
- EDG-5506-210
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 76 participants (actual)
- Lead sponsor
- Edgewise Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 24 Oct 2022 (actual)
- Primary completion
- Jan 2027 (estimated)
- Study completion
- Jan 2027 (estimated)
- First posted
- 15 Sep 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.
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an open-label part B.
Detailed description
This is a 2-part, multi-center, Phase 2 study to evaluate the effect of sevasemten (EDG-5506) on safety, pharmacokinetics and biomarkers of muscle damage in approximately 72 children with DMD treated with oral, once-daily sevasemten for up to 48 months. This study will have up to a 4-week Screening period, a 12-week randomized, double-blind, placebo controlled treatment period (Part A), up to a 196-week open-label extension period (Part B), and a 2-week follow up period.
Approximately 72 participants aged 4 to 9 years inclusive will be randomized to sevasemten or placebo in a 2:1 ratio. Five dose cohorts (C1, C2, C3, C4 and C5) of approximately 9 participants each will be enrolled sequentially. Approximately 18 total additional participants may be added across Cohorts 2, 3, or 4.
An additional cohort, Cohort 2NS, to include participants (aged 4 to 7 years inclusive) not currently treated with corticosteroids, will enroll approximately 9 participants after Cohort 2 safety review and in parallel with the additional cohorts.
After review of emerging data, the protocol was amended so all dose cohorts receive the same dose in Part B.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: Sevasemten Dose 1, Drug: PlaceboDrug: Sevasemten Drug: Placebo |
| Cohort 2 | Experimental | Drug: Sevasemten Dose 2, Drug: PlaceboDrug: Sevasemten Drug: Placebo |
| Cohort 3 | Experimental | Drug: Sevasemten Dose 3, Drug: PlaceboDrug: Sevasemten Drug: Placebo |
| Cohort 4 | Experimental | Drug: Sevasemten Dose 4, Drug: PlaceboDrug: Sevasemten Drug: Placebo |
| Cohort 5 | Experimental | Drug: Sevasemten Dose 5, Drug: PlaceboDrug: Sevasemten Drug: Placebo |
| Cohort 2NS | Experimental | Drug: Sevasemten Dose 2, Drug: PlaceboDrug: Sevasemten Drug: Placebo |
Who may qualify
- Ages
- 4 to 9 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
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 Dose 1Sevasemten is administered orally once per day | Drug | Cohort 1 |
| Sevasemten Dose 2Sevasemten is administered orally once per day | Drug | Cohort 2, Cohort 2NS |
| Sevasemten Dose 3Sevasemten is administered orally once per day | Drug | Cohort 3 |
| Sevasemten Dose 4Sevasemten is administered orally once per day | Drug | Cohort 4 |
| Sevasemten Dose 5Sevasemten is administered orally once per day | Drug | Cohort 5 |
| PlaceboPlacebo is administered orally once per day | Drug | Cohort 1, Cohort 2, Cohort 2NS, Cohort 3, Cohort 4, Cohort 5 |
Primary outcome
Number of adverse events during treatment with sevasemten or placebo
Time frame: 48 months
All participants
Severity of adverse events during treatment with sevasemten or placebo
Time frame: 48 months
All participants
4 secondary outcomes
Incidence of laboratory test-related treatment emergent adverse events
Time frame: 48 months
Pharmacokinetics as measured by steady state plasma concentration
Time frame: 48 months
Change from Baseline in serum creatinine kinase
Time frame: 12 weeks
Change from Baseline in fast skeletal muscle troponin I
Time frame: 12 weeks
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 12 Nov 2025.
- Sponsor WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Duchenne Muscular Dystrophy
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
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.
The record lists no contact. Use the official record to reach the sponsor.
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sevasemten Dose 1, Sevasemten Dose 2 and Sevasemten Dose 3 in people with Duchenne Muscular Dystrophy, run by Edgewise Therapeutics, Inc. at 14 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: under way, not looking for participants, as of the sponsor's last update on 12 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States. 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 during treatment with sevasemten or placebo (time frame: 48 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 12 Nov 2025.