A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
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
1 other identifier
interventional
76
1 country
14
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Oct 2022
Typical duration for phase_2
14 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 6, 2022
CompletedFirst Posted
Study publicly available on registry
September 15, 2022
CompletedStudy Start
First participant enrolled
October 24, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
January 1, 2027
November 12, 2025
November 1, 2025
4.2 years
September 6, 2022
November 10, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Number of adverse events during treatment with sevasemten or placebo
All participants
48 months
Severity of adverse events during treatment with sevasemten or placebo
All participants
48 months
Secondary Outcomes (4)
Incidence of laboratory test-related treatment emergent adverse events
48 months
Pharmacokinetics as measured by steady state plasma concentration
48 months
Change from Baseline in serum creatinine kinase
12 weeks
Change from Baseline in fast skeletal muscle troponin I
12 weeks
Study Arms (6)
Cohort 1
EXPERIMENTALDrug: Sevasemten Drug: Placebo
Cohort 2
EXPERIMENTALDrug: Sevasemten Drug: Placebo
Cohort 3
EXPERIMENTALDrug: Sevasemten Drug: Placebo
Cohort 4
EXPERIMENTALDrug: Sevasemten Drug: Placebo
Cohort 5
EXPERIMENTALDrug: Sevasemten Drug: Placebo
Cohort 2NS
EXPERIMENTALDrug: Sevasemten Drug: Placebo
Interventions
Placebo is administered orally once per day
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (14)
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
UCLA Medical Center
Los Angeles, California, 90095, United States
UC Davis Medical Center
Sacramento, California, 95817, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
University of Florida
Gainesville, Florida, 32610, United States
Rare Disease Research
Atlanta, Georgia, 30329, 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
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 6, 2022
First Posted
September 15, 2022
Study Start
October 24, 2022
Primary Completion (Estimated)
January 1, 2027
Study Completion (Estimated)
January 1, 2027
Last Updated
November 12, 2025
Record last verified: 2025-11
Data Sharing
- IPD Sharing
- Will not share