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.
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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
76
Sevasemten is administered orally once per day
Sevasemten is administered orally once per day
Sevasemten is administered orally once per day
Arkansas Children's Hospital
Little Rock, Arkansas, United States
UCLA Medical Center
Los Angeles, California, United States
UC Davis Medical Center
Sacramento, California, United States
Number of adverse events during treatment with sevasemten or placebo
All participants
Time frame: 48 months
Severity of adverse events during treatment with sevasemten or placebo
All participants
Time frame: 48 months
Incidence of laboratory test-related treatment emergent adverse events
All participants
Time frame: 48 months
Pharmacokinetics as measured by steady state plasma concentration
All participants
Time frame: 48 months
Change from Baseline in serum creatinine kinase
All participants
Time frame: 12 weeks
Change from Baseline in fast skeletal muscle troponin I
All participants
Time frame: 12 weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Sevasemten is administered orally once per day
Sevasemten is administered orally once per day
Placebo is administered orally once per day
Children's Hospital Colorado
Aurora, Colorado, United States
University of Florida
Gainesville, Florida, United States
Rare Disease Research
Atlanta, Georgia, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, United States
...and 4 more locations