The FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized, double-blind, placebo-controlled Part A, followed by an open-label part B.
FOX 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 48 children and adolescents with Duchenne muscular dystrophy treated with oral, once-daily sevasemten. This study will have up to a 4-week Screening period, a 12-week randomized double-blind, placebo-controlled treatment period (Part A), followed by up to a 144-week open-label extension period (Part B). Approximately forty-eight (48) participants aged 6 to 17, inclusive, will be randomized to sevasemten or placebo in a 2:1 ratio. Three dose cohorts (Cohort 1, Cohort 2 and Cohort 3) of approximately 12 participants each will be enrolled. Approximately 12 additional participants may be added to 1 of these 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
43
Sevasemten is administered orally once per day
Sevasemten is administered orally once per day
Sevasemten is administered orally once per day
UCLA Medical Center
Los Angeles, California, United States
UC Davis Medical Center
Sacramento, California, United States
University of Florida
Gainesville, Florida, United States
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, United States
Number of adverse events during treatment with sevasemten or placebo
All participants
Time frame: 36 months
Severity of adverse events during treatment with sevasemten or placebo
All participants
Time frame: 36 months
Incidence of laboratory test-related treatment emergent adverse events
All participants
Time frame: 36 months
Pharmacokinetics as measured by steady state plasma concentration
All participants
Time frame: 36 months
Change from Baseline in serum creatine kinase
All participants
Time frame: 12 weeks
Change from Baseline in fast skeletal muscle troponin I
All participants
Time frame: 12 weeks
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Placebo is administered orally once per day
Washington University School of Medicine
St Louis, Missouri, United States
Rare Disease Research
Hillsborough, North Carolina, United States
Nationwide Children's Hospital
Columbus, Ohio, United States