The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with Duchenne Muscular Dystrophy (DMD) on stable background therapy.
This is a Phase 2, multicenter, open-label study of KER-065. The study will consist of 3 periods: * Screening Period (up to 6 weeks) * Treatment Period (48 weeks) * Safety Follow-up Period (4 weeks) Participants will be enrolled in parallel into 1 of 3 treatment cohorts: * Cohort A1 (Late Ambulatory) * Cohort A2 (Late Ambulatory) * Cohort N1 (Early Nonambulatory)
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
36
KER-065 will be administered subcutaneously (SC)
Number of participants with treatment-emergent adverse events (TEAEs and serious adverse events (SAEs)
To evaluate the safety and tolerability of KER-065 in ambulatory and nonambulatory participants with DMD
Time frame: Up to approximately 14 months
KER-065 serum concentration by visit, as appropriate
To assess the pharmacokinetics (PK) of KER-065 in ambulatory and nonambulatory participants with DMD
Time frame: Up to Week 52
Number and proportion of participants with treatment-emergent ADA (antidrug antibody) by visit
To assess the immunogenicity of KER-065 in ambulatory and nonambulatory participants with DMD
Time frame: Up to Week 52
Change from baseline by visit in bone mineral density (BMD), fat mass, and lean body mass, as measured by dual- energy X-ray absorptiometry (DXA)
To assess the effect of KER-065 on body composition in ambulatory and nonambulatory participants with DMD
Time frame: Week 12, Week 24 and Week 48
Change from baseline by visit in muscle volume and intramuscular fat by skeletal muscle Magnetic Resonance Imaging (MRI)
To assess the effect of KER-065 on skeletal muscle in ambulatory and nonambulatory participants with DMD
Time frame: Week 24 and Week 48
Ambulatory: Change from baseline by visit in North Star Ambulatory Assessment (NSAA) total score
To assess the effect of KER-065 on motor function in ambulatory participants with DMD
Time frame: Week 24 and Week 48
Ambulatory: Change from baseline by visit in 4-stair climb (4SC)
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To assess the effect of KER-065 on motor function in ambulatory participants with DMD
Time frame: Week 24 and Week 48
Ambulatory: Change from baseline by visit in 10-meter walk/run (10MWR) test
To assess the effect of KER-065 on motor function in ambulatory participants with DMD
Time frame: Week 24 and Week 48
Ambulatory: Change from baseline by visit in time to rise (TTR)
To assess the effect of KER-065 on motor function in ambulatory participants with DMD
Time frame: Week 24 and Week 48
Nonambulatory: Change from baseline by visit in Performance of Upper Limb (PUL) v2.0 score
To assess the effect of KER-065 on motor function in nonambulatory participants with DMD
Time frame: Week 24 and Week 48