Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \< 10 years. The trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy.
This is a global phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \< 10 years. The trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy. One dose of SAT-3247 and placebo will be studied in the US and Canada; two doses of SAT-3247 and placebo will be studied in UK, EU, Serbia, and Australia. Enrollment of up to 51 ambulatory DMD participants aged ≥ 7 and \< 10 years of age is planned globally. Randomization will be stratified by baseline corticosteroid regimen and prior DMD concomitant medications. Each participant will receive once daily doses of SAT-3247 or matched placebo for 12 weeks. Participants will be screened within 28 days before initiating dosing of investigational product at Baseline. Following the Screening period, participants will complete a Baseline visit (Visit 2), a follow-up phone call at Week 1, and visits at Week 4 (Visit 3), Week 8 (Visit 4), and Week 12 (Visit 5).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
51
SAT-3247 is a selective AAK1 inhibitor for oral tablet administration which promotes functional rescue of asymmetric satellite cell division, resulting in the robust production of muscle progenitor cells, subsequent improvement in muscle regeneration, and enhanced muscle function.
matching placebo oral tablets
University of California Los Angeles
Los Angeles, California, United States
NOT_YET_RECRUITINGSafety of SAT-3247
Occurrence of treatment emergent adverse events and relationship to investigational product
Time frame: 12 weeks
Tolerability of SAT-3247
occurrence of clinically significant changes in physical exam, clinical laboratory measures, vital signs, and ECG
Time frame: 12 weeks
SAT-3247 effects on muscle strength
change from baseline in muscle force as determined by dynamometry
Time frame: 12 weeks
SAT-3247 effects on muscle quality
change from baseline in intramuscular fat fraction in quantitative magnetic resonance in vastus lateralis (thigh muscle)
Time frame: 12 weeks
SAT-3247 effects on muscle function
changes from baseline in north star ambulatory assessment
Time frame: 12 weeks
SAT-3247 effects on muscle function
changes from baseline in stride velocity 95th Centile
Time frame: 12 weeks
SAT-3247 effects on muscle regeneration
changes from baseline in the regeneration index as measured from an open biopsy of biceps brachii
Time frame: 12 weeks
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