Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
Start Date
12/8/2025
Completion Date
6/30/2027
Summary
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.
Detailed Description
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).
Eligibility Criteria
Age Range: 7 years to 9 years
Interventions
SAT-3247
Placebo
Conditions
Locations
University of California Los Angeles
Los Angeles, California 90095
United States
Colorado Children's
Aurora, Colorado 80045
United States
Lurie Children's
Chicago, Illinois 60611
United States
UMass Memorial Medical Center
Worcester, Massachusetts 01655
United States
Washington University
St Louis, Missouri 63110
United States
Nationwide Children's Hospital
Columbus, Ohio 43205
United States
Seattle Children's
Seattle, Washington 98105
United States
Children's Hospital at Westmead
Westmead, New South Wales 2145
Australia
Royal Children's Hospital Melbourne
Melbourne, Victoria 3052
Australia
Hôpital De La Citadelle (CHR)
Liège, Liège 4000
Belgium
UZ Gent
Ghent, Oost-Vlaanderen 9000
Belgium
Children's Hospital Eastern Ontario
Ottawa, Ontario K1H8L1
Canada
Klinika Neurologii Rozwojowej Uniwersyteckie
Gdansk, Pomeranian Voivodeship 80-952
Poland
Instytut Centrum Zdrowia Matki Polki
Lodz, Łódź Voivodeship 93-338
Poland
Clinic of Neurology and Psychiatry for Children and Youth
Belgrade, Serbia 11000
Serbia
University Children's Clinic Tirsova
Belgrade, Serbia 11000
Serbia
Mother and Child Health Care Institute
Belgrade, Serbia 11070
Serbia
Hospital Universitario Donostia
Donostia / San Sebastian, Basque Country 20014
Spain
Hospital Universitario y Politécnico La Fe
Valencia, Valencia 46026
Spain
Hospital Infantil i Hospital de la Dona
Barcelona, 08035
Spain
Great Ormond Street
London, UK WC1N 3JH
United Kingdom