NCT07287189

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 51 (estimated)
• Sponsor: Satellos Bioscience, Inc. · Industry
• Sex: Male
• Age: 7 Years – 9 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Duchenne Muscular Dystrophy
• Duchenne
• DMD
• Neuromuscular Diseases
• Muscular Dystrophies

Interventions

Type	Name	Description
DRUG	SAT-3247	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.
DRUG	Placebo	matching placebo oral tablets

Timeline

• Start date: 2025-12-08
• Primary completion: 2027-03-31
• Completion: 2027-06-30
• First posted: 2025-12-17
• Last updated: 2026-04-15

Locations

25 sites across 8 countries: United States, Australia, Belgium, Canada, Poland, Serbia, Spain, United Kingdom

Regulatory

• FDA-regulated drug study