Actively Recruiting
A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study Comparing Doses of Oral SAT-3247 in Ambulatory Boys with Duchenne Muscular Dystrophy Aged 7 to Under 10 Years
Led by Satellos Bioscience, Inc. · Updated on 2026-06-05
51
Participants Needed
21
Research Sites
13 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating SAT-3247, a selective oral drug, in ambulatory male patients with Duchenne Muscular Dystrophy (DMD) aged 7 to under 10 years. This global phase 2a trial aims to identify the best dose, safety, tolerability, and initial effects of SAT-3247 on muscle strength and function compared to placebo. The study includes participants from the US, Canada, UK, EU, Serbia, and Australia, and is sponsored by Satellos Bioscience, Inc. Participants will be randomly assigned to receive either a low dose (60 mg) or high dose (120 mg) of SAT-3247, or a matching placebo, taken orally once daily on weekdays for 12 weeks. The 120 mg dose is not given in the US and Canada. The trial is double-blind, meaning neither participants nor researchers know who receives which dose or placebo. Treatment groups are balanced based on prior corticosteroid use and other medications. Before starting treatment, participants undergo screening within 28 days. Study visits occur at baseline, week 4, week 8, and week 12, with a follow-up phone call at week 1. Researchers monitor safety, tolerability, muscle strength, muscle quality, function, and regeneration throughout the 12 weeks. The trial includes assessments such as muscle tests and clinical evaluations to track effects and side effects during the study period.
CONDITIONS
Brief Title
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male patients aged 7 to under 10 years at screening
- Definitive diagnosis of Duchenne Muscular Dystrophy confirmed by clinical findings and genetic testing
- Ambulatory patients able to walk
- Stable dose of systemic glucocorticoids for at least 3 months prior to screening or stopped steroids at least 3 months before screening
- Stable doses of supportive prescription or over-the-counter medicines for at least 1 month before screening
- Previous treatments with certain gene therapies or exon skippers allowed if stable or discontinued per specified timelines
- Stable participation in physical therapy or strength training for at least 2 months before screening
- Enrollment planned for up to 51 participants globally
You will not qualify if you...
- Patients expected to lose ability to walk within 12 months
- Contraindications to MRI or open muscle biopsy
- Significant liver dysfunction at screening
- Impaired heart function or symptomatic cardiomyopathy
- Forced vital capacity less than 60% predicted at screening
- Participation in other therapeutic clinical trials or follow-up studies
- Consumption of grapefruit juice or grapefruit products
- Severe behavioral or cognitive problems preventing study participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - Up to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 12 weeks
Participants receive once daily doses of SAT-3247 or placebo for 12 weeks to assess safety, tolerability, and effects on muscle function.
1 baseline visit, 1 follow-up phone call at Week 1, and 3 in-person visits at Weeks 4, 8, and 12
Trial Site Locations
Total: 21 locations
1
University of California Los Angeles
Los Angeles, California, United States, 90095
Actively Recruiting
2
Colorado Children's
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Lurie Children's
Chicago, Illinois, United States, 60611
Actively Recruiting
4
UMass Memorial Medical Center
Worcester, Massachusetts, United States, 01655
Actively Recruiting
5
Washington University
St Louis, Missouri, United States, 63110
Actively Recruiting
6
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
7
Seattle Children's
Seattle, Washington, United States, 98105
Actively Recruiting
8
Children's Hospital at Westmead
Westmead, New South Wales, Australia, 2145
Not Yet Recruiting
9
Royal Children's Hospital Melbourne
Melbourne, Victoria, Australia, 3052
Actively Recruiting
10
Hôpital De La Citadelle (CHR)
Liège, Liège, Belgium, 4000
Not Yet Recruiting
11
UZ Gent
Ghent, Oost-Vlaanderen, Belgium, 9000
Actively Recruiting
12
Children's Hospital Eastern Ontario
Ottawa, Ontario, Canada, K1H8L1
Actively Recruiting
13
Klinika Neurologii Rozwojowej Uniwersyteckie
Gdansk, Pomeranian Voivodeship, Poland, 80-952
Not Yet Recruiting
14
Instytut Centrum Zdrowia Matki Polki
Lodz, Łódź Voivodeship, Poland, 93-338
Actively Recruiting
15
Clinic of Neurology and Psychiatry for Children and Youth
Belgrade, Serbia, Serbia, 11000
Not Yet Recruiting
16
University Children's Clinic Tirsova
Belgrade, Serbia, Serbia, 11000
Not Yet Recruiting
17
Mother and Child Health Care Institute
Belgrade, Serbia, Serbia, 11070
Not Yet Recruiting
18
Hospital Universitario Donostia
Donostia / San Sebastian, Basque Country, Spain, 20014
Not Yet Recruiting
19
Hospital Universitario y Politécnico La Fe
Valencia, Valencia, Spain, 46026
Not Yet Recruiting
20
Hospital Infantil i Hospital de la Dona
Barcelona, Spain, 08035
Actively Recruiting
21
Great Ormond Street
London, UK, United Kingdom, WC1N 3JH
Not Yet Recruiting
Research Team
S
Satellos Medical Information
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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