Actively Recruiting
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
Led by Satellos Bioscience, Inc. · Updated on 2026-05-14
51
Participants Needed
21
Research Sites
81 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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 less than 10 years with a confirmed diagnosis of Duchenne Muscular Dystrophy by clinical findings and genetic testing
- Ambulatory patients
- Stable dose of systemic glucocorticoids for at least 3 months prior to screening or stopped glucocorticoids at least 3 months before screening
- Stable doses of prescription medicines (excluding glucocorticoids) and over-the-counter medicines or supplements for at least 1 month prior to screening
- Previous treatment with delandistrogene moxeparvovec at least 18 months prior with stable or declining muscle function for at least 3 months prior to screening
- Previous treatment with an exon skipper at least 6 months prior with stable or declining muscle function for at least 3 months prior to screening
- Stable dose of givinostat for at least 18 months prior to screening or discontinued givinostat at least 30 days prior if intolerant
- Prior investigational gene therapy (other than delandistrogene moxeparvovec) at least 24 months prior
- Stable physical therapy or strength training regimen for at least 2 months prior to screening
You will not qualify if you...
- Ambulatory patients expected to lose ability to walk within 12 months
- Contraindications to MRI or open muscle biopsy
- Significant liver dysfunction with GLDH greater than twice the upper limit of normal at screening
- Impaired heart function with left ventricular ejection fraction below 50% or symptomatic cardiomyopathy
- Forced vital capacity less than 60% predicted at screening
- Participation in another therapeutic clinical trial or follow-up study
- Consumption of grapefruit juice or grapefruit-containing products
- Severe behavioral or cognitive problems preventing study participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
Not Yet 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
Not Yet 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
CONTACT
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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