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NCT07287189PHASE2Recruiting

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

Satellos Bioscience, Inc.

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

Key Inclusion Criteria: * Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene. * Male DMD patients who are ambulatory and aged ≥ 7 to \< 10 years at the time of screening. * Stable dose of systemic glucocorticoids (i.e., prednisolone, deflazacort, or vamorolone) according to the standard of care for ≥ 3 months prior to the Screening Visit and for the duration of the trial. Patients who are not receiving glucocorticosteroids are also eligible if stopped ≥ 3 months prior to the Screening Visit. * Stable doses of prescription medicines including ACE inhibitors, β-blockers, and diuretics (excluding glucocorticosteroids) and over-the-counter medicines and/or herbal supplements for supportive care ≥ 1 month prior to the Screening Visit and for the duration of the trial. * Participants that have previously received delandistrogene moxeparvovec (brand name Elevidys) either in a prior clinical trial or in the commercial setting \> 18 months prior to screening whose muscle function tests have stabilized or demonstrated decline ≥ 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible. * Participants that have previously received an exon skipper \> 6 months prior to Screening whose muscle function tests have stabilized or demonstrated decline ≥ 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible. * Participants receiving a stable dose of givinostat (brand name Duvyzat) for at least 18 months or longer prior to the Screening Visit will be eligible. Participants unable to tolerate givinostat who discontinued treatment before 18 months are eligible to enroll if date of last dose is ≥ 30 days from the Screening date. Givinostat should not be discontinued, if tolerated, to meet study entry criteria. * Participants that have received prior treatment with an investigational gene therapy product (other than delandistrogene moxeparvovec) ≥ 24 months prior to the Screening Visit. * If participating in a physical therapy/strength training regimen, must be stable for ≥ 2 months prior to the Screening Visit and for the duration of the trial. Key Exclusion Criteria: * Ambulatory patients expected to experience loss of ambulation within ≤ 12 months. * Participants for whom MRI or open muscle biopsy are contraindicated. * Evidence of significant hepatic dysfunction, defined as GLDH \> 2X upper limit of normal (ULN) at the Screening Visit. * Impaired cardiac function defined as a left ventricular ejection fraction of \< 50% on screening cardiac assessments (echocardiogram or MRI) or evidence of symptomatic cardiomyopathy. * A forced vital capacity \< 60% predicted at the Screening Visit. * Ongoing participation in any other therapeutic clinical trial or follow-up study for a therapeutic intervention * Consumption of grapefruit juice or grapefruit containing products * Severe behavioural or cognitive problems that preclude participation in the study, in the opinion of the investigator. Additional entry criteria will be reviewed with the clinical site investigator.

Interventions

DRUG

SAT-3247

DRUG

Placebo

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Conditions

Duchenne Muscular DystrophyDuchenneDMDNeuromuscular DiseasesMuscular Dystrophies

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