Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

NCT07287189 | Active Not Recruiting Phase 2 DMC FDA Drug

• 3 other identifiers: SAT-3247-CL-2012025-522522-13-012025
• Study Type: interventional
• Target: 51
• Locations: 8 countries
• Sites: 20

Brief Summary

Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \< 10 years. The trial has two parts. In Part 1, 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. In Part 2, the trial will study two doses of SAT-3247 for an additional 9 months.

Conditions

DMD; Neuromuscular Diseases; Duchenne; Duchenne Muscular Dystrophy; Muscular Dystrophies

Keywords

muscle regeneration; satellite cell; asymmetric division; dystrophin

Outcome Measures

Primary Outcomes (3)

• Safety of SAT-3247

  Occurrence of treatment emergent adverse events and relationship to investigational product

  12 weeks in Part 1 and up to 12 months in part 2

• Tolerability of SAT-3247

  occurrence of clinically significant changes in physical exam, clinical laboratory measures, vital signs, and ECG

  12 weeks in Part 1 and up to 12 months in Part 2

• SAT-3247 effects on muscle strength

  change from baseline in muscle force as determined by dynamometry

  12 weeks in Part 1 and up to 12 months in Part 2

Secondary Outcomes (4)

• SAT-3247 effects on muscle quality

  12 weeks in Part 1 and up to 12 months in Part 2

• SAT-3247 effects on muscle function

  12 weeks in Part 1 and up to 12 months in Part 2

• SAT-3247 effects on muscle function

  12 weeks in Part 1 and up to 12 months in Part 2

• SAT-3247 effects on muscle regeneration

  12 weeks

Study Arms (3)

SAT-3247 60 mg

ACTIVE COMPARATOR

Part 1: SAT-3247 60 mg oral tablets administered daily for 12 weeks. Part 2: SAT-3247 60 mg oral tablets administered daily for an additional 9 months after completing Part 1

Drug: SAT-3247

SAT-3247 120 mg

ACTIVE COMPARATOR

Part 1: SAT-3247 120 mg oral tablets administered daily for 12 weeks. Part 2: SAT-3247 120 mg oral tablets administered daily for an additional 9 months after completing Part 1; note the 120 mg dose will not be studied in the US and Canada

Drug: SAT-3247

placebo

PLACEBO COMPARATOR

Part 1: placebo oral tablets administered daily for 12 weeks

Drug: Placebo

Interventions

Placebo DRUG

matching placebo oral tablets

placebo

SAT-3247 DRUG

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.

SAT-3247 120 mg; SAT-3247 60 mg

Eligibility Criteria

• Age: 7 Years - 9 Years
• Gender Eligibility Details: gender assigned at birth
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Satellos Bioscience, Inc.: lead

Study Sites (20)

University of California Los Angeles

Los Angeles, California, 90095, United States

Location

Colorado Children's

Aurora, Colorado, 80045, United States

Location

Lurie Children's

Chicago, Illinois, 60611, United States

Location

UMass Memorial Medical Center

Worcester, Massachusetts, 01655, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Seattle Children's

Seattle, Washington, 98105, United States

Location

Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia

Location

Royal Children's Hospital Melbourne

Melbourne, Victoria, 3052, Australia

Location

Hôpital De La Citadelle (CHR)

Liège, Liège, 4000, Belgium

Location

UZ Gent

Ghent, Oost-Vlaanderen, 9000, Belgium

Location

Children's Hospital Eastern Ontario

Ottawa, Ontario, K1H8L1, Canada

Location

Klinika Neurologii Rozwojowej Uniwersyteckie

Gdansk, Pomeranian Voivodeship, 80-952, Poland

Location

Instytut Centrum Zdrowia Matki Polki

Lodz, Łódź Voivodeship, 93-338, Poland

Location

University Children's Clinic Tirsova

Belgrade, Serbia, 11000, Serbia

Location

Mother and Child Health Care Institute

Belgrade, Serbia, 11070, Serbia

Location

Hospital Universitario Donostia

Donostia / San Sebastian, Basque Country, 20014, Spain

Location

Hospital Universitario y Politécnico La Fe

Valencia, Valencia, 46026, Spain

Location

Hospital Infantil i Hospital de la Dona

Barcelona, 08035, Spain

Location

Great Ormond Street

London, UK, WC1N 3JH, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Neuromuscular Diseases; Muscular Dystrophies

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Satellos Chief Medical Officer

  Satellos Bioscience, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: double-blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: randomized, placebo-controlled, 2-dose comparison

Study Record Dates

• First Submitted: December 11, 2025
• First Posted: December 17, 2025
• Study Start: December 8, 2025
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): February 28, 2028
• Last Updated: October 2, 2026

Record last verified: 2026-08

Locations

AU (2); CA (1); ES (3); BE (2); PL (2); GB (1); US (7); SE (2)