Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients (BASECAMP)

May 22, 2026 updated by: Satellos Bioscience, Inc.

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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).

Study Type

Interventional

Enrollment (Estimated)

51

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
  • Belgium
    • Liège
      • Liège, Liège, Belgium, 4000
    • Oost-Vlaanderen
      • Ghent, Oost-Vlaanderen, Belgium, 9000
  • Canada
    • Ontario
      • Ottawa, Ontario, Canada, K1H8L1
  • Poland
    • Pomeranian Voivodeship
      • Gdansk, Pomeranian Voivodeship, Poland, 80-952
        • Not yet recruiting
        • Klinika Neurologii Rozwojowej Uniwersyteckie
        • Contact:
    • Łódź Voivodeship
      • Lodz, Łódź Voivodeship, Poland, 93-338
  • Serbia
    • Serbia
      • Belgrade, Serbia, Serbia, 11000
        • Not yet recruiting
        • Clinic of Neurology and Psychiatry for Children and Youth
        • Contact:
      • Belgrade, Serbia, Serbia, 11000
      • Belgrade, Serbia, Serbia, 11070
        • Not yet recruiting
        • Mother and Child Health Care Institute
        • Contact:
  • Spain
      • Barcelona, Spain, 08035
        • Recruiting
        • Hospital Infantil i Hospital de la Dona
        • Contact:
    • Basque Country
      • Donostia / San Sebastian, Basque Country, Spain, 20014
    • Valencia
      • Valencia, Valencia, Spain, 46026
        • Not yet recruiting
        • Hospital Universitario y Politécnico La Fe
        • Contact:
  • United Kingdom
    • UK
      • London, UK, United Kingdom, WC1N 3JH
  • United States
    • California
      • Los Angeles, California, United States, 90095
    • Colorado
    • Illinois
      • Chicago, Illinois, United States, 60611
    • Massachusetts
      • Worcester, Massachusetts, United States, 01655
    • Missouri
    • Ohio
    • Washington

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: SAT-3247 60 mg
SAT-3247 60 mg oral tablets administered daily for 12 weeks
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.
Active Comparator: SAT-3247 120 mg
SAT-3247 120 mg oral tablets administered daily for 12 weeks; note the 120 mg dose will not be studied in the US and Canada
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.
Placebo Comparator: placebo
placebo oral tablets administered daily for 12 weeks
Drug: Placebo
matching placebo oral tablets

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety of SAT-3247
Time Frame: 12 weeks
Occurrence of treatment emergent adverse events and relationship to investigational product
12 weeks
Tolerability of SAT-3247
Time Frame: 12 weeks
occurrence of clinically significant changes in physical exam, clinical laboratory measures, vital signs, and ECG
12 weeks
SAT-3247 effects on muscle strength
Time Frame: 12 weeks
change from baseline in muscle force as determined by dynamometry
12 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
SAT-3247 effects on muscle quality
Time Frame: 12 weeks
change from baseline in intramuscular fat fraction in quantitative magnetic resonance in vastus lateralis (thigh muscle)
12 weeks
SAT-3247 effects on muscle function
Time Frame: 12 weeks
changes from baseline in north star ambulatory assessment
12 weeks
SAT-3247 effects on muscle function
Time Frame: 12 weeks
changes from baseline in stride velocity 95th Centile
12 weeks
SAT-3247 effects on muscle regeneration
Time Frame: 12 weeks
changes from baseline in the regeneration index as measured from an open biopsy of biceps brachii
12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Satellos Bioscience, Inc.

Investigators

  • Study Director: Satellos Chief Medical Officer, Satellos Bioscience, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 8, 2025

Primary Completion (Estimated)

March 31, 2027

Study Completion (Estimated)

June 30, 2027

Study Registration Dates

First Submitted

December 11, 2025

First Submitted That Met QC Criteria

December 11, 2025

First Posted (Actual)

December 17, 2025

Study Record Updates

Last Update Posted (Actual)

May 27, 2026

Last Update Submitted That Met QC Criteria

May 22, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SAT-3247-CL-201
  • 2025-522522-13-01 (Ctis)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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