Recruiting

Phase 2

SAT-3247Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

Sponsor:

Satellos Bioscience, Inc.

Code:

NCT07287189

Conditions

Duchenne Muscular Dystrophy

Duchenne

DMD

Neuromuscular Diseases

Muscular Dystrophies

Eligibility Criteria

Sex: Male

Age: 7 - 9

Healthy Volunteers: Not accepted

Interventions

SAT-3247

Placebo

Study Details

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

Duchenne Muscular Dystrophy

Duchenne

DMD

Neuromuscular Diseases

Muscular Dystrophies

Study ID

NCT07287189

Start date

Dec 8, 2025

Status verified date

Aug, 2026

Completion date

Feb 28, 2028

Anticipated

Primary completion date

Mar 31, 2027

Anticipated

Eligibility Criteria

Sex: Male

Age: 7 - 9

Healthy Volunteers: Not accepted

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 Design

Enrollment

51 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

active comparator: SAT-3247 60 mg

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

active comparator: SAT-3247 120 mg

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

placebo comparator: placebo

Part 1: placebo oral tablets administered daily for 12 weeks

Interventions

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

matching placebo oral tablets

Primary outcome measure

Safety of SAT-3247 [ Time Frame: 12 weeks in Part 1 and up to 12 months in part 2 ]
Tolerability of SAT-3247 [ Time Frame: 12 weeks in Part 1 and up to 12 months in Part 2 ]
SAT-3247 effects on muscle strength [ Time Frame: 12 weeks in Part 1 and up to 12 months in Part 2 ]

Central Contacts and Locations

Central contacts

Satellos Medical Information

+1 647-660-1780 medicalinfo@satellos.com

Locations

University of California Los Angeles

Recruiting

Los Angeles, California, United States, 90095

Contacts

Denisse Velazquez

310-825-3264 Denissevelazquez@mednet.ucla.edu

Colorado Children's

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Nana Welnick

720-777-8608 Nanastasia.Welnick@childrenscolorado.org

Lurie Children's

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Alka Maheshwari

312-227-3813 amaheshwari@luriechildrens.org

UMass Memorial Medical Center

Recruiting

Worcester, Massachusetts, United States, 01655

Contacts

Sarah Figueira

508-856-1604 Sarah.figueira@umassmed.edu

Washington University

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Natalie Goedeker

314-362-4919 NeuromusclePediatricResearch@wustl.edu

Nationwide Children's Hospital

Recruiting

Columbus, Ohio, United States, 43205

Contacts

Jeremy Thompson

614-355-2923 jeremy.thompson@nationwidechildrens.org

Seattle Children's

Recruiting

Seattle, Washington, United States, 98105

Contacts

Marissa Robertson

206-987-5457 marissa.robertson@seattlechildrens.org

Children's Hospital Eastern Ontario

Recruiting

Ottawa, Ontario, Canada, K1H8L1

Contacts

Emilie Hill-Smith

EHillSmith@cheo.on.ca

More Information

Sponsor

Satellos Bioscience, Inc.

Last update posted

Aug 14, 2026

Last verified

Aug, 2026

Keywords

muscle regeneration
satellite cell
asymmetric division
dystrophin

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Satellos Bioscience, Inc. on 2026-08-14.