Not Yet RecruitingPhase 2ACTRN12625001370493

A Study to Test the Safety and How Well a New Medicine (SAT-3247) Works in Children with Duchenne Muscular Dystrophy Who Can Walk Unassisted – BASECAMP

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

Sponsor

Satellos Bioscience, Inc.

Enrollment

51 participants

Start Date

Dec 15, 2025

Study Type

Interventional

Conditions

Duchenne Muscular Dystrophy

Summary

This is a phase 2a trial of SAT-3247 in ambulatory DMD patients aged from 7 and less than 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. Enrollment of up to 51 ambulatory DMD participants aged from 7 and less than 10 years of age is planned. 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), Week 4 (Visit 3), Week 8 (Visit 4), and Week 12 (Visit 5) follow-up visits.

Eligibility

Sex: MalesMin Age: 7 YearssMax Age: 9 Yearss

Inclusion Criteria7

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, ß-blockers, and diuretics (excluding glucocorticosteroids) and over-the-counter medicines and/or herbal supplements for supportive care a 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 declined =/> 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible.
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.

Exclusion Criteria12

Ambulatory patients expected to experience loss of ambulation within < /= 12 months.
Participants for whom MRI or open muscle biopsy are contraindicated.
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
Prior treatment with an investigational gene therapy product < 24 months prior to the Screening Visit
Receipt of a stable dose of an approved exon-skipping therapy or any medication indicated for DMD (other than corticosteroids including vamorolone) < 6 months prior to the Screening Visit.
Participants that have received a commercially available gene therapy product (i.e., delandistrogene moxeparvovec (brand name Elevidys)) < 18 months prior to screening.
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 equal or more than 30 days from Screening date. Givinostat should not be discontinued, if tolerated, in order to meet study entry criteria.
Use of deflazacort (brand name Emflaza) or vamorolone (brand name Agamree) in jurisdictions where these are investigational as they have not received health authority marketing authorization will not be exclusionary; however, simultaneous participation in a clinical trial of deflazacort or vamorolone will be excluded.
Severe behavioural or cognitive problems that preclude participation in the study, in the opinion of the investigator.

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Interventions

Treatment: Drugs - SAT-3247 Experimental: Treatment Arm - SAT-3247 60 mg administered orally for 12 weeks in a 5-days on/2-days off (weekday) dosing regimen Treatment Arm - SAT-3247 120 mg admin

Treatment: Drugs - SAT-3247 Experimental: Treatment Arm - SAT-3247 60 mg administered orally for 12 weeks in a 5-days on/2-days off (weekday) dosing regimen Treatment Arm - SAT-3247 120 mg administered orally for 12 weeks in a 5-days on/2-days off (weekday) dosing regimen The treatment, for all groups, is formulated as oral tablets. The participant should return the blister provided in order the Investigator can check the adherence to the treatment by counting the returned tablets.