SAT-3247 OXALATE for Duchenne muscular dystrophy

Stratification factors

• Prior DMD treatments (gene therapy, exon skipper, givinostat, or none)
• Baseline corticosteroid regimen (either daily or weekend dose regimen)

Inclusion criteria

• {"criterion_text":"- Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene\n- If participant is taking (or has taken within 4 weeks prior to enrollment) herbal remedies and supplements which can impact muscle strength and function (e.g., Co-enzyme Q10, creatine, etc.), these are maintained on a stable regimen for the duration of the trial.\n- 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. a.\tEnrollment of participants previously treated with gene therapy, whether delandistrogene moxeparvovec or another investigational gene therapy product, will be capped at 25% and stratified between cohorts\n- 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.\n- 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.\n- Male DMD patients who are ambulatory and aged ≥ 7 to < 10 years at the time of screening\n- Completed two four-stair climb assessments at Visit 1 with a mean of 8 seconds or less (≤1 s variance).\n- Have a time to rise of at least 3 seconds but less than 10 seconds.\n- Healthy, as determined by investigator including medical history, psychiatric history, and no clinically significant findings on physical examination, laboratory tests, and cardiac monitoring.\n- Ability for participant and caregiver to communicate satisfactorily with the investigator and to participate in, and comply with the requirements of, the entire trial including scheduled visits, procedures/assessments, questionnaires, laboratory tests, and study restrictions\n- Participant’s parent(s) or legal guardian(s) have provided written consent to participate after reading the information and consent form, and after having the opportunity to discuss the trial with the investigator or their delegate; participants will be asked to give written or verbal assent according to local requirements.\n- Stable dose of daily 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. a.\tPatients who are not receiving glucocorticosteroids are also eligible if stopped ≥ 3 months prior to the Screening Visit.\n- 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."}

Exclusion criteria

• {"criterion_text":"- Ambulatory patients expected to experience loss of ambulation within ≤ 12 months.\n- Participants for whom MRI or open muscle biopsy are contraindicated.\n- Surgery (e.g., stomach bypass) or medical condition that might affect absorption of medicines.\n- Have acute gastrointestinal symptoms (e.g., nausea, vomiting, diarrhea, heartburn) or acute infection (such as influenza) or a significant infection or known inflammatory process at Screening.\n- Impaired cardiac function defined as a left ventricular ejection fraction of < 50% on screening cardiac assessments (echocardiogram or MRI) or evidence of symptomatic cardiomyopathy.\n- A forced vital capacity < 60% predicted at the Screening Visit.\n- Presence or history of severe adverse reaction to any excipients (lactose monohydrate, microcrystalline cellulose, colloidal silicon dioxide, copovidone, crospovidone, sodium strearyl fumarate) in the study medication tablets.\n- Participants with any known and discernable history of substance abuse and/or dependence.\n- Participants maintained on a ten day on/ten day off corticosteroid regimen\n- 11\tOngoing participation in any other therapeutic clinical trial or follow-up study for a therapeutic intervention, prior treatment with an investigational gene therapy product (other than delandistrogene moxeparvovec) < 24 months prior to the Screening Visit, or receipt of a stable dose of an approved exon-skipping therapy or any medication indicated for DMD (other than corticosteroids including vamorolone and givinostat in accordance with exception 11b below) within 6 months prior to the Screening Visit. a.\tParticipants that have received a commercially available gene therapy product (i.e., delandistrogene moxeparvovec) > 18 months prior to screening whose muscle function tests have stabilized or demonstrated decline as determined by investigator and documented in the medical record ≥ 3 months prior to screening will be eligible. b.\tParticipants receiving a stable dose of givinostat (brand name Duvyzat) for at least18 months or longer prior to the Screening Visit will be eligible. a.\tParticipants unable to tolerate givinostat who discontinued treatment are eligible to enroll if date of last dose is ≥ 30 days from Screening date. Givinostat should not be discontinued, if tolerated, in order to meet study entry criteria. c.\tUse 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.\n- Received SAT-3247 in another study.\n- Severe behavioural or cognitive problems that preclude participation in the study, in the opinion of the investigator.\n- Positive test for hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCV) or HIV at Screening Visit.\n- Employee of the Sponsor, the CRO and/or study site or their relatives\n- Presence of acute or chronic illness or history of chronic illness (other than DMD) sufficient to invalidate participation in the trial or make it unnecessarily hazardous in the judgment of the investigator.\n- Prior or ongoing medical condition (e.g., concomitant illness, psychiatric condition, behavioral disorder), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow up would be completed, or could impair the assessment of study results."}

Primary endpoints

• {"endpoint_text":"- The primary efficacy endpoint is defined as the change from baseline in muscle force measurements as determined by dynamometry at Week 12.","definition_or_measurement_approach":"Change from baseline in muscle force measurements as determined by dynamometry at Week 12 (dynamometry measurement of muscle force)."}
• {"endpoint_text":"- Safety endpoints include incidence, severity, and relationship to SAT-3247 of adverse events as well as occurrence of clinically significant changes in physical examination, clinical laboratory measures, vital signs, ECG, and C-SSRS","definition_or_measurement_approach":"Incidence, severity and relationship to SAT-3247 of adverse events; monitoring for clinically significant changes in physical examination, clinical laboratory measures, vital signs, ECG, and Columbia-Suicide Severity Rating Scale (C-SSRS)."}

Secondary endpoints

• {"endpoint_text":"- Changes from baseline in intramuscular fat fraction in muscle quantitative magnetic resonance (qMR) in vastus lateralis at Week 12","definition_or_measurement_approach":"Change from baseline in intramuscular fat fraction measured by quantitative magnetic resonance (qMR) in vastus lateralis at Week 12."}
• {"endpoint_text":"- Changes from baseline in proton muscle transverse relaxation time (T2) in vastus lateralis at Week 12.","definition_or_measurement_approach":"Change from baseline in proton muscle transverse relaxation time (T2) measured in vastus lateralis at Week 12."}
• {"endpoint_text":"- Changes from baseline in Regeneration Index in open muscle biopsy of the biceps brachii at Week 12","definition_or_measurement_approach":"Change from baseline in Regeneration Index assessed from open muscle biopsy of biceps brachii at Week 12 (histopathology assessment)."}
• {"endpoint_text":"- Changes from baseline in function as determined by NSAA assessment at Week 12","definition_or_measurement_approach":"Change from baseline in function as measured by North Star Ambulatory Assessment (NSAA) at Week 12."}
• {"endpoint_text":"- Changes from baseline in SV95C at Week 12","definition_or_measurement_approach":"Change from baseline in SV95C at Week 12 (measurement method as defined in protocol)."}

Other endpoints

• {"endpoint_text":"- Exploratory end point: change from baseline in inflammatory cytokine profile at Week 12","definition_or_measurement_approach":"Change from baseline in inflammatory cytokine profile measured by laboratory cytokine panel at Week 12."}
• {"endpoint_text":"- Exploratory end point: change from baseline in creatine kinase at Week 12","definition_or_measurement_approach":"Change from baseline in creatine kinase (CK) measured in clinical laboratory at Week 12."}
• {"endpoint_text":"- Exploratory end point: change from baseline in maximum percent predicted forced vial capacity as measured by spirometry at Week 12","definition_or_measurement_approach":"Change from baseline in maximum percent predicted forced vital capacity measured by spirometry at Week 12."}
• {"endpoint_text":"- Exploratory end point: change in biceps brachii muscle fiber size and fiber size distribution as determined from histopathology at 12 weeks","definition_or_measurement_approach":"Change in biceps brachii muscle fiber size and distribution determined by histopathology at 12 weeks."}
• {"endpoint_text":"- Exploratory end point: change in the proportion of embryonic myosin positive fibers as determined from histopathology at 12 weeks.","definition_or_measurement_approach":"Change in proportion of embryonic myosin-positive fibers determined by histopathology at 12 weeks."}
• {"endpoint_text":"- Exploratory end point: change in the number of satellite cells as determined from histopathology at 12 weeks","definition_or_measurement_approach":"Change in number of satellite cells determined by histopathology at 12 weeks."}
• {"endpoint_text":"- Exploratory end point: change in endomysial fibrosis and adipose tissue infiltration as determined from histopathology at 12 weeks.","definition_or_measurement_approach":"Change in endomysial fibrosis and adipose tissue infiltration determined by histopathology at 12 weeks."}
• {"endpoint_text":"- Exploratory end point: change in NSAA score over 12 weeks as compared to natural history","definition_or_measurement_approach":"Change in NSAA score over 12 weeks compared with natural history data."}

Methods

• Recruitment posters and trifold materials distributed at sites and patient centers (country-specific materials available for BE, PL, ES).
• Jumo Health digital materials and licensed 'Jumo Shorts' (digital video content) and a Jumo website listed among recruitment materials (digital channel targeted to patients/caregivers).
• Interactive Adherence Posters and GIFs (digital/online materials) and email communications targeted to caregivers/patients.
• Pediatrician letters and site welcome/retention/thank-you notes used to engage clinicians and families (country-specific letters for BE/PL/ES).
• Patient brochures, quick start guides for wearable device use, and clinic-based recruitment and retention materials provided at sites.

Belgium

Earliest CTIS Part Ii Submission Date

19-12-2025

Latest Decision Or Authorization Date

30-01-2026

Processing Time Days

42

Number Of Sites

2

Number Of Participants

18

Poland

Earliest CTIS Part Ii Submission Date

02-12-2025

Latest Decision Or Authorization Date

30-01-2026

Processing Time Days

59

Number Of Sites

2

Number Of Participants

6

Spain

Earliest CTIS Part Ii Submission Date

02-12-2025

Latest Decision Or Authorization Date

17-02-2026

Processing Time Days

77

Number Of Sites

3

Number Of Participants

8

Primary sponsor

Full Name

Satellos Bioscience Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

Canada

Third parties

• {"country":"Germany","full_name":"Labor Dr. Wisplinghoff GbR","duties_or_roles":"lab safety analyses","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"participants' travel and reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Medassessment Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pharma Start LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Rules Based Medicine Inc.","duties_or_roles":"cytokine panel","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Mednet Solutions Inc.","duties_or_roles":"site payments","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"video recording","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Germany","full_name":"Clinigen Clinical Supplies Management GmbH","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"Sysnav","duties_or_roles":"wearables","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"LabConnect GmbH","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Canada","full_name":"Biospective Inc.","duties_or_roles":"Imaging","organisation_type":"Pharmaceutical company"}
• {"country":"Italy","full_name":"Innopharma S.r.l.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"Australia","full_name":"Agilex Biolabs Pty Limited","duties_or_roles":"PK","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Arup Laboratories Inc.","duties_or_roles":"CK Isoenzymes","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Diverge Translational Science Laboratory","duties_or_roles":"Biopsy sample histology; Biopsy sample collection; site training","organisation_type":"Laboratory/Research/Testing facility"}