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Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

ConditionsDuchenne Muscular DystrophyDuchenneDMDNeuromuscular DiseasesMuscular Dystrophies
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexMale
Age7-9
SponsorSatellos Bioscience, Inc.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Parallel

Treatments tested in this trial

  • 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.

  • Placebo

    Drug

    matching placebo oral tablets

Treatment groups

51 Participants
are divided into 3 treatment groups
Group A: SAT-3247 60 mgActive comparator 1 intervention
Group B: SAT-3247 120 mgActive comparator 1 intervention
Group C: placeboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Safety of SAT-3247

Occurrence of treatment emergent adverse events and relationship to investigational product

Time frame
12 weeks
2

Tolerability of SAT-3247

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

Time frame
12 weeks
3

SAT-3247 effects on muscle strength

change from baseline in muscle force as determined by dynamometry

Time frame
12 weeks

Secondary outcomes

1

SAT-3247 effects on muscle quality

change from baseline in intramuscular fat fraction in quantitative magnetic resonance in vastus lateralis (thigh muscle)

Time frame
12 weeks
2

SAT-3247 effects on muscle function

changes from baseline in north star ambulatory assessment

Time frame
12 weeks
3

SAT-3247 effects on muscle function

changes from baseline in stride velocity 95th Centile

Time frame
12 weeks
4

SAT-3247 effects on muscle regeneration

changes from baseline in the regeneration index as measured from an open biopsy of biceps brachii

Time frame
12 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Satellos Bioscience, Inc.

Lead sponsor

Source ClinicalTrials.gov
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