Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
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
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
Treatments tested in this trial
- SAT-3247
- Placebo
Treatment groups
51 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov