A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients
Satellos Bioscience, Inc.
Summary
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.
Description
This is a global 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. One dose of SAT-3247 and placebo will be studied in the US and Canada; two doses of SAT-3247 and placebo will be studied in UK, EU, Serbia, and Australia. Enrollment of up to 51 ambulatory DMD participants aged ≥ 7 and \< 10 years of age is planned globally. Randomization will be stratified by baseli…
Eligibility
- Age range
- 7–9 years
- Sex
- Male
- Healthy volunteers
- No
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 dose…
Interventions
- DrugSAT-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.
- DrugPlacebo
matching placebo oral tablets
Locations (25)
- University of California Los AngelesLos Angeles, California
- Colorado Children'sAurora, Colorado
- Lurie Children'sChicago, Illinois
- University of KansasKansas City, Kansas
- Kennedy Krieger InstituteBaltimore, Maryland
- UMass Memorial Medical CenterWorcester, Massachusetts