The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males with DMD aged greater than or equal to (\>=) 2 to lesser than (\<) 5 years. The study will comprise of 2 periods (Period 1: 52-week safety and pharmacokinetics \[PK\], and Period 2: 52-week extension). Participants will be randomized in a 1:1 ratio to one of 2 treatment arms: 0.9 mg/kg deflazacort, and 0.45 mg/kg of deflazacort. A historic control group (which should match the study population as closely as possible) will be used as a comparator to characterize the safety and tolerability of deflazacort.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Deflazacort tablets will be administered as per schedule and dose specified in respective arms.
Period 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: 52 weeks
Period 1 and 2: Change From Baseline in Vital Signs and Electrocardiogram (ECG) at Week 52
Time frame: Baseline, Week 52
Period 1 and 2: Change From Baseline in the Child Behavior Checklist Score at Week 52
Time frame: Baseline, Week 52
Period 1 and 2: Change From Baseline in the Normalized Measure of Bone Density Change (Z-score) for the Dual Energy X-ray Absorptiometry (DEXA) at Week 52
Time frame: Baseline, Week 52
Period 1 and 2: Mean Change From Baseline in Height at Week 52
Time frame: Baseline, Week 52
Period 1 and 2: Mean Change From Baseline in Body Weight at Week 52
Time frame: Baseline, Week 52
Period 1 and 2: Mean Change From Baseline in Height Percentile for Age at Week 52
Time frame: Baseline, Week 52
Period 1 and 2: Number of Participants With Clinically Significant Laboratory Tests
Time frame: 52 weeks
Period 1: Peak Plasma Concentration (Cmax) of Deflazacort
Time frame: Pre-dose, 0.25, 2, 4, and 6 hours post-dose at Baseline (Week 1) and Week 13
Period 1: Area Under the Curve (AUC) of Deflazacort
Time frame: Pre-dose, 0.25, 2, 4, and 6 hours post-dose at Baseline (Week 1) and Week 13
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Period 1: Volume of Distribution (Vd) of Deflazacort
Time frame: Pre-dose, 0.25, 2, 4, and 6 hours post-dose at Baseline (Week 1) and Week 13
Period 1: Clearance (CL) of Deflazacort
Time frame: Pre-dose, 0.25, 2, 4, and 6 hours post-dose at Baseline (Week 1) and Week 13