Part 1 (dose escalation) will evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram \[mg/kg\] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will evaluate the efficacy and safety of the high doses (100 mg/kg and 200 mg/kg) of eteplirsen compared with that of the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
160
Solution for intravenous (IV) infusion.
University of Alabama at Birmingham
Birmingham, Alabama, United States
University of Florida
Gainesville, Florida, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
Hospital Universitario San Ignacio
Bogotá, Colombia
Instituto Neurologico de Colombia (INDEC)
Medellín, Colombia
Part 1: Incidence of Adverse Events (AEs)
Time frame: Up to Week 148
Part 2: Change From Baseline at Week 144 in the NSAA Total Score (for Final Analysis)
Time frame: Baseline, Week 144
Part 2: Change from Baseline at Week 72 or Week 96 in NSAA Total Score (for Conditional Efficacy Interim Analysis)
Time frame: Baseline, Week 72 or Week 96
Part 2: Change From Baseline in Time to Rise From the Floor, Time to Complete 10-Meter Walk/Run, and the Timed Stair Ascend Test
Time frame: Baseline, Week 144
Part 2: Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT)
Time frame: Baseline, Week 144
Part 2: Change from Baseline at Week 144 in Forced Vital Capacity Percent Predicted (FVC%p)
Time frame: Baseline, Week 144
Part 2: Time to Loss of Ambulation (LOA)
Time frame: Baseline up to Week 144
Part 2: Change From Baseline in Skeletal Muscle Dystrophin Expression
Time frame: Baseline, Postdose (at Week 24, Week 48, or Week 144)
Part 2: Incidence of Adverse Events (AEs)
Time frame: Baseline up to Week 148
Part 2: Pharmacokinetic (PK) Plasma Concentration of Eteplirsen
Time frame: 0 (predose) to 2 hours postdose up to Week 144
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Hospital Pablo Tobón Uribe
Medellín, Colombia
Brno Klinika detske neurologie
Brno, Czechia
Fakultni nemocnice v Motole
Prague, Czechia
Rigshospitalet Copenhagen University Hospital
Copenhagen, Denmark
Hopital Femme Mere Enfant
Bron, France
...and 49 more locations