DMD muscular dystrophy
Conditions
Interventions
Drug: Eteplirsen
• Solution for intravenous (IV) infusion.
• Other Names:
• AVI-4658
• EXONDYS 51
• EXONDYS
Sponsors
Sarepta Therapeutics, Inc.
Eligibility
Age
2 Years to 15 Years
Inclusion criteria
Inclusion criteria: Inclusion Criteria: • Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping. • Ambulatory participant, able to perform TTRISE in 10 seconds or less at the time of screening visit. • Able to walk independently without assistive devices. • Have intact right and left biceps muscles or an alternative upper arm muscle group. • Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines throughout the study. • For ages 7 years and older, has stable pulmonary function (forced vital capacity >=50 percent (%) of predicted and no requirement for nocturnal ventilation). For ages 4 to 6 years, does not require support from ventilator or non-invasive ventilation at time of screening.
Exclusion criteria
Exclusion criteria: Exclusion Criteria: • Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization. • Current or previous treatment with any other experimental pharmacologic treatment for DMD or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; except the following: Ezutromid in the last 12 weeks prior to first dose; Drisapersen in the last 36 weeks prior to first dose; Suvodirsen in the last 12 weeks prior to first dose; Vamorolone in the last 12 weeks prior to first dose; and Eteplirsen (previous or current use). • Major surgery within 3 months prior to randomization. • Presence of any other significant neuromuscular or genetic disease other than DMD. • Presence of any known impairment of renal function and/or other clinically significant illness. • Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction less than =450 millisecond based on the screening electrocardiograms (ECGs). Other inclusion/exclusion criteria apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part 1: Incidence of Adverse Events (AEs) Time Frame: Up to Week 148 Part 2: Change From Baseline in the NSAA Total Score at Week 144 Time Frame: Baseline, Week 144 | — |
Secondary
| Measure | Time frame |
|---|---|
| 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 Baseline. 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 in Forced Vital Capacity Percent Predicted (FVC%p) at Week 144 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 | — |
Countries
Netherlands
Outcome results
None listed