Study of Eteplirsen in DMD Patients

• Status: Completed
• Trial end date: 2019-06-14
• Target enrollment: 0
• Participant gender: Male

Summary

The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sarepta Therapeutics; Sarepta Therapeutics, Inc.

Criteria

Inclusion Criteria:

• Male 7-16 years old

• Diagnosed with DMD, genotypically confirmed

• Stable dose of corticosteroids for at least 24 weeks

• Have intact right and left alternative upper muscle groups

• Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters)

• Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and LVEF of greater than 50%

Exclusion Criteria:

• Previous treatment with drisapersen or any other RNA antisense agent or any gene therapy within the last 6 months

• Participation in any other DMD interventional clinical study within 12 weeks

• Major surgery within 3 months

• Presence of other clinically significant illness

• Major change in the physical therapy regime within 3 months

Other inclusion/exclusion criteria apply.