Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2018-12-17
• Target enrollment: 0
• Participant gender: Male

Summary

This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

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

Criteria

Inclusion Criteria:

• Male 4-6 years of age.

• Diagnosis of DMD, genotypically confirmed.

• Stable dose of oral corticosteroids for at least 12 weeks or has not received corticosteroids for at least 12 weeks.

• Intact right and left biceps muscles or two alternative upper arm muscle groups.

• Parent that is willing to provide consent and comply with study procedures.

Exclusion Criteria:

• Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids).

• Previous or current treatment with any other experimental treatments within 12 weeks or participation in any other clinical trial within 6 months.

• Major surgery within 3 months prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study activities.

• Presence of other clinically significant illness.