Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping

• Status: Completed
• Trial end date: 2021-03-10
• Target enrollment: 0
• Participant gender: Male

Summary

This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, PK, and efficacy of once-weekly IV infusions of eteplirsen in approximately 12 male patients, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping.

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

Details

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

Criteria

Inclusion Criteria:

• Male between 6 months to 48 months of age (inclusive)

• Diagnosis of DMD with a deletion mutation amenable to exon 51 skipping

• Parent(s) or legal guardian(s) who is willing to provide written informed consent

Exclusion Criteria:

• Received treatment that might have an effect on muscle strength or function within 12 weeks prior to dosing

• Received previous or current treatment with any experimental treatment

• Clinically significant illness other than DMD

• Clinically significant laboratory abnormality

• Any other condition that could interfere with the patient's participation