Phase I/II Study of SRP-4053 in DMD Patients

• Status: Completed
• Trial end date: 2019-03-25
• Target enrollment: 0
• Participant gender: Male

Summary

This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sarepta Therapeutics; Sarepta Therapeutics, Inc.
• Collaborators: Catholic University of the Sacred Heart; Consultants for Research in Imaging and Spectroscopy; Great Ormond Street Hospital for Children NHS Foundation Trust; Institut de Myologie, France; Royal Holloway University; Sysnav; University College, London; University of Newcastle Upon-Tyne

Criteria

Inclusion Criteria:

• Diagnosed with DMD, genotypically confirmed.

• Intact right and left biceps muscles or an alternative upper arm muscle group.

• Stable pulmonary and cardiac function.

• Minimum performance on 6MWT, North Star Ambulatory Assessment, and rise (Gowers) test as specified in the study protocol.

• On a stable dose of corticosteroids for at least 6 months.

Exclusion Criteria:

• Previous treatment with the experimental agents BMN-195 (SMT C1100) or PRO053.

• Current or previous treatment with any other experimental treatments within 12 weeks prior to study entry.

• Major surgery within the last 3 months.

• Presence of other clinically significant illness.

• Major change in physical therapy regime within the last 3 months.

Other inclusion and exclusion criteria may apply.