Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2020-10-20
• Target enrollment: 0
• Participant gender: Male

Summary

This is a phase I/II study to evaluate the safety, tolerability, efficacy, and pharmacokinetic (PK) profile of DS-5141b in patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping and to determine the dosage for subsequent studies.

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

Details

• Lead Sponsor: Daiichi Sankyo Co., Ltd.
• Collaborator: Orphan Disease Treatment Institute Co., Ltd.

Criteria

Inclusion Criteria:

• Confirmation of out-of-frame deletion(s) that could be corrected by dystrophin gene exon 45 skipping.

• Intact muscles of adequate quality for biopsy to allow evaluation of the efficacy of the study drug.

• Boys aged from 5 years to <11 years.

• Patients able to walk at least 325 meters in the 6-minutes walk test.

• Glucocorticoid-naive patients, or patients who have used glucocorticoids for at least 6 months prior to enrollment in this study with no dose changes for at least 3 months prior to enrollment.

Exclusion Criteria:

• A genetic mutation that can not be expected the expression of dystrophin protein by dystrophin gene exon 45 skipping.

• A concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function.

• Current or history of severe disorder.

• Left ventricular ejection fraction (LEVF) <55%.

• Corrected QT interval (QTc) >0.45 sec.