Phase I/II Study of DS-5141b

Phase 1

Completed

• Conditions: Duchenne muscular dystrophy

• Registration Number: JPRN-jRCT2080223018
• Lead Sponsor: DAIICHISANKYO Co.,Ltd.

Brief Summary

The safety, tolerability, efficacy, and pharmacokinetics of DS-5141b in 8 subjects with DMD were evaluated. No safety concerns were found at either the DS-5141b 2.0 mg/kg or 6.0 mg/kg dose, indicating that the drug was well tolerated. Plasma DS-5141a concentrations generally increased dose-proportionally within the assessed DS-5141b dose range, with only minor cumulative Cmax and AUC168h at steady state. A certain level of efficacy was found in subjects with DMD.

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-11-18
• Study Completion: 2020-10-20
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: completed
• Sex: Male
• Target Recruitment: 8

Inclusion Criteria

1)Confirmation of out-of-frame deletion(s) that could be corrected by dystrophin gene exon 45 skipping.
2)Intact muscles of adequate quality for biopsy to allow evaluation of the efficacy of the study drug.
3)Boys aged from 5 years to <11 years.
4)Patients able to walk at least 325 meters in the 6-minutes walk test.
5)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

1)A genetic mutation that can not be expected the expression of dystrophin protein by dystrophin gene exon 45 skipping.
2)A concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function.
3)Current or history of severe disorder.
4)Left ventricular ejection fraction (LEVF) <55%.
5)Corrected QT interval (QTc) >0.45 sec.

Study & Design

• Study Type: Interventional
• Study Design: Not specified