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Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
study id #: NCT02667483
condition: Duchenne Muscular Dystrophy
status: active, not recruitingpurpose:
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.
mechanism of action: Exon-skipping to promote dystrophin production
start date: October 2015
estimated completion: December 2019
phase of development: Phase 1/Phase 2
size / enrollment: 7
- Number of participants with treatment-emergent adverse events (TEAEs) by the end of the trial [Time Frame: 48 Weeks of Part 2-Extension]
TEAEs are adverse events (including clinically significant laboratory values) temporally associated with use of DS-5141b, whether or not attributable to the product.
- Maximum concentration (Cmax) of DS-5141b [Time Frame: Week 48 of Part 2-Extension]
- Area under the curve (AUC) for DS-5141b [Time Frame: Week 48 of Part 2-Extension]
- Time to maximum concentration (Tmax) of DS-5141b [Time Frame: Week 48 of Part 2-Extension]
- Half-life (T1/2) of DS-5141b [Time Frame: Week 48 of Part 2-Extension]
- Dystrophin protein expression in muscle tissue [Time Frame: Week 48 of Part 2-Extension]
- Production of exon 45-skipped dystrophin mRNA in muscle tissue [Time Frame: Week 48 of Part 2-Extension]
• 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
• A genetic mutation that cannot 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.
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