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

study id #: NCT02667483

condition: Duchenne Muscular Dystrophy

status: active, not recruiting

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.

intervention: DS-5141b

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT02667483

last updated: July 08, 2019

start date: October 2015

estimated completion: December 2019

phase of development: Phase 1/Phase 2

size / enrollment: 7

primary outcomes:

• 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]

secondary outcomes:

• Production of exon 45-skipped dystrophin mRNA in muscle tissue [Time Frame: Week 48 of Part 2-Extension]

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 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.

sponsor: Daiichi Sankyo Co., Ltd.

investigators: Global Clinical Leader

trial center locations: Japan