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

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ClinicalTrials.gov Identifier: NCT02667483
Recruitment Status : Completed
First Posted : January 29, 2016
Last Update Posted : December 16, 2020
Sponsor:
Collaborator:
Orphan Disease Treatment Institute Co., Ltd.
Information provided by (Responsible Party):
Daiichi Sankyo, Inc. ( Daiichi Sankyo Co., Ltd. )

Tracking Information
First Submitted Date  ICMJE January 26, 2016
First Posted Date  ICMJE January 29, 2016
Last Update Posted Date December 16, 2020
Actual Study Start Date  ICMJE October 2015
Actual Primary Completion Date October 20, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 24, 2018)
  • 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 ]
Original Primary Outcome Measures  ICMJE
 (submitted: January 28, 2016)
  • number and severity of adverse events [ Time Frame: week 0 (baseline) to week 12 ]
    number and severity of treatment emergent adverse events
  • Cmax maximum concentration [ Time Frame: Week 12 ]
    pharmacokinetic profile
  • AUC area under the curve [ Time Frame: Week 12 ]
    pharmacokinetic profile
  • Tmax time of maximum concentration [ Time Frame: Week 12 ]
    pharmacokinetic profile
  • T1/2 half-life [ Time Frame: Week 12 ]
    pharmacokinetic profile
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 23, 2018)
Production of exon 45-skipped dystrophin mRNA in muscle tissue [ Time Frame: Week 48 of Part 2-Extension ]
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Official Title  ICMJE Phase I/II Study of DS-5141b: Open-label Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Brief 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Drug: DS-5141b
DS-5141b, Subcutaneous injection
Study Arms  ICMJE Experimental: DS-5141b

DS-5141b, Subcutaneous injection

Part 1: DS-5141b will be injected subcutaneously once a week for 2 weeks at the following dose levels. Dose escalation will be performed. DS-5141b will be administered at dose levels 1 and 3 in Cohort 1 and at dose levels 2 and 4 in Cohort 2.

  • Level 1: 0.1 mg/kg
  • Level 2: 0.5 mg/kg
  • Level 3: 2.0 mg/kg
  • Level 4: 6.0 mg/kg

Part 2: Two doses of DS-5141b will be selected based on the results obtained in Part 1. Each selected dose will be administered subcutaneously once a week for 12 weeks.

Part 2-Extension: Two doses, 2.0 mg/kg or 6.0 mg/kg, of DS-5141b will be administered subcutaneously once a week for 48 weeks.

Intervention: Drug: DS-5141b
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 24, 2018)
7
Original Estimated Enrollment  ICMJE
 (submitted: January 28, 2016)
6
Actual Study Completion Date  ICMJE October 20, 2020
Actual Primary Completion Date October 20, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 5 Years to 10 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Japan
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02667483
Other Study ID Numbers  ICMJE DS5141-A-J101
153072 ( Registry Identifier: JAPIC CTI )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Daiichi Sankyo, Inc. ( Daiichi Sankyo Co., Ltd. )
Study Sponsor  ICMJE Daiichi Sankyo Co., Ltd.
Collaborators  ICMJE Orphan Disease Treatment Institute Co., Ltd.
Investigators  ICMJE
Study Director: Global Clinical Leader Daiichi Sankyo, Inc.
PRS Account Daiichi Sankyo, Inc.
Verification Date December 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP