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Trial record 32 of 1002 for:    BMD

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02667483
Recruitment Status : Active, not recruiting
First Posted : January 29, 2016
Last Update Posted : April 2, 2019
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 April 2, 2019
Actual Study Start Date  ICMJE October 2015
Estimated Primary Completion Date December 2019   (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 Complete list of historical versions of study NCT02667483 on ClinicalTrials.gov Archive Site
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 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 Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: May 24, 2018)
7
Original Estimated Enrollment  ICMJE
 (submitted: January 28, 2016)
6
Estimated Study Completion Date  ICMJE December 2019
Estimated Primary Completion Date December 2019   (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: Yes
Plan Description: De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/
Supporting Materials: Study Protocol
Supporting Materials: Statistical Analysis Plan (SAP)
Supporting Materials: Clinical Study Report (CSR)
Time Frame: Studies for which the medicine and indication have received European Union (EU) and United States (US), and/or Japan (JP) marketing approval on or after 01 January 2014 or by the US or EU or JP Health Authorities when regulatory submissions in all regions are not planned and after the primary study results have been accepted for publication.
Access Criteria: Formal request from qualified scientific and medical researchers on IPD and clinical study documents from clinical trials supporting products submitted and licensed in the United States, the European Union and/or Japan from 01 January 2014 and beyond for the purpose of conducting legitimate research. This must be consistent with the principle of safeguarding study participants' privacy and consistent with provision of informed consent.
URL: https://vivli.org/ourmember/daiichi-sankyo/
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 May 2018

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