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Trial record 1 of 2 for:    DS-5141b
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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 : 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. )

Study Description
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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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: DS-5141b Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I/II Study of DS-5141b: Open-label Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Actual Study Start Date : October 2015
Actual Primary Completion Date : October 20, 2020
Actual Study Completion Date : October 20, 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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.

 Drug: DS-5141b
DS-5141b, Subcutaneous injection


Outcome Measures
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Primary Outcome Measures :
  1. 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.

  2. Maximum concentration (Cmax) of DS-5141b [ Time Frame: Week 48 of Part 2-Extension ]
  3. Area under the curve (AUC) for DS-5141b [ Time Frame: Week 48 of Part 2-Extension ]
  4. Time to maximum concentration (Tmax) of DS-5141b [ Time Frame: Week 48 of Part 2-Extension ]
  5. Half-life (T1/2) of DS-5141b [ Time Frame: Week 48 of Part 2-Extension ]
  6. Dystrophin protein expression in muscle tissue [ Time Frame: Week 48 of Part 2-Extension ]

Secondary Outcome Measures :
  1. Production of exon 45-skipped dystrophin mRNA in muscle tissue [ Time Frame: Week 48 of Part 2-Extension ]

Eligibility Criteria
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Ages Eligible for Study:   5 Years to 10 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02667483


Locations
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Japan
Kobe University Hospital
Hyogo, Kobe-shi, Japan, 650-0017
National Center of Neurology and Psychiatry
Tokyo, Kodaira-shi, Japan, 187-8551
Sponsors and Collaborators
Daiichi Sankyo Co., Ltd.
Orphan Disease Treatment Institute Co., Ltd.
Investigators
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Study Director: Global Clinical Leader Daiichi Sankyo, Inc.
More Information
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Responsible Party: Daiichi Sankyo Co., Ltd.
ClinicalTrials.gov Identifier: NCT02667483    
Other Study ID Numbers: DS5141-A-J101
153072 ( Registry Identifier: JAPIC CTI )
First Posted: January 29, 2016    Key Record Dates
Last Update Posted: December 16, 2020
Last Verified: December 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Daiichi Sankyo, Inc. ( Daiichi Sankyo Co., Ltd. ):
Duchenne muscular dystrophy
DMD
Oligonucleotides, Antisense
Exon skipping
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked