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Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Primary Purpose

Duchenne Muscular Dystrophy

Status
Active
Phase
Phase 2
Locations
Japan
Study Type
Interventional
Intervention
DS-5141b
Sponsored by
Daiichi Sankyo Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Duchenne muscular dystrophy, DMD, Oligonucleotides, antisense, Exon skipping, DS-5141b

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • Has competed a study of DS5141-A-J101

Exclusion Criteria:

  • Significant safety issues in a study of DS5141-A-J101
  • Patient who does not consent to use appropriate contraception
  • Patient not appropriate to participant in the study as determined by the Investigator

Sites / Locations

  • National Center of Neurology and Psychiatry
  • Kobe University Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

DS-5141b 2.0 mg/kg

DS-5141b 6.0 mg/kg

Arm Description

Participants who will receive DS-5141b 2.0 mg/kg once weekly.

Participants who will receive DS-5141b 6.0 mg/kg once weekly.

Outcomes

Primary Outcome Measures

Incidence of Adverse Events (AEs)
Change in distance walked during 6-minute walk test (6MWT)
Change in time to stand (TTSTAND)
Change in time in Timed up and go test
Change in time in 10-meter Run/Walk test
Change in score in the North Star Ambulatory Assessment (NSAA)
Change in score in the Performance of Upper Limb (PUL)
Change in Left Ventricular Ejection Fraction percentage (LVEF %)
Change in in Forced Vital Capacity (FVC) (percent predicted)
Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment

Secondary Outcome Measures

Concentration of DS-5141a in plasma

Full Information

First Posted
June 12, 2020
Last Updated
July 26, 2023
Sponsor
Daiichi Sankyo Co., Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT04433234
Brief Title
Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Official Title
A Phase II, Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 30, 2020 (Actual)
Primary Completion Date
January 31, 2027 (Anticipated)
Study Completion Date
January 31, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Daiichi Sankyo Co., Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed DS5141-A-J101.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Duchenne muscular dystrophy, DMD, Oligonucleotides, antisense, Exon skipping, DS-5141b

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
8 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
DS-5141b 2.0 mg/kg
Arm Type
Experimental
Arm Description
Participants who will receive DS-5141b 2.0 mg/kg once weekly.
Arm Title
DS-5141b 6.0 mg/kg
Arm Type
Experimental
Arm Description
Participants who will receive DS-5141b 6.0 mg/kg once weekly.
Intervention Type
Drug
Intervention Name(s)
DS-5141b
Other Intervention Name(s)
Renadirsen
Intervention Description
Administered via subcutaneous injection once weekly
Primary Outcome Measure Information:
Title
Incidence of Adverse Events (AEs)
Time Frame
From first injection to after the last injection of DS-5141b in this study (within approximately 2 years)
Title
Change in distance walked during 6-minute walk test (6MWT)
Time Frame
Every 3 months (within approximately 2 years)
Title
Change in time to stand (TTSTAND)
Time Frame
Every 3 months (within approximately 2 years)
Title
Change in time in Timed up and go test
Time Frame
Every 3 months (within approximately 2 years)
Title
Change in time in 10-meter Run/Walk test
Time Frame
Every 3 months (within approximately 2 years)
Title
Change in score in the North Star Ambulatory Assessment (NSAA)
Time Frame
Every 3 months (within approximately 2 years)
Title
Change in score in the Performance of Upper Limb (PUL)
Time Frame
Every 3 months (within approximately 2 years)
Title
Change in Left Ventricular Ejection Fraction percentage (LVEF %)
Time Frame
Every 6 months (within approximately 2 years)
Title
Change in in Forced Vital Capacity (FVC) (percent predicted)
Time Frame
Every 6 months (within approximately 2 years)
Title
Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment
Time Frame
Every 3 months (within approximately 2 years)
Secondary Outcome Measure Information:
Title
Concentration of DS-5141a in plasma
Time Frame
Every 3 months (within approximately 2 years)

10. Eligibility

Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Has competed a study of DS5141-A-J101 Exclusion Criteria: Significant safety issues in a study of DS5141-A-J101 Patient who does not consent to use appropriate contraception Patient not appropriate to participant in the study as determined by the Investigator
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Study Leader
Organizational Affiliation
Daiichi Sankyo Co., Ltd.
Official's Role
Study Director
Facility Information:
Facility Name
National Center of Neurology and Psychiatry
City
Tokyo
State/Province
Kodaira-Shi
ZIP/Postal Code
187-8551
Country
Japan
Facility Name
Kobe University Hospital
City
Hyōgo, Kobe-shi
ZIP/Postal Code
650-0017
Country
Japan

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

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