Back to resultsLong-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
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
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
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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