NCT04433234

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
8

participants targeted

Target at below P25 for phase_2

Timeline
8mo left

Started Jun 2020

Longer than P75 for phase_2

Geographic Reach
1 country

2 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress89%
Jun 2020Jan 2027

First Submitted

Initial submission to the registry

June 12, 2020

Completed
4 days until next milestone

First Posted

Study publicly available on registry

June 16, 2020

Completed
14 days until next milestone

Study Start

First participant enrolled

June 30, 2020

Completed
6.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 31, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 31, 2027

Last Updated

September 19, 2025

Status Verified

September 1, 2025

Enrollment Period

6.6 years

First QC Date

June 12, 2020

Last Update Submit

September 18, 2025

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne muscular dystrophyDMDOligonucleotides, antisenseExon skippingDS-5141b

Outcome Measures

Primary Outcomes (10)

  • Incidence of Adverse Events (AEs)

    From first injection to after the last injection of DS-5141b in this study (within approximately 2 years)

  • Change in distance walked during 6-minute walk test (6MWT)

    Every 3 months (within approximately 2 years)

  • Change in time to stand (TTSTAND)

    Every 3 months (within approximately 2 years)

  • Change in time in Timed up and go test

    Every 3 months (within approximately 2 years)

  • Change in time in 10-meter Run/Walk test

    Every 3 months (within approximately 2 years)

  • Change in score in the North Star Ambulatory Assessment (NSAA)

    Every 3 months (within approximately 2 years)

  • Change in score in the Performance of Upper Limb (PUL)

    Every 3 months (within approximately 2 years)

  • Change in Left Ventricular Ejection Fraction percentage (LVEF %)

    Every 6 months (within approximately 2 years)

  • Change in in Forced Vital Capacity (FVC) (percent predicted)

    Every 6 months (within approximately 2 years)

  • Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment

    Every 3 months (within approximately 2 years)

Secondary Outcomes (1)

  • Concentration of DS-5141a in plasma

    Every 3 months (within approximately 2 years)

Study Arms (2)

DS-5141b 2.0 mg/kg

EXPERIMENTAL

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

Drug: DS-5141b

DS-5141b 6.0 mg/kg

EXPERIMENTAL

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

Drug: DS-5141b

Interventions

DS-5141bDRUG

Administered via subcutaneous injection once weekly

Also known as: Renadirsen
DS-5141b 2.0 mg/kgDS-5141b 6.0 mg/kg

Eligibility Criteria

Age5 Years+
Sexmale(Gender-based eligibility)
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Has competed a study of DS5141-A-J101

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

National Center of Neurology and Psychiatry

Tokyo, Kodaira-Shi, 187-8551, Japan

Location

Kobe University Hospital

Hyōgo, 650-0017, Japan

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Clinical Study Leader

    Daiichi Sankyo Co., Ltd.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 12, 2020

First Posted

June 16, 2020

Study Start

June 30, 2020

Primary Completion (Estimated)

January 31, 2027

Study Completion (Estimated)

January 31, 2027

Last Updated

September 19, 2025

Record last verified: 2025-09

Data Sharing

IPD Sharing
Will not share

Locations

JP(2)