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

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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04433234

Enrollment

Registered

2020-06-16

Start date

2020-06-30

Completion date

2027-01-31

Last updated

2025-09-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne muscular dystrophy, DMD, Oligonucleotides, antisense, Exon skipping, DS-5141b

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.

Interventions

DRUGDS-5141b

Administered via subcutaneous injection once weekly

Study design

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

MALE

Age

5 Years to No maximum

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

Design outcomes

Primary

Measure	Time frame
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)
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)

Secondary

Measure	Time frame
Concentration of DS-5141a in plasma	Every 3 months (within approximately 2 years)

Countries

Japan

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026