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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04433234
• Other study ID #: DS5141-A-J201
• Secondary ID: 205321
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: June 30, 2020
• Est. completion date: January 31, 2027

Study information

• Verified date: July 2023
• Source: Daiichi Sankyo, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 8
• Est. completion date: January 31, 2027
• Est. primary completion date: January 31, 2027
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• DS-5141b
Administered via subcutaneous injection once weekly

Locations

Country	Name	City	State
Japan	Kobe University Hospital	Hyogo, Kobe-shi
Japan	National Center of Neurology and Psychiatry	Tokyo	Kodaira-Shi

Sponsors (1)

Lead Sponsor	Collaborator
Daiichi Sankyo Co., Ltd.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Adverse Events (AEs)		From first injection to after the last injection of DS-5141b in this study (within approximately 2 years)
Primary	Change in distance walked during 6-minute walk test (6MWT)		Every 3 months (within approximately 2 years)
Primary	Change in time to stand (TTSTAND)		Every 3 months (within approximately 2 years)
Primary	Change in time in Timed up and go test		Every 3 months (within approximately 2 years)
Primary	Change in time in 10-meter Run/Walk test		Every 3 months (within approximately 2 years)
Primary	Change in score in the North Star Ambulatory Assessment (NSAA)		Every 3 months (within approximately 2 years)
Primary	Change in score in the Performance of Upper Limb (PUL)		Every 3 months (within approximately 2 years)
Primary	Change in Left Ventricular Ejection Fraction percentage (LVEF %)		Every 6 months (within approximately 2 years)
Primary	Change in in Forced Vital Capacity (FVC) (percent predicted)		Every 6 months (within approximately 2 years)
Primary	Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment		Every 3 months (within approximately 2 years)
Secondary	Concentration of DS-5141a in plasma		Every 3 months (within approximately 2 years)