Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Clinical Trial

Official title:

Phase I/II Study of DS-5141b: Open-label Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02667483
• Other study ID #: DS5141-A-J101
• Secondary ID: 153072
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: October 2015
• Est. completion date: October 20, 2020

Study information

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

Clinical Trial Summary

This is a phase I/II study to evaluate the safety, tolerability, efficacy, and pharmacokinetic (PK) profile of DS-5141b in patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping and to determine the dosage for subsequent studies.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: October 20, 2020
• Est. primary completion date: October 20, 2020
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years to 10 Years

Eligibility

Inclusion Criteria:

• Confirmation of out-of-frame deletion(s) that could be corrected by dystrophin gene exon 45 skipping.

• Intact muscles of adequate quality for biopsy to allow evaluation of the efficacy of the study drug.

• Boys aged from 5 years to <11 years.

• Patients able to walk at least 325 meters in the 6-minutes walk test.

• Glucocorticoid-naive patients, or patients who have used glucocorticoids for at least 6 months prior to enrollment in this study with no dose changes for at least 3 months prior to enrollment.

Exclusion Criteria:

• A genetic mutation that can not be expected the expression of dystrophin protein by dystrophin gene exon 45 skipping.

• A concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function.

• Current or history of severe disorder.

• Left ventricular ejection fraction (LEVF) <55%.

• Corrected QT interval (QTc) >0.45 sec.

Related Conditions & MeSH terms

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

Intervention

Drug:
• DS-5141b
DS-5141b, Subcutaneous injection

Locations

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

Sponsors (2)

Lead Sponsor	Collaborator
Daiichi Sankyo Co., Ltd.	Orphan Disease Treatment Institute Co., Ltd.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants Reporting at Least One Treatment-emergent Adverse Event (TEAE) In Participants With Duchenne Muscular Dystrophy	A treatment-emergent adverse event (TEAE) is defined as an adverse event that emerges during treatment having been absent prior to treatment or reemerges during treatment or worsens in severity during treatment.	48 Weeks of Part 2-Extension-2
Primary	Pharmacokinetic Parameter Maximum Concentration (Cmax) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy	Pharmacokinetic parameters were assessed using non-compartmental methods.	Week 48 of Part 2-Extension-2
Primary	Pharmacokinetic Parameter Area Under the Curve (AUC) Tau of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy	Pharmacokinetic parameters were assessed using non-compartmental methods.	Week 48 of Part 2-Extension-2
Primary	Pharmacokinetic Parameter Time to Maximum Concentration (Tmax) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy	Pharmacokinetic parameters were assessed using non-compartmental methods.	Week 48 of Part 2-Extension-2
Primary	Pharmacokinetic Parameter Half-life (T1/2) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Dystrophy	Pharmacokinetic parameters were assessed using non-compartmental methods.	Week 48 of Part 2-Extension-2
Primary	Mean Dystrophin Protein Expression in Muscle Tissue		Week 48 of Part 2-Extension-2
Secondary	Number of Participants With Exon 45-skipped Dystrophin mRNA Expression in Muscle Tissue Posttreatment		Week 48 of Part 2-Extension-2