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

NCT ID: NCT02667483

Last Updated: 2024-03-07

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-10-31
• Study Completion Date: 2020-10-20

Brief 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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

DS-5141b

DS-5141b, Subcutaneous injection

Part 1: DS-5141b will be injected subcutaneously once a week for 2 weeks at the following dose levels. Dose escalation will be performed. DS-5141b will be administered at dose levels 1 and 3 in Cohort 1 and at dose levels 2 and 4 in Cohort 2.

• Level 1: 0.1 mg/kg
• Level 2: 0.5 mg/kg
• Level 3: 2.0 mg/kg
• Level 4: 6.0 mg/kg

Part 2: Two doses of DS-5141b will be selected based on the results obtained in Part 1. Each selected dose will be administered subcutaneously once a week for 12 weeks.

Part 2-Extension-2: Two doses, 2.0 mg/kg or 6.0 mg/kg, of DS-5141b will be administered subcutaneously once a week for 48 weeks.

Group Type: EXPERIMENTAL

DS-5141b

Intervention Type: DRUG

DS-5141b, Subcutaneous injection

Interventions

DS-5141b

DS-5141b, Subcutaneous injection

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 10 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Daiichi Sankyo

INDUSTRY

Sponsor Role: collaborator

Daiichi Sankyo Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Global Clinical Leader

Role: STUDY_DIRECTOR

Daiichi Sankyo

Locations

Kobe University Hospital

Hyōgo, Kobe-shi, Japan

National Center of Neurology and Psychiatry

Tokyo, Kodaira-shi, Japan

Countries

Japan

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

153072

Identifier Type: REGISTRY

Identifier Source: secondary_id

DS5141-A-J101

Identifier Type: -

Identifier Source: org_study_id