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

Daiichi Sankyo Co., Ltd.8 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

DS-5141bDRUG

DS-5141b, Subcutaneous injection

Eligibility

Sex: MALEMin age: 5 YearsMax age: 10 Years

Medical Language ↔ Plain English

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.

Locations (2)

Kobe University Hospital

Hyōgo, Kobe-shi, Japan

National Center of Neurology and Psychiatry

Tokyo, Kodaira-shi, Japan

Outcomes

Primary Outcomes

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.

Time frame: 48 Weeks of Part 2-Extension-2

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.