A Study of TAS-205 for Duchenne Muscular Dystrophy

Taiho Pharmaceutical Co., Ltd.23 enrolled

Overview

The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.

Duchenne Muscular Dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3500 lives male births. DMD patients suffer from a relentless decline in muscle strength that impairs the ability of walking and breathing, resulting in their lives with wheelchairs and loss of upper body function. The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 after single and multiple doses in DMD patients. It is also evaluated if TAS-205 affects the urinary excretion of pharmacodynamic (PD) marker in DMD patients.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

TAS-205DRUG

* Single-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step, single oral administration after meals * Multiple-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step (the same patients between single- and multiple-dose phases), repeated oral administration for 7 days, BID after meals

PlaceboDRUG

* Single-dose phase: 3steps (low dose, middle dose or high dose group), 2 patients/step, single oral administration after meals * Multiple-dose phase: 3 steps (low dose, middle dose or high dose group), 2 patients/step (the same patients between single- and multiple-dose phases), repeated oral administration for 7 days, BID after meals

Eligibility

Sex: MALEMin age: 5 YearsMax age: 15 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Able to give an informed consent. If applicable, able to give an informed assent. * Male and \>= 5 years and \< 16 years of age. * Bodyweight of \>= 15.0 kg and \< 75.0 kg. * Phenotypic evidence of DMD. * Able to take tablets. * If taking oral glucocorticosteroids no significant change in total daily dosage or dosing regimen after enrollment. * Confirmed the urinary PD marker over its criteria. * Able to follow the study protocol. Exclusion Criteria: * Current diagnosis or history of any drug allergy. * A forced vital capacity (FVC) \< 50% of predicted value. * A left ventricular ejection fraction (EF) \< 50% or fractional shortening (FS) \< 25% based on echocardiogram (ECHO). * Ongoing immunosuppressive therapy (other than corticosteroids). * With severe disease such as hepatic disease, kidney disease and others. * With any systemic allergic disease or any chronic inflammatory disease. * Treated with any other investigational agents within 90 days. * Positive reaction in hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test.

Locations (1)

National Center of Neurology and Psychiatry

Tokyo, Japan

Outcomes

Primary Outcomes

Incidence of Adverse Events

Source Vocabulary Name for Table Default: CTCAE (4.03)

Time frame: From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days)

Secondary Outcomes

Peak Plasma Concentration (Cmax) of TAS-205

Due to inspection missing, some data were not analyzed.

Time frame: Single-dose phase: immediately before dosing, 0, 0.5, 1, 2, 4, 8, 24, 48 hours post-dose, Multiple-dose phase: Days 1 and 7, immediately before morning dose, 0.5, 1, 2, 4, and 8 hours post-dose and Day 4, immediately before morning dose.

Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205

Due to inspection missing, some data were not analyzed.

Time frame: Administration period (ie. single-dose phase: from single administration day to 48 hours after the administration, multiple-dose phase: from the first administration day to 8 hours after the last administration)

The Urinary Excretion of PD Marker

Ratio of prostaglandin E2 metabolite / creatinine Due to inspection missing, some data were not analyzed.

Time frame: Single-dose: Day -1 before administration, 0-24 hr post-dose, and 24-48 hr post-dose, Multiple-doses: Day -1 before administration, 0 hr after administration on Day 1 and 4 to the following day (Day 2 and 5), and 0-24 hr after administration on Day 7.

Data from ClinicalTrials.gov

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