A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy

Phase 2CompletedNCT02752048

Taiho Pharmaceutical Co., Ltd.36 enrolled

Overview

The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner.

Duchenne Muscular Dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in 3,500 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 then loss of upper body function. The main objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with DMD in an exploratory manner. The objective of this study is also to evaluate the safety, the dose-response and the urinary excretion of pharmacodynamic (PD) marker after 24-week repeated oral doses of TAS-205 in DMD patients.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

TAS-205DRUG

2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal

PlaceboDRUG

1 group: Placebo group. Oral administration for 24 weeks, BID after meal

Eligibility

Sex: MALEMin age: 5 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Able to give an informed consent. If applicable, able to give an informed assent. * Phenotypic evidence of DMD. * Male and ≧5 years of age. * Bodyweight ≧7.5 kg and \<60 kg. * Able to complete the 6MWD test with a distance of at least 75 m. * Able to take tablets. * If taking oral glucocorticoids no significant change in the total daily or dosing 6 months before enrollment. Exclusion Criteria: * Any serious drug allergy. * A forced vital capacity (FVC) of \<50% of predicted value. * Wearing a respirator continuously (except for the use during sleep). * A left ventricular ejection fraction (EF) of \<40% or fractional shortening (FS) of \<25% on echocardiogram. * Clinically significant cardiac failure and respiratory failure. * Ongoing immunosuppressive therapy (other than corticosteroids) . * Surgical history or plan for surgery that may affect muscular strength or motor function. * Any injury that may affect muscular strength or motor function. * With any systemic allergic disease or any chronic inflammatory disease. * Previous gene therapy (exon skipping, or stop codon read through therapy), cell-based therapy, or any other investigational agents.

Locations (11)

Nagoya City University Hospital

Aichi, Japan

National Hospital Organization Nagara Medical Center

Gifu, Japan

Kobe University Hospital

Hyōgo, Japan

Outcomes

Primary Outcomes

Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)

The distance the subject can walk as fast as possible in 6 minutes will be evaluated.

Time frame: baseline, 24 weeks

Secondary Outcomes

Mean Change From Baseline in Time to Rise From the Floor

The time required for the subject to rise from a supine position on the floor as quickly as possible will be evaluated.

Time frame: baseline, and 24 weeks

Mean Change From Baseline in Time to Walk/Run for 10meters

The time required for the subject to run or walk as quickly as possible a 10 m-wide passage with marks affixed on the floor will be evaluated.

Time frame: baseline, and 24 weeks

Mean Change From Baseline in Time to up and go (TUG)

This test will assess the extent of the subject's composite mobility, including standing up, walking, repositioning the body, and balancing.

Time frame: baseline, and 24 weeks

Data from ClinicalTrials.gov

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