A Study of TAS-205 for Duchenne Muscular Dystrophy

Phase 1

Completed

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

Detailed Description: 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.

Recruitment & Eligibility

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.

Arm && Interventions

Primary Outcome Measures

Name	Time	Method
Incidence of Adverse Events	From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days)	Source Vocabulary Name for Table Default: CTCAE (4.03)

Secondary Outcome Measures

Name	Time	Method
Peak Plasma Concentration (Cmax) of TAS-205	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.	Due to inspection missing, some data were not analyzed.
Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205	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)	Due to inspection missing, some data were not analyzed.
The Urinary Excretion of PD Marker	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.	Ratio of prostaglandin E2 metabolite / creatinine Due to inspection missing, some data were not analyzed.