Skip to main content
OpenTrials
Completed

NCT Number: NCT02246478

A Study of TAS-205 for Duchenne Muscular Dystrophy

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

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

5 year–15 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1

Primary location

National Center of Neurology and Psychiatry

Tokyo, 187-8551, Japan

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

TAS-205

Drug
  • 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

Placebo

Drug
  • 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

Primary outcomes

  1. Incidence of Adverse Events

    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)

    Source Vocabulary Name for Table Default: CTCAE (4.03)

Secondary outcomes

  1. Peak Plasma Concentration (Cmax) of TAS-205

    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.

    Due to inspection missing, some data were not analyzed.

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

    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)

    Due to inspection missing, some data were not analyzed.

  3. The Urinary Excretion of PD Marker

    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.

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

Sponsors and collaborators

Lead sponsor

Taiho Pharmaceutical Co., Ltd.

Industry

Registry information

Official study title

A Phase I Study of Single and Multiple Doses of TAS-205 in Patients With Duchenne Muscular Dystrophy

Important dates

Study start
2014
Primary completion
2015
Study completion
2015
First posted
Sep 22, 2014
Registry last updated
Jun 4, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.