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

April 8, 2020 updated by: Taiho Pharmaceutical Co., Ltd.

A Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular Dystrophy

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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

Enrollment (Actual)

36

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Japan
      • Aichi, Japan, 467-8601
        • Nagoya City University Hospital
      • Gifu, Japan, 502-8558
        • National Hospital Organization Nagara Medical Center
      • Hyogo, Japan, 650-0017
        • Kobe University Hospital
      • Kyoto, Japan, 616-8255
        • National Hospital Organization Utano Hospital
      • Nagano, Japan, 390-8621
        • Shinshu University Hospital
      • Niigata, Japan, 945-8585
        • National Hospital Organization Niigata National Hospital
      • Osaka, Japan, 560-8552
        • National Hospital Organization Toneyama National Hospital
      • Saitama, Japan, 349-0196
        • National Hospital Organization Higashisaitama Hospital
      • Tokyo, Japan, 162-8666
        • Tokyo Women's Medical University Hospital
      • Tokyo, Japan, 187-8551
        • National Center of Neurology and Psychiatry
      • Tottori, Japan, 683-8504
        • Tottori University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: TAS-205（Low dose group）
Low dose group：Oral administration of tablets for 24 weeks, bis in die (BID) after meal The number of tablets of the study drug corresponding to the dosage (6.67-13.33 mg/kg/dose) by body weight within 14 days before enrollment was to be administered within 30 minutes after breakfast and dinner.
Drug: TAS-205
2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
Experimental: TAS-205（High dose group）
High dose group: Oral administration of tablets for 24 weeks, bis in die (BID) after meal The number of tablets of the study drug corresponding to the dosage (13.33-26.67 mg/kg/dose) by body weight within 14 days before enrollment was to be administered within 30 minutes after breakfast and dinner.
Drug: TAS-205
2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
Placebo Comparator: Placebo
Placebo group: Oral administration of tablets for 24 weeks, BID after meal
Drug: Placebo
1 group: Placebo group. Oral administration for 24 weeks, BID after meal

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)
Time Frame: baseline, 24 weeks
The distance the subject can walk as fast as possible in 6 minutes will be evaluated.
baseline, 24 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Mean Change From Baseline in Time to Rise From the Floor
Time Frame: baseline, and 24 weeks
The time required for the subject to rise from a supine position on the floor as quickly as possible will be evaluated.
baseline, and 24 weeks
Mean Change From Baseline in Time to Walk/Run for 10meters
Time Frame: baseline, and 24 weeks
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.
baseline, and 24 weeks
Mean Change From Baseline in Time to up and go (TUG)
Time Frame: baseline, and 24 weeks
This test will assess the extent of the subject's composite mobility, including standing up, walking, repositioning the body, and balancing.
baseline, and 24 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Taiho Pharmaceutical Co., Ltd.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2016

Primary Completion (Actual)

May 15, 2017

Study Completion (Actual)

October 17, 2017

Study Registration Dates

First Submitted

April 6, 2016

First Submitted That Met QC Criteria

April 21, 2016

First Posted (Estimate)

April 26, 2016

Study Record Updates

Last Update Posted (Actual)

April 20, 2020

Last Update Submitted That Met QC Criteria

April 8, 2020

Last Verified

March 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Taiho10053040

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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