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

Primary Purpose

Duchenne Muscular Dystrophy

Status
Completed
Phase
Phase 2
Locations
Japan
Study Type
Interventional
Intervention
TAS-205
Placebo
Sponsored by
Taiho Pharmaceutical Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Nagoya City University Hospital
  • National Hospital Organization Nagara Medical Center
  • Kobe University Hospital
  • National Hospital Organization Utano Hospital
  • Shinshu University Hospital
  • National Hospital Organization Niigata National Hospital
  • National Hospital Organization Toneyama National Hospital
  • National Hospital Organization Higashisaitama Hospital
  • Tokyo Women's Medical University Hospital
  • National Center of Neurology and Psychiatry
  • Tottori University Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

TAS-205（Low dose group）

TAS-205（High dose group）

Placebo

Arm Description

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.

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.

Placebo group: Oral administration of tablets for 24 weeks, BID after meal

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.
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.
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.

Full Information

First Posted
April 6, 2016
Last Updated
April 8, 2020
Sponsor
Taiho Pharmaceutical Co., Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT02752048
Brief Title
A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
Official Title
A Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular Dystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
March 2020
Overall Recruitment Status
Completed
Study Start Date
May 2016 (undefined)
Primary Completion Date
May 15, 2017 (Actual)
Study Completion Date
October 17, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Taiho Pharmaceutical Co., Ltd.

4. Oversight

5. Study Description

Brief Summary
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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
36 (Actual)

8. Arms, Groups, and Interventions

Arm Title
TAS-205（Low dose group）
Arm Type
Experimental
Arm Description
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.
Arm Title
TAS-205（High dose group）
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo group: Oral administration of tablets for 24 weeks, BID after meal
Intervention Type
Drug
Intervention Name(s)
TAS-205
Intervention Description
2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Primary Outcome Measure Information:
Title
Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)
Description
The distance the subject can walk as fast as possible in 6 minutes will be evaluated.
Time Frame
baseline, 24 weeks
Secondary Outcome Measure Information:
Title
Mean Change From Baseline in Time to Rise From the Floor
Description
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
Title
Mean Change From Baseline in Time to Walk/Run for 10meters
Description
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
Title
Mean Change From Baseline in Time to up and go (TUG)
Description
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

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Taiho Pharmaceutical Co., Ltd.
Organizational Affiliation
Taiho Pharmaceutical Co., Ltd.
Official's Role
Study Director
Facility Information:
Facility Name
Nagoya City University Hospital
City
Aichi
ZIP/Postal Code
467-8601
Country
Japan
Facility Name
National Hospital Organization Nagara Medical Center
City
Gifu
ZIP/Postal Code
502-8558
Country
Japan
Facility Name
Kobe University Hospital
City
Hyogo
ZIP/Postal Code
650-0017
Country
Japan
Facility Name
National Hospital Organization Utano Hospital
City
Kyoto
ZIP/Postal Code
616-8255
Country
Japan
Facility Name
Shinshu University Hospital
City
Nagano
ZIP/Postal Code
390-8621
Country
Japan
Facility Name
National Hospital Organization Niigata National Hospital
City
Niigata
ZIP/Postal Code
945-8585
Country
Japan
Facility Name
National Hospital Organization Toneyama National Hospital
City
Osaka
ZIP/Postal Code
560-8552
Country
Japan
Facility Name
National Hospital Organization Higashisaitama Hospital
City
Saitama
ZIP/Postal Code
349-0196
Country
Japan
Facility Name
Tokyo Women's Medical University Hospital
City
Tokyo
ZIP/Postal Code
162-8666
Country
Japan
Facility Name
National Center of Neurology and Psychiatry
City
Tokyo
ZIP/Postal Code
187-8551
Country
Japan
Facility Name
Tottori University Hospital
City
Tottori
ZIP/Postal Code
683-8504
Country
Japan

12. IPD Sharing Statement

Citations:
PubMed Identifier
31957953
Citation
Komaki H, Maegaki Y, Matsumura T, Shiraishi K, Awano H, Nakamura A, Kinoshita S, Ogata K, Ishigaki K, Saitoh S, Funato M, Kuru S, Nakayama T, Iwata Y, Yajima H, Takeda S. Early phase 2 trial of TAS-205 in patients with Duchenne muscular dystrophy. Ann Clin Transl Neurol. 2020 Feb;7(2):181-190. doi: 10.1002/acn3.50978. Epub 2020 Jan 20.
Results Reference
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A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy

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