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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02752048
Recruitment Status : Completed
First Posted : April 26, 2016
Results First Posted : April 20, 2020
Last Update Posted : April 20, 2020
Sponsor:
Information provided by (Responsible Party):
Taiho Pharmaceutical Co., Ltd.

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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: TAS-205 Drug: Placebo Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular Dystrophy
Study Start Date : May 2016
Actual Primary Completion Date : May 15, 2017
Actual Study Completion Date : October 17, 2017


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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.



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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02752048


Locations
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Japan
Nagoya City University Hospital
Aichi, Japan, 467-8601
National Hospital Organization Nagara Medical Center
Gifu, Japan, 502-8558
Kobe University Hospital
Hyogo, Japan, 650-0017
National Hospital Organization Utano Hospital
Kyoto, Japan, 616-8255
Shinshu University Hospital
Nagano, Japan, 390-8621
National Hospital Organization Niigata National Hospital
Niigata, Japan, 945-8585
National Hospital Organization Toneyama National Hospital
Osaka, Japan, 560-8552
National Hospital Organization Higashisaitama Hospital
Saitama, Japan, 349-0196
Tokyo Women's Medical University Hospital
Tokyo, Japan, 162-8666
National Center of Neurology and Psychiatry
Tokyo, Japan, 187-8551
Tottori University Hospital
Tottori, Japan, 683-8504
Sponsors and Collaborators
Taiho Pharmaceutical Co., Ltd.
Investigators
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Study Director: Taiho Pharmaceutical Co., Ltd. Taiho Pharmaceutical Co., Ltd.
  Study Documents (Full-Text)

Documents provided by Taiho Pharmaceutical Co., Ltd.:
Study Protocol  [PDF] April 7, 2016
Statistical Analysis Plan  [PDF] August 14, 2018


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Taiho Pharmaceutical Co., Ltd.
ClinicalTrials.gov Identifier: NCT02752048    
Other Study ID Numbers: Taiho10053040
First Posted: April 26, 2016    Key Record Dates
Results First Posted: April 20, 2020
Last Update Posted: April 20, 2020
Last Verified: March 2020
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked