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

Phase 2
Completed
Conditions
Duchenne Muscular Dystrophy
Interventions
Drug: Placebo
Drug: TAS-205
Registration Number
NCT02752048
Lead Sponsor
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.

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.

Recruitment & Eligibility

Status
COMPLETED
Sex
Male
Target Recruitment
36
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.
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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.
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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboPlacebo group: Oral administration of tablets for 24 weeks, BID after meal
TAS-205(Low dose group)TAS-205Low 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.
TAS-205(High dose group)TAS-205High 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.
Primary Outcome Measures
NameTimeMethod
Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)baseline, 24 weeks

The distance the subject can walk as fast as possible in 6 minutes will be evaluated.

Secondary Outcome Measures
NameTimeMethod
Mean Change From Baseline in Time to Rise From the Floorbaseline, 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.

Mean Change From Baseline in Time to Walk/Run for 10metersbaseline, 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.

Mean Change From Baseline in Time to up and go (TUG)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.

Trial Locations

Locations (11)

Nagoya City University Hospital

🇯🇵

Aichi, Japan

National Hospital Organization Nagara Medical Center

🇯🇵

Gifu, Japan

National Hospital Organization Utano Hospital

🇯🇵

Kyoto, Japan

Kobe University Hospital

🇯🇵

Hyogo, Japan

Shinshu University Hospital

🇯🇵

Nagano, Japan

National Center of Neurology and Psychiatry

🇯🇵

Tokyo, Japan

Tottori University Hospital

🇯🇵

Tottori, Japan

National Hospital Organization Higashisaitama Hospital

🇯🇵

Saitama, Japan

National Hospital Organization Niigata National Hospital

🇯🇵

Niigata, Japan

National Hospital Organization Toneyama National Hospital

🇯🇵

Osaka, Japan

Tokyo Women's Medical University Hospital

🇯🇵

Tokyo, Japan

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