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A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
study id #: NCT02752048
condition: 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.
intervention: TAS-205, Placebo
mechanism of action: HPGDS inhibitor to modulate effects of inflammation
last updated: May 03, 2020
start date: May 2016
estimated completion: October 17, 2017
phase of development: Phase 2
size / enrollment: 36
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.
- 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.
- 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.
- 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.
- 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.
• Eligible Sexes: male
• 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.
• 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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