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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: February 14, 2019
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 ]
- Mean change from baseline to 12 weeks in the 6MWD [ Time Frame: baseline, 12 weeks ]
- Mean change from baseline in time to rise from the floor [ Time Frame: baseline, 12 and 24 weeks ]
- Mean change from baseline in time to walk/run for 10meters [ Time Frame: baseline, 12 and 24 weeks ]
- Mean change from baseline in time to up and go (TUG) [ Time Frame: baseline, 12 and 24 weeks ]
- Mean change from baseline to 24 weeks in skeletal muscle mass using muscle CT [ Time Frame: baseline, 24 weeks ]
- Mean change from baseline in lean body mass on bioelectrical impedance analysis (BIA) [ Time Frame: baseline, 1, 2, 4, 8, 12, 18, 24 weeks ]
- Mean change from baseline in quantitative muscle strength using hand-held dynamometer (ex.microFET2) [ Time Frame: baseline, 12, 24 weeks ]
- Mean change from baseline in serum Creatine kinase concentration [ Time Frame: baseline, 1, 12, 24 weeks ]
- Mean change from baseline in pulmonary function as measured by spirometry [ Time Frame: baseline, 1, 4, 12, 24 weeks ]
- Incidence of adverse events and side effects [ Time Frame: up to 27 weeks ]
- The urinary excretion amount of PD marker (PGD2 metabolite) in 24 hr-pooled urine [ Time Frame: baseline, 0.5, 12, 24 weeks ]
• 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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