A Study of TAS-205 for Duchenne Muscular Dystrophy

study id #: NCT02246478

condition: Duchenne Muscular Dystrophy

status: completed

The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.

intervention: TAS-205, Placebo

mechanism of action: HPGDS inhibitor to modulate effects of inflammation

results: https://clinicaltrials.gov/ct2/show/results/NCT02246478

last updated: November 22, 2018

start date: September 2014

estimated completion: September 2015

phase of development: Phase 1

size / enrollment: 21

study description:
Duchenne Muscular Dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3500 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 loss of upper body function. The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 after single and multiple doses in DMD patients. It is also evaluated if TAS-205 affects the urinary excretion of pharmacodynamic (PD) marker in DMD patients.

primary outcomes:

• Incidence of adverse events and side effects as safety [ Time Frame: From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days) ]

secondary outcomes:

• Peak plasma concentration (Cmax) of TAS-205 [ Time Frame: Administration period (ie. single-dose phase: from single administration day to 48 hours after the administration, multiple-dose phase: from the first administration day to 8 hours after the last administration) ]
  
• Area under the plasma concentration versus time curve (AUC) of TAS-205 [ Time Frame: Administration period (ie. single-dose phase: from single administration day to 48 hours after the administration, multiple-dose phase: from the first administration day to 8 hours after the last administration) ]
  
• The urinary excretion of PD marker [ Time Frame: From the day before the administration to the end of the observation period (ie. single dose phase: 9 days, multiple doses phase: 15 days) ]

inclusion criteria:
• Able to give an informed consent. If applicable, able to give an informed assent.
• Male and >= 5 years and < 16 years of age.
• Bodyweight of >= 15.0 kg and < 75.0 kg.
• Phenotypic evidence of DMD.
• Able to take tablets.
• If taking oral glucocorticosteroids no significant change in total daily dosage or dosing regimen after enrollment.
• Confirmed the urinary PD marker over its criteria.
• Able to follow the study protocol.

exclusion criteria:
• Current diagnosis or history of any drug allergy.
• A forced vital capacity (FVC) < 50% of predicted value.
• A left ventricular ejection fraction (EF) < 50% or fractional shortening (FS) < 25% based on echocardiogram (ECHO).
• Ongoing immunosuppressive therapy (other than corticosteroids).
• With severe disease such as hepatic disease, kidney disease and others.
• With any systemic allergic disease or any chronic inflammatory disease.
• Treated with any other investigational agents within 90 days.
• Positive reaction in hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test.

sponsor: Taiho Pharmaceutical Co., Ltd.

investigators: Taiho Pharmaceutical Co.,Ltd.

locations: Japan