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A Study of TAS-205 for Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02246478
Recruitment Status : Completed
First Posted : September 22, 2014
Last Update Posted : September 25, 2015
Sponsor:
Information provided by (Responsible Party):
Taiho Pharmaceutical Co., Ltd.

Tracking Information
First Submitted Date  ICMJE September 9, 2014
First Posted Date  ICMJE September 22, 2014
Last Update Posted Date September 25, 2015
Study Start Date  ICMJE September 2014
Actual Primary Completion Date June 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 18, 2014)
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) ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02246478 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 18, 2014)
  • 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) ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of TAS-205 for Duchenne Muscular Dystrophy
Official Title  ICMJE A Phase I Study of Single and Multiple Doses of TAS-205 in Patients With Duchenne Muscular Dystrophy
Brief Summary The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.
Detailed 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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Drug: TAS-205
    • Single-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step, single oral administration after meals
    • Multiple-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step (the same patients between single- and multiple-dose phases), repeated oral administration for 7 days, BID after meals
  • Drug: Placebo
    • Single-dose phase: 3steps (low dose, middle dose or high dose group), 2 patients/step, single oral administration after meals
    • Multiple-dose phase: 3 steps (low dose, middle dose or high dose group), 2 patients/step (the same patients between single- and multiple-dose phases), repeated oral administration for 7 days, BID after meals
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    Intervention: Drug: Placebo
  • Active Comparator: TAS-205 low dose
    Intervention: Drug: TAS-205
  • Active Comparator: TAS-205 middle dose
    Intervention: Drug: TAS-205
  • Active Comparator: TAS-205 high dose
    Intervention: Drug: TAS-205
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 18, 2014)
21
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE September 2015
Actual Primary Completion Date June 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 5 Years to 15 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Japan
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02246478
Other Study ID Numbers  ICMJE Taiho10053030
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Taiho Pharmaceutical Co., Ltd.
Study Sponsor  ICMJE Taiho Pharmaceutical Co., Ltd.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Taiho Pharmaceutical Co.,Ltd. Taiho Pharmaceutical Co., Ltd.
PRS Account Taiho Pharmaceutical Co., Ltd.
Verification Date September 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP