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A Study of TAS-205 for Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02246478
Recruitment Status : Completed
First Posted : September 22, 2014
Results First Posted : June 4, 2021
Last Update Posted : June 4, 2021
Sponsor:
Information provided by (Responsible Party):
Taiho Pharmaceutical Co., Ltd.

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

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: TAS-205 Drug: Placebo Phase 1

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase I Study of Single and Multiple Doses of TAS-205 in Patients With Duchenne Muscular Dystrophy
Study Start Date : September 2014
Actual Primary Completion Date : June 2015
Actual Study Completion Date : September 2015

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Placebo Comparator: Placebo 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
Active Comparator: TAS-205 low dose 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
Active Comparator: TAS-205 middle dose 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
Active Comparator: TAS-205 high dose 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


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of Adverse Events [ 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) ]
    Source Vocabulary Name for Table Default: CTCAE (4.03)


Secondary Outcome Measures :
  1. Peak Plasma Concentration (Cmax) of TAS-205 [ Time Frame: Single-dose phase: immediately before dosing, 0, 0.5, 1, 2, 4, 8, 24, 48 hours post-dose, Multiple-dose phase: Days 1 and 7, immediately before morning dose, 0.5, 1, 2, 4, and 8 hours post-dose and Day 4, immediately before morning dose. ]
    Due to inspection missing, some data were not analyzed.

  2. 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) ]
    Due to inspection missing, some data were not analyzed.

  3. The Urinary Excretion of PD Marker [ Time Frame: Single-dose: Day -1 before administration, 0-24 hr post-dose, and 24-48 hr post-dose, Multiple-doses: Day -1 before administration, 0 hr after administration on Day 1 and 4 to the following day (Day 2 and 5), and 0-24 hr after administration on Day 7. ]
    Ratio of prostaglandin E2 metabolite / creatinine Due to inspection missing, some data were not analyzed.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 15 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02246478


Locations
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Japan
National Center of Neurology and Psychiatry
Tokyo, Japan, 187-8551
Sponsors and Collaborators
Taiho Pharmaceutical Co., Ltd.
Investigators
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Study Director: Taiho Pharmaceutical Co.,Ltd. Taiho Pharmaceutical Co., Ltd.
More Information
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Responsible Party: Taiho Pharmaceutical Co., Ltd.
ClinicalTrials.gov Identifier: NCT02246478    
Other Study ID Numbers: Taiho10053030
First Posted: September 22, 2014    Key Record Dates
Results First Posted: June 4, 2021
Last Update Posted: June 4, 2021
Last Verified: May 2021
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked