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A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02752048
Recruitment Status : Completed
First Posted : April 26, 2016
Results First Posted : April 20, 2020
Last Update Posted : April 20, 2020
Sponsor:
Information provided by (Responsible Party):
Taiho Pharmaceutical Co., Ltd.

Tracking Information
First Submitted Date  ICMJE April 6, 2016
First Posted Date  ICMJE April 26, 2016
Results First Submitted Date  ICMJE March 4, 2020
Results First Posted Date  ICMJE April 20, 2020
Last Update Posted Date April 20, 2020
Study Start Date  ICMJE May 2016
Actual Primary Completion Date May 15, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 8, 2020)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: April 21, 2016)
Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD) [ Time Frame: baseline, 24 weeks ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 8, 2020)
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: April 21, 2016)
  • 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, 1, 2, 4, 8, 12, 18, 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
Official Title  ICMJE A Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular Dystrophy
Brief Summary 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.
Detailed Description 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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
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
    2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
  • Drug: Placebo
    1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Study Arms  ICMJE
  • Experimental: TAS-205(Low dose group)
    Low dose group:Oral administration of tablets for 24 weeks, bis in die (BID) after meal The number of tablets of the study drug corresponding to the dosage (6.67-13.33 mg/kg/dose) by body weight within 14 days before enrollment was to be administered within 30 minutes after breakfast and dinner.
    Intervention: Drug: TAS-205
  • Experimental: TAS-205(High dose group)
    High dose group: Oral administration of tablets for 24 weeks, bis in die (BID) after meal The number of tablets of the study drug corresponding to the dosage (13.33-26.67 mg/kg/dose) by body weight within 14 days before enrollment was to be administered within 30 minutes after breakfast and dinner.
    Intervention: Drug: TAS-205
  • Placebo Comparator: Placebo
    Placebo group: Oral administration of tablets for 24 weeks, BID after meal
    Intervention: Drug: Placebo
Publications * Komaki H, Maegaki Y, Matsumura T, Shiraishi K, Awano H, Nakamura A, Kinoshita S, Ogata K, Ishigaki K, Saitoh S, Funato M, Kuru S, Nakayama T, Iwata Y, Yajima H, Takeda S. Early phase 2 trial of TAS-205 in patients with Duchenne muscular dystrophy. Ann Clin Transl Neurol. 2020 Feb;7(2):181-190. doi: 10.1002/acn3.50978. Epub 2020 Jan 20.

*   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: January 30, 2019)
36
Original Estimated Enrollment  ICMJE
 (submitted: April 21, 2016)
33
Actual Study Completion Date  ICMJE October 17, 2017
Actual Primary Completion Date May 15, 2017   (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.
  • 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.

Exclusion Criteria:

  • 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 5 Years and older   (Child, Adult, Older Adult)
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 NCT02752048
Other Study ID Numbers  ICMJE Taiho10053040
Has Data Monitoring Committee Not Provided
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 March 2020

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