A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
A Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular Dystrophy
1 other identifier
interventional
36
1 country
11
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started May 2016
Shorter than P25 for phase_2
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 6, 2016
CompletedFirst Posted
Study publicly available on registry
April 26, 2016
CompletedStudy Start
First participant enrolled
May 1, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 15, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
October 17, 2017
CompletedResults Posted
Study results publicly available
April 20, 2020
CompletedApril 20, 2020
March 1, 2020
1 year
April 6, 2016
March 4, 2020
April 8, 2020
Conditions
Outcome Measures
Primary Outcomes (1)
Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)
The distance the subject can walk as fast as possible in 6 minutes will be evaluated.
baseline, 24 weeks
Secondary Outcomes (3)
Mean Change From Baseline in Time to Rise From the Floor
baseline, and 24 weeks
Mean Change From Baseline in Time to Walk/Run for 10meters
baseline, and 24 weeks
Mean Change From Baseline in Time to up and go (TUG)
baseline, and 24 weeks
Study Arms (3)
TAS-205（Low dose group）
EXPERIMENTALLow 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.
TAS-205（High dose group）
EXPERIMENTALHigh 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.
Placebo
PLACEBO COMPARATORPlacebo group: Oral administration of tablets for 24 weeks, BID after meal
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (11)
Nagoya City University Hospital
Aichi, 467-8601, Japan
National Hospital Organization Nagara Medical Center
Gifu, 502-8558, Japan
Kobe University Hospital
Hyōgo, 650-0017, Japan
National Hospital Organization Utano Hospital
Kyoto, 616-8255, Japan
Shinshu University Hospital
Nagano, 390-8621, Japan
National Hospital Organization Niigata National Hospital
Niigata, 945-8585, Japan
National Hospital Organization Toneyama National Hospital
Osaka, 560-8552, Japan
National Hospital Organization Higashisaitama Hospital
Saitama, 349-0196, Japan
Tokyo Women's Medical University Hospital
Tokyo, 162-8666, Japan
National Center of Neurology and Psychiatry
Tokyo, 187-8551, Japan
Tottori University Hospital
Tottori, 683-8504, Japan
Related Publications (1)
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.
PMID: 31957953DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Taiho Pharmaceutical Co., Ltd.
- Organization
- Clinical Trial Registration Contact
Study Officials
- STUDY DIRECTOR
Taiho Pharmaceutical Co., Ltd.
Taiho Pharmaceutical Co., Ltd.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 6, 2016
First Posted
April 26, 2016
Study Start
May 1, 2016
Primary Completion
May 15, 2017
Study Completion
October 17, 2017
Last Updated
April 20, 2020
Results First Posted
April 20, 2020
Record last verified: 2020-03