Back to resultsA Study of TAS-205 for Duchenne Muscular Dystrophy
Primary Purpose
Duchenne Muscular Dystrophy
Status
Completed
Phase
Phase 1
Locations
Japan
Study Type
Interventional
Intervention
TAS-205
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy
Eligibility 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.
Sites / Locations
- National Center of Neurology and Psychiatry
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Placebo Comparator
Active Comparator
Active Comparator
Active Comparator
Arm Label
Placebo
TAS-205 low dose
TAS-205 middle dose
TAS-205 high dose
Arm Description
Outcomes
Primary Outcome Measures
Incidence of Adverse Events
Source Vocabulary Name for Table Default: CTCAE (4.03)
Secondary Outcome Measures
Peak Plasma Concentration (Cmax) of TAS-205
Due to inspection missing, some data were not analyzed.
Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205
Due to inspection missing, some data were not analyzed.
The Urinary Excretion of PD Marker
Ratio of prostaglandin E2 metabolite / creatinine Due to inspection missing, some data were not analyzed.
Full Information
NCT ID
NCT02246478
First Posted
September 9, 2014
Last Updated
May 11, 2021
Sponsor
Taiho Pharmaceutical Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT02246478
Brief Title
A Study of TAS-205 for Duchenne Muscular Dystrophy
Official Title
A Phase I Study of Single and Multiple Doses of TAS-205 in Patients With Duchenne Muscular Dystrophy
Study Type
Interventional
2. Study Status
Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
September 2014 (undefined)
Primary Completion Date
June 2015 (Actual)
Study Completion Date
September 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Taiho Pharmaceutical Co., Ltd.
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
23 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Title
TAS-205 low dose
Arm Type
Active Comparator
Arm Title
TAS-205 middle dose
Arm Type
Active Comparator
Arm Title
TAS-205 high dose
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
TAS-205
Intervention Description
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
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
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
Primary Outcome Measure Information:
Title
Incidence of Adverse Events
Description
Source Vocabulary Name for Table Default: CTCAE (4.03)
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 Outcome Measure Information:
Title
Peak Plasma Concentration (Cmax) of TAS-205
Description
Due to inspection missing, some data were not analyzed.
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.
Title
Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205
Description
Due to inspection missing, some data were not analyzed.
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)
Title
The Urinary Excretion of PD Marker
Description
Ratio of prostaglandin E2 metabolite / creatinine Due to inspection missing, some data were not analyzed.
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.
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
15 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Taiho Pharmaceutical Co.,Ltd.
Organizational Affiliation
Taiho Pharmaceutical Co., Ltd.
Official's Role
Study Director
Facility Information:
Facility Name
National Center of Neurology and Psychiatry
City
Tokyo
ZIP/Postal Code
187-8551
Country
Japan
12. IPD Sharing Statement
Learn more about this trial
A Study of TAS-205 for Duchenne Muscular Dystrophy
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