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A Phase IIa 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: 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.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Duchenne Muscular Dystrophy
Interventions Drug: TAS-205
Drug: Placebo
Enrollment 36
Recruitment Details  
Pre-assignment Details  
Arm/Group Title TAS-205(Low Dose Group) TAS-205(High Dose Group) Placebo
Hide Arm/Group Description TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Period Title: Overall Study
Started 11 12 13
Completed 11 12 10
Not Completed 0 0 3
Reason Not Completed
Withdrawal by Subject             0             0             3
Arm/Group Title TAS-205(Low Dose Group) TAS-205(High Dose Group) Placebo Total
Hide Arm/Group Description TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal Total of all reporting groups
Overall Number of Baseline Participants 11 11 10 32
Hide Baseline Analysis Population Description
Analysis population was per protocol set. One patients excluded from TAS-205 High dose group, and three excluded from placebo group.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 11 participants 10 participants 32 participants
<=18 years
11
 100.0%
11
 100.0%
10
 100.0%
32
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 11 participants 10 participants 32 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
11
 100.0%
11
 100.0%
10
 100.0%
32
 100.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 11 participants 10 participants 32 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
11
 100.0%
11
 100.0%
10
 100.0%
32
 100.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Japan Number Analyzed 11 participants 11 participants 10 participants 32 participants
11 11 10 32
1.Primary Outcome
Title Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)
Hide Description The distance the subject can walk as fast as possible in 6 minutes will be evaluated.
Time Frame baseline, 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title TAS-205(Low Dose Group) TAS-205(High Dose Group) Placebo
Hide Arm/Group Description:
TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Overall Number of Participants Analyzed 11 11 10
Mean (Standard Deviation)
Unit of Measure: meter
-3.5  (67.3) -7.5  (37.3) -17.0  (55.6)
2.Secondary Outcome
Title Mean Change From Baseline in Time to Rise From the Floor
Hide Description The time required for the subject to rise from a supine position on the floor as quickly as possible will be evaluated.
Time Frame baseline, and 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title TAS-205(Low Dose Group) TAS-205(High Dose Group) Placebo
Hide Arm/Group Description:
TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Overall Number of Participants Analyzed 11 11 10
Mean (Standard Deviation)
Unit of Measure: second
4.011  (6.209) 1.283  (2.547) 2.633  (4.246)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection TAS-205(Low Dose Group), Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments Change in time to rise from the floor from baseline to Week 24 were calculated to evaluate
Statistical Test of Hypothesis P-Value 0.596
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -1.378
Confidence Interval (2-Sided) 95%
-6.757 to 4.000
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection TAS-205(High Dose Group), Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments Change in time to rise from the floor from baseline to Week 24 were calculated to evaluate
Statistical Test of Hypothesis P-Value 0.433
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.349
Confidence Interval (2-Sided) 95%
-2.220 to 4.918
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Mean Change From Baseline in Time to Walk/Run for 10meters
Hide Description 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.
Time Frame baseline, and 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title TAS-205(Low Dose Group) TAS-205(High Dose Group) Placebo
Hide Arm/Group Description:
TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Overall Number of Participants Analyzed 11 11 10
Mean (Standard Deviation)
Unit of Measure: second
1.113  (1.140) 1.025  (1.296) 0.629  (1.272)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection TAS-205(Low Dose Group), Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments Change in 10-m walk/run test from baseline to Week 24 were calculated to evaluate
Statistical Test of Hypothesis P-Value 0.382
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -0.484
Confidence Interval (2-Sided) 95%
-1.618 to 0.650
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection TAS-205(High Dose Group), Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments Change in 10-m walk/run test from baseline to Week 24 were calculated to evaluate
Statistical Test of Hypothesis P-Value 0.501
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -0.397
Confidence Interval (2-Sided) 95%
-1.610 to 0.817
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Mean Change From Baseline in Time to up and go (TUG)
Hide Description This test will assess the extent of the subject's composite mobility, including standing up, walking, repositioning the body, and balancing.
Time Frame baseline, and 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title TAS-205(Low Dose Group) TAS-205(High Dose Group) Placebo
Hide Arm/Group Description:
TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Overall Number of Participants Analyzed 11 11 10
Mean (Standard Deviation)
Unit of Measure: second
0.594  (2.156) 0.286  (1.626) 0.061  (1.626)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection TAS-205(Low Dose Group), Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments Change in time to up and go from baseline to Week 24 were calculated to evaluate
Statistical Test of Hypothesis P-Value 0.549
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -0.533
Confidence Interval (2-Sided) 95%
-2.363 to 1.298
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection TAS-205(High Dose Group), Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments Change in time to up and go from baseline to Week 24 were calculated to evaluate
Statistical Test of Hypothesis P-Value 0.767
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -0.225
Confidence Interval (2-Sided) 95%
-1.801 to 1.351
Estimation Comments [Not Specified]
Time Frame From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title TAS-205(Low Dose Group) TAS-205(High Dose Group) Placebo
Hide Arm/Group Description TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
All-Cause Mortality
TAS-205(Low Dose Group) TAS-205(High Dose Group) Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/11 (0.00%)      0/12 (0.00%)      0/12 (0.00%)    
Hide Serious Adverse Events
TAS-205(Low Dose Group) TAS-205(High Dose Group) Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/11 (0.00%)      0/12 (0.00%)      0/12 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
TAS-205(Low Dose Group) TAS-205(High Dose Group) Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   9/11 (81.82%)      9/12 (75.00%)      10/12 (83.33%)    
Blood and lymphatic system disorders       
Lymphadenopathy  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Cardiac disorders       
Constipation  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Dental caries  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/12 (8.33%)  1
Diarrhoea  1  1/11 (9.09%)  1 1/12 (8.33%)  1 0/12 (0.00%)  0
Stomatitis  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
General disorders       
Mucosal inflammation  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Pyrexia  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/12 (8.33%)  1
Peripheral swelling  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Infections and infestations       
Bronchitis  1  1/11 (9.09%)  1 1/12 (8.33%)  1 2/12 (16.67%)  2
Gastroenteritis  1  2/11 (18.18%)  2 3/12 (25.00%)  3 0/12 (0.00%)  0
Herpes zoster  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/12 (8.33%)  1
Impetigo  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Influenza  1  0/11 (0.00%)  0 0/12 (0.00%)  0 2/12 (16.67%)  2
Mumps  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Pneumonia mycoplasmal  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Rhinitis  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Sinusitis  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/12 (0.00%)  0
Tonsillitis  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/12 (0.00%)  0
Upper respiratory tract infection  1  1/11 (9.09%)  1 2/12 (16.67%)  2 1/12 (8.33%)  1
Viral upper respiratory tract infection  1  0/11 (0.00%)  0 1/12 (8.33%)  1 1/12 (8.33%)  1
Beta haemolytic streptococcal infection  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/12 (0.00%)  0
Pneumonia bacterial  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Injury, poisoning and procedural complications       
Chillblains  1  0/11 (0.00%)  0 0/12 (0.00%)  0 2/12 (16.67%)  2
Excoriation  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Investigations       
Blood corticotrophin decreased  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Cortisol decreased  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Glucose urine present  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Muscle enzyme increased  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Cystatin C increased  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/12 (0.00%)  0
Urobilinogen urine increased  1  0/11 (0.00%)  0 2/12 (16.67%)  2 0/12 (0.00%)  0
Metabolism and nutrition disorders       
Dehydration  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/12 (8.33%)  1
Musculoskeletal and connective tissue disorders       
Arthralgia  1  1/11 (9.09%)  1 1/12 (8.33%)  1 2/12 (16.67%)  2
Muscle spasms  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/12 (0.00%)  0
Myalgia  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Pain in extremity  1  2/11 (18.18%)  2 1/12 (8.33%)  1 2/12 (16.67%)  2
Nervous system disorders       
Headache  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/12 (0.00%)  0
Vomiting psychogenic  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/12 (8.33%)  1
Psychiatric disorders       
Head banging  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/12 (8.33%)  1
Respiratory, thoracic and mediastinal disorders       
Asthma  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Cough  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/12 (8.33%)  1
Epistaxis  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/12 (8.33%)  1
Rhinorrhoea  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Upper respiratory tract inflammation  1  2/11 (18.18%)  2 3/12 (25.00%)  3 0/12 (0.00%)  0
Skin and subcutaneous tissue disorders       
Dyshidrotic eczema  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/12 (0.00%)  0
Eczema asteatotic  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/12 (8.33%)  1
Erythema  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Leukoderma  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/12 (0.00%)  0
Palmar erythema  1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/12 (0.00%)  0
Papule  1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/12 (0.00%)  0
Rash  1  1/11 (9.09%)  1 1/12 (8.33%)  1 1/12 (8.33%)  1
Rash erythematous  1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/12 (8.33%)  1
Urticaria  1  2/11 (18.18%)  2 1/12 (8.33%)  1 0/12 (0.00%)  0
1
Term from vocabulary, MedDRA (20.0)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
 
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Taiho Pharmaceutical Co., Ltd.
Organization: Clinical Trial Registration Contact
Phone: +81-3-3294-4527
EMail: toiawase@taiho.co.jp
Layout table for additonal information
Responsible Party: Taiho Pharmaceutical Co., Ltd.
ClinicalTrials.gov Identifier: NCT02752048    
Other Study ID Numbers: Taiho10053040
First Submitted: April 6, 2016
First Posted: April 26, 2016
Results First Submitted: March 4, 2020
Results First Posted: April 20, 2020
Last Update Posted: April 20, 2020