Trial Outcomes & Findings for A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy (NCT NCT02752048)

NCT ID: NCT02752048

Last Updated: 2020-04-20

Results Overview

The distance the subject can walk as fast as possible in 6 minutes will be evaluated.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

36 participants

Primary outcome timeframe

baseline, 24 weeks

Results posted on

2020-04-20

Participant Flow

Participant milestones

Participant milestones
Measure	TAS-205（Low Dose Group） TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	TAS-205（High Dose Group） TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	Placebo Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Overall Study STARTED	11	12	13
Overall Study COMPLETED	11	12	10
Overall Study NOT COMPLETED	0	0	3

Reasons for withdrawal

Reasons for withdrawal
Measure	TAS-205（Low Dose Group） TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	TAS-205（High Dose Group） TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	Placebo Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Overall Study Withdrawal by Subject	0	0	3

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	TAS-205（Low Dose Group） n=11 Participants TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	TAS-205（High Dose Group） n=11 Participants TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	Placebo n=10 Participants Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal	Total n=32 Participants Total of all reporting groups
Age, Categorical <=18 years	11 Participants n=5 Participants	11 Participants n=7 Participants	10 Participants n=5 Participants	32 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Sex: Female, Male Male	11 Participants n=5 Participants	11 Participants n=7 Participants	10 Participants n=5 Participants	32 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	11 Participants n=5 Participants	11 Participants n=7 Participants	10 Participants n=5 Participants	32 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Region of Enrollment Japan	11 participants n=5 Participants	11 participants n=7 Participants	10 participants n=5 Participants	32 participants n=4 Participants

PRIMARY outcome

Timeframe: baseline, 24 weeks

The distance the subject can walk as fast as possible in 6 minutes will be evaluated.

Outcome measures

Outcome measures
Measure	TAS-205（Low Dose Group） n=11 Participants TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	TAS-205（High Dose Group） n=11 Participants TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	Placebo n=10 Participants Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)	-3.5 meter Standard Deviation 67.3	-7.5 meter Standard Deviation 37.3	-17.0 meter Standard Deviation 55.6

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	TAS-205（Low Dose Group） n=11 Participants TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	TAS-205（High Dose Group） n=11 Participants TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	Placebo n=10 Participants Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Mean Change From Baseline in Time to Rise From the Floor	4.011 second Standard Deviation 6.209	1.283 second Standard Deviation 2.547	2.633 second Standard Deviation 4.246

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	TAS-205（Low Dose Group） n=11 Participants TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	TAS-205（High Dose Group） n=11 Participants TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	Placebo n=10 Participants Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Mean Change From Baseline in Time to Walk/Run for 10meters	1.113 second Standard Deviation 1.140	1.025 second Standard Deviation 1.296	0.629 second Standard Deviation 1.272

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	TAS-205（Low Dose Group） n=11 Participants TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	TAS-205（High Dose Group） n=11 Participants TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	Placebo n=10 Participants Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Mean Change From Baseline in Time to up and go (TUG)	0.594 second Standard Deviation 2.156	0.286 second Standard Deviation 1.626	0.061 second Standard Deviation 1.626

Adverse Events

TAS-205（Low Dose Group）

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

TAS-205（High Dose Group）

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Taiho Pharmaceutical Co., Ltd.

Clinical Trial Registration Contact

Phone: +81-3-3294-4527

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER

Other adverse events
Measure	TAS-205（Low Dose Group） n=11 participants at risk TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	TAS-205（High Dose Group） n=12 participants at risk TAS-205: 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal	Placebo n=12 participants at risk Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal
Blood and lymphatic system disorders Lymphadenopathy	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Cardiac disorders Constipation	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Cardiac disorders Dental caries	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Cardiac disorders Diarrhoea	9.1% 1/11 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Cardiac disorders Stomatitis	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
General disorders Mucosal inflammation	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
General disorders Pyrexia	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
General disorders Peripheral swelling	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Bronchitis	9.1% 1/11 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	16.7% 2/12 • Number of events 2 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Gastroenteritis	18.2% 2/11 • Number of events 2 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	25.0% 3/12 • Number of events 3 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Herpes zoster	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Impetigo	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Influenza	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	16.7% 2/12 • Number of events 2 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Mumps	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Pneumonia mycoplasmal	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Rhinitis	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Sinusitis	9.1% 1/11 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Tonsillitis	9.1% 1/11 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Upper respiratory tract infection	9.1% 1/11 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	16.7% 2/12 • Number of events 2 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Viral upper respiratory tract infection	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Beta haemolytic streptococcal infection	9.1% 1/11 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Infections and infestations Pneumonia bacterial	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Injury, poisoning and procedural complications Chillblains	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	16.7% 2/12 • Number of events 2 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Injury, poisoning and procedural complications Excoriation	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Investigations Blood corticotrophin decreased	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Investigations Cortisol decreased	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Investigations Glucose urine present	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Investigations Muscle enzyme increased	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Investigations Cystatin C increased	9.1% 1/11 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Investigations Urobilinogen urine increased	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	16.7% 2/12 • Number of events 2 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Metabolism and nutrition disorders Dehydration	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Musculoskeletal and connective tissue disorders Arthralgia	9.1% 1/11 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	16.7% 2/12 • Number of events 2 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Musculoskeletal and connective tissue disorders Muscle spasms	9.1% 1/11 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Musculoskeletal and connective tissue disorders Myalgia	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Musculoskeletal and connective tissue disorders Pain in extremity	18.2% 2/11 • Number of events 2 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	16.7% 2/12 • Number of events 2 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Nervous system disorders Headache	9.1% 1/11 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Psychiatric disorders Head banging	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Nervous system disorders Vomiting psychogenic	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Respiratory, thoracic and mediastinal disorders Asthma	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/11 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	0.00% 0/12 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration	8.3% 1/12 • Number of events 1 • From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration