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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TAS-205（Low dose group） | Experimental | 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. |
|
| TAS-205（High dose group） | Experimental | 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. |
|
| Placebo | Placebo Comparator | Placebo group: Oral administration of tablets for 24 weeks, BID after meal |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TAS-205 | Drug | 2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change From Baseline in Time to Rise From the Floor | The time required for the subject to rise from a supine position on the floor as quickly as possible will be evaluated. | baseline, and 24 weeks |
| Mean Change From Baseline in Time to Walk/Run for 10meters |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Taiho Pharmaceutical Co., Ltd. | Taiho Pharmaceutical Co., Ltd. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nagoya City University Hospital | Aichi | 467-8601 | Japan | |||
| National Hospital Organization Nagara Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31957953 | Derived | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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 |
| FG001 | 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 |
| FG002 | Placebo | Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Analysis population was per protocol set. One patients excluded from TAS-205 High dose group, and three excluded from placebo group.
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| ID | Title | Description |
|---|---|---|
| BG000 | 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 |
| BG001 | 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 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Mean | Standard Deviation | meter | baseline, 24 weeks |
|
From registration point to the end of follow-up period which was after four weeks from the end of 24-weeks administration
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA (20.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Taiho Pharmaceutical Co., Ltd. | Clinical Trial Registration Contact | +81-3-3294-4527 | toiawase@taiho.co.jp |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 7, 2016 | Mar 4, 2020 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 14, 2018 | Mar 4, 2020 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| C000712251 | TAS-205 |
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| Placebo | Drug | 1 group: Placebo group. Oral administration for 24 weeks, BID after meal |
|
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. |
| baseline, and 24 weeks |
| Mean Change From Baseline in Time to up and go (TUG) | This test will assess the extent of the subject's composite mobility, including standing up, walking, repositioning the body, and balancing. | baseline, and 24 weeks |
| 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 |
| BG002 | Placebo | Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal |
| BG003 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal |
|
|
| Secondary | Mean Change From Baseline in Time to Rise From the Floor | The time required for the subject to rise from a supine position on the floor as quickly as possible will be evaluated. | Posted | Mean | Standard Deviation | second | baseline, and 24 weeks |
|
|
|
|
| Secondary | Mean Change From Baseline in Time to Walk/Run for 10meters | 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. | Posted | Mean | Standard Deviation | second | baseline, and 24 weeks |
|
|
|
|
| Secondary | Mean Change From Baseline in Time to up and go (TUG) | This test will assess the extent of the subject's composite mobility, including standing up, walking, repositioning the body, and balancing. | Posted | Mean | Standard Deviation | second | baseline, and 24 weeks |
|
|
|
|
| 0 |
| 11 |
| 0 |
| 11 |
| 9 |
| 11 |
| EG001 | 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 | 0 | 12 | 0 | 12 | 9 | 12 |
| EG002 | Placebo | Placebo: 1 group: Placebo group. Oral administration for 24 weeks, BID after meal | 0 | 12 | 0 | 12 | 10 | 12 |
| Constipation | Cardiac disorders | MedDRA (20.0) | Systematic Assessment |
|
| Dental caries | Cardiac disorders | MedDRA (20.0) | Systematic Assessment |
|
| Diarrhoea | Cardiac disorders | MedDRA (20.0) | Systematic Assessment |
|
| Stomatitis | Cardiac disorders | MedDRA (20.0) | Systematic Assessment |
|
| Mucosal inflammation | General disorders | MedDRA (20.0) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (20.0) | Systematic Assessment |
|
| Peripheral swelling | General disorders | MedDRA (20.0) | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Herpes zoster | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Impetigo | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Mumps | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Pneumonia mycoplasmal | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Tonsillitis | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Beta haemolytic streptococcal infection | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Pneumonia bacterial | Infections and infestations | MedDRA (20.0) | Systematic Assessment |
|
| Chillblains | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
|
| Excoriation | Injury, poisoning and procedural complications | MedDRA (20.0) | Systematic Assessment |
|
| Blood corticotrophin decreased | Investigations | MedDRA (20.0) | Systematic Assessment |
|
| Cortisol decreased | Investigations | MedDRA (20.0) | Systematic Assessment |
|
| Glucose urine present | Investigations | MedDRA (20.0) | Systematic Assessment |
|
| Muscle enzyme increased | Investigations | MedDRA (20.0) | Systematic Assessment |
|
| Cystatin C increased | Investigations | MedDRA (20.0) | Systematic Assessment |
|
| Urobilinogen urine increased | Investigations | MedDRA (20.0) | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA (20.0) | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
|
| Head banging | Psychiatric disorders | MedDRA (20.0) | Systematic Assessment |
|
| Vomiting psychogenic | Nervous system disorders | MedDRA (20.0) | Systematic Assessment |
|
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Upper respiratory tract inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA (20.0) | Systematic Assessment |
|
| Dyshidrotic eczema | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Eczema asteatotic | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Leukoderma | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Palmar erythema | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Papule | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (20.0) | Systematic Assessment |
|
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| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| 0.433 |
| Mean Difference (Final Values) |
| 1.349 |
| 2-Sided |
| 95 |
| -2.220 |
| 4.918 |
| Other |
Change in time to rise from the floor from baseline to Week 24 were calculated to evaluate |
| 0.501 |
| Mean Difference (Final Values) |
| -0.397 |
| 2-Sided |
| 95 |
| -1.610 |
| 0.817 |
| Other |
Change in 10-m walk/run test from baseline to Week 24 were calculated to evaluate |
| 0.767 |
| Mean Difference (Final Values) |
| -0.225 |
| 2-Sided |
| 95 |
| -1.801 |
| 1.351 |
| Other |
Change in time to up and go from baseline to Week 24 were calculated to evaluate |