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The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator |
| |
| TAS-205 low dose | Active Comparator |
| |
| TAS-205 middle dose | Active Comparator |
| |
| TAS-205 high dose | Active Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TAS-205 | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Adverse Events | Source Vocabulary Name for Table Default: CTCAE (4.03) | From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days) |
| Measure | Description | Time Frame |
|---|---|---|
| Peak Plasma Concentration (Cmax) of TAS-205 | Due to inspection missing, some data were not analyzed. | 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. |
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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 |
|---|---|---|---|---|---|---|
| National Center of Neurology and Psychiatry | Tokyo | 187-8551 | Japan |
Two participants in 21 participants of Single-dose phase discontinued after Single-dose phase. So, 19 participants moved to Multiple-dose phase. And new two participants enrolled in Multiple-dose phase.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Placebo: ・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 |
| FG001 | TAS-205 Low Dose | TAS-205: ・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 |
| FG002 | TAS-205 Middle Dose | TAS-205: ・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 |
| FG003 | TAS-205 High Dose | TAS-205: ・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 |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Single-dose Phase |
| |||||||||||||
| Multiple-dose Phase |
|
Two participant were discontinued in Single-dose phase, and other two participants were added in Multiple-dose phase.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Placebo: ・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 |
| 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 | Incidence of Adverse Events | Source Vocabulary Name for Table Default: CTCAE (4.03) | About repeated-dose period, one participants of placebo group and one of TAS-205 high dose group were excluded from analysis population, due to significant deviation from the protocol. | Posted | Number | participants | From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days) |
|
From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days)
Adverse Events were formed by participants of single-dose period and multiple-dose period.
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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 | Placebo, Single-dose Phase | Single-dose phase: 3steps (low dose, middle dose or high dose group), 2 patients/step, single oral administration after meals |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Catheter site pain | General disorders | CTCAE (4.03) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Taiho Pharmaceutical Co., Ltd. | Clinical Trial Registration Contact | toiawase@taiho.co.jp |
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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 |
|
|
| Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205 |
Due to inspection missing, some data were not analyzed. |
| 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) |
| The Urinary Excretion of PD Marker | Ratio of prostaglandin E2 metabolite / creatinine Due to inspection missing, some data were not analyzed. | 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. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| BG001 |
| TAS-205 Low Dose |
TAS-205: ・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 |
| BG002 | TAS-205 Middle Dose | TAS-205: ・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 |
| BG003 | TAS-205 High Dose | TAS-205: ・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 |
| BG004 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| OG001 |
| TAS-205 Low Dose |
TAS-205: ・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 |
| OG002 | TAS-205 Middle Dose | TAS-205: ・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 |
| OG003 | TAS-205 High Dose | TAS-205: ・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 |
|
|
| Secondary | Peak Plasma Concentration (Cmax) of TAS-205 | Due to inspection missing, some data were not analyzed. | About repeated-dose period, one participants of TAS-205 high dose group was excluded from analysis population, due to significant deviation from the protocol. | Posted | Mean | Standard Deviation | ng/mL | 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. |
|
|
|
| Secondary | Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205 | Due to inspection missing, some data were not analyzed. | About repeated-dose period, one paticipant of TAS-205 high dose group was excluded from analysis population, due to significant deviation from the protocol. | Posted | Mean | Standard Deviation | ng*hr/mL | 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) |
|
|
|
| Secondary | The Urinary Excretion of PD Marker | Ratio of prostaglandin E2 metabolite / creatinine Due to inspection missing, some data were not analyzed. | About repeated-dose period, one participants of placebo group and one of TAS-205 high dose group were excluded from analysis population, due to significant deviation from the protocol. | Posted | Mean | Standard Deviation | ratio | 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. |
|
|
|
| 0 |
| 6 |
| 0 |
| 6 |
| 0 |
| 6 |
| EG001 | TAS-205 Low Dose, Single-dose Phase | Single-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step, single oral administration after meals | 0 | 5 | 0 | 5 | 3 | 5 |
| EG002 | TAS-205 Middle Dose, Single-dose Phase | Single-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step, single oral administration after meals | 0 | 5 | 0 | 5 | 1 | 5 |
| EG003 | TAS-205 High Dose, Single-dose | Single-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step, single oral administration after meals | 0 | 5 | 0 | 5 | 1 | 5 |
| EG004 | Placebo, Multiple-dose Phase | 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 | 0 | 5 | 0 | 5 | 0 | 5 |
| EG005 | TAS-205 Low Dose, Multiple-dose Phase | 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 | 0 | 5 | 0 | 5 | 1 | 5 |
| EG006 | TAS-205 Middle Dose, Multiple-dose Phase | 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 | 0 | 5 | 0 | 5 | 3 | 5 |
| EG007 | TAS-205 High Dose, Multiple-dose Phase | 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 | 0 | 4 | 0 | 4 | 1 | 4 |
| Blood bilirubin increased | Investigations | CTCAE (4.03) | Systematic Assessment |
|
| Cystatin C increased | Investigations | CTCAE (4.03) | Systematic Assessment |
|
| Hyperuricemia | Metabolism and nutrition disorders | CTCAE (4.03) | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | CTCAE (4.03) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | CTCAE (4.03) | Systematic Assessment |
|
| Arthropod sting | Injury, poisoning and procedural complications | CTCAE (4.03) | Systematic Assessment |
|
| Ligament sprain | Injury, poisoning and procedural complications | CTCAE (4.03) | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | CTCAE (4.03) | Systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | CTCAE (4.03) | Systematic Assessment |
|
| Nail bed bleeding | Skin and subcutaneous tissue disorders | CTCAE (4.03) | 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 |
| Title | Measurements |
|---|---|
|
| Repeated-dose Period (Day 1) |
|
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| Repeated-dose Period (Day 7) |
|
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| Repeated-dose Period (Day 1) |
|
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| Repeated-dose Period (Day 7) |
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| Repeated-dose Period (Day 1) |
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| Repeated-dose Period (Day 7) |
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