Trial Outcomes & Findings for A Study of TAS-205 for Duchenne Muscular Dystrophy (NCT NCT02246478)

NCT ID: NCT02246478

Last Updated: 2021-06-04

Results Overview

Source Vocabulary Name for Table Default: CTCAE (4.03)

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

23 participants

Primary outcome timeframe

From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days)

Results posted on

2021-06-04

Participant Flow

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.

Participant milestones

Participant milestones
Measure	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	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	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	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
Single-dose Phase STARTED	6	5	5	5
Single-dose Phase COMPLETED	6	5	5	5
Single-dose Phase NOT COMPLETED	0	0	0	0
Multiple-dose Phase STARTED	6	5	5	5
Multiple-dose Phase COMPLETED	5	5	5	4
Multiple-dose Phase NOT COMPLETED	1	0	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	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	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	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	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
Multiple-dose Phase Protocol Violation	1	0	0	1

Baseline Characteristics

A Study of TAS-205 for Duchenne Muscular Dystrophy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=6 Participants 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	TAS-205 Low Dose n=6 Participants 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	TAS-205 Middle Dose n=5 Participants 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	TAS-205 High Dose n=6 Participants 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	Total n=23 Participants Total of all reporting groups
Age, Categorical <=18 years	6 Participants n=5 Participants	6 Participants n=7 Participants	5 Participants n=5 Participants	6 Participants n=4 Participants	23 Participants n=21 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	0 Participants n=21 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	0 Participants n=21 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	0 Participants n=21 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	6 Participants n=7 Participants	5 Participants n=5 Participants	6 Participants n=4 Participants	23 Participants n=21 Participants
Region of Enrollment Japan	6 participants n=5 Participants	6 participants n=7 Participants	5 participants n=5 Participants	6 participants n=4 Participants	23 participants n=21 Participants

PRIMARY outcome

Timeframe: From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days)

Population: 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.

Source Vocabulary Name for Table Default: CTCAE (4.03)

Outcome measures

Outcome measures
Measure	Placebo n=6 Participants 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	TAS-205 Low Dose n=6 Participants 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	TAS-205 Middle Dose n=5 Participants 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	TAS-205 High Dose n=6 Participants 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
Incidence of Adverse Events Single-dose Period	0 participants	3 participants	1 participants	1 participants
Incidence of Adverse Events Repeated-dose Period	0 participants	1 participants	3 participants	1 participants

SECONDARY outcome

Timeframe: 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.

Population: About repeated-dose period, one participants of TAS-205 high dose group was excluded from analysis population, due to significant deviation from the protocol.

Due to inspection missing, some data were not analyzed.

Outcome measures

Outcome measures
Measure	Placebo n=5 Participants 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	TAS-205 Low Dose n=5 Participants 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	TAS-205 Middle Dose n=5 Participants 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	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
Peak Plasma Concentration (Cmax) of TAS-205 Single-dose Period	839 ng/mL Standard Deviation 383	1847 ng/mL Standard Deviation 996	3202 ng/mL Standard Deviation 1493	—
Peak Plasma Concentration (Cmax) of TAS-205 Repeated-dose Period (Day 1)	1004 ng/mL Standard Deviation 656	1894 ng/mL Standard Deviation 691	2635 ng/mL Standard Deviation 2813	—
Peak Plasma Concentration (Cmax) of TAS-205 Repeated-dose Period (Day 7)	891 ng/mL Standard Deviation 297	2322 ng/mL Standard Deviation 776	4660 ng/mL Standard Deviation 3671	—

SECONDARY outcome

Timeframe: 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)

Population: About repeated-dose period, one paticipant of TAS-205 high dose group was excluded from analysis population, due to significant deviation from the protocol.

Due to inspection missing, some data were not analyzed.

Outcome measures

Outcome measures
Measure	Placebo n=5 Participants 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	TAS-205 Low Dose n=5 Participants 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	TAS-205 Middle Dose n=5 Participants 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	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
Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205 Single-dose Period	2604 ng*hr/mL Standard Deviation 851	5776 ng*hr/mL Standard Deviation 2951	9118 ng*hr/mL Standard Deviation 3334	—
Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205 Repeated-dose Period (Day 1)	2525 ng*hr/mL Standard Deviation 1208	5281 ng*hr/mL Standard Deviation 2553	6391 ng*hr/mL Standard Deviation 4731	—
Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205 Repeated-dose Period (Day 7)	2642 ng*hr/mL Standard Deviation 673	6087 ng*hr/mL Standard Deviation 2561	9452 ng*hr/mL Standard Deviation 4405	—

SECONDARY outcome

Timeframe: 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.

Population: 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.

Ratio of prostaglandin E2 metabolite / creatinine Due to inspection missing, some data were not analyzed.