Trial Outcomes & Findings for Phase I/II Study of OPB-31121 in Patients With Progressive Hepatocellular Carcinoma (NCT NCT01406574)
NCT ID: NCT01406574
Last Updated: 2015-06-08
Results Overview
Treatment emergent adverse events observed during outcome measure time frame.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
23 participants
Primary outcome timeframe
From first study medication to on Day 32 (after repeated 28 days medication from Day 4 to 32)
Results posted on
2015-06-08
Participant Flow
Participant milestones
| Measure |
OPB-31121: 50mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
OPB-31121: 100mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
OPB-31121: 200mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
OPB-31121: 400mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
7
|
4
|
7
|
5
|
|
Overall Study
COMPLETED
|
6
|
3
|
6
|
1
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
1
|
4
|
Reasons for withdrawal
| Measure |
OPB-31121: 50mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
OPB-31121: 100mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
OPB-31121: 200mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
OPB-31121: 400mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
|---|---|---|---|---|
|
Overall Study
Adverse Event
|
0
|
0
|
1
|
3
|
|
Overall Study
Physician Decision
|
0
|
0
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
0
|
0
|
|
Overall Study
Definite progression of primary disease
|
1
|
0
|
0
|
0
|
Baseline Characteristics
Phase I/II Study of OPB-31121 in Patients With Progressive Hepatocellular Carcinoma
Baseline characteristics by cohort
| Measure |
OPB-31121
n=23 Participants
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
12 Participants
n=5 Participants
|
|
Age, Continuous
|
64.0 years
STANDARD_DEVIATION 7.3 • n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
|
Region of Enrollment
Japan
|
23 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From first study medication to on Day 32 (after repeated 28 days medication from Day 4 to 32)Population: Safety population No statistical analysis provided for Subjects With Treatment Emergent Adverse Events.
Treatment emergent adverse events observed during outcome measure time frame.
Outcome measures
| Measure |
OPB-31121
n=23 Participants
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
OPB-31121: 100 mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
OPB-31121: 200 mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
OPB-31121: 400 mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
|---|---|---|---|---|
|
Subjects With Treatment Emergent Adverse Events
|
23 participants
|
—
|
—
|
—
|
PRIMARY outcome
Timeframe: From first study medication to on Day 32 (after repeated 28 days medication from Day 4 to 32)Population: DLT evaluated subjects who had achieved ≧75% study drug compliance during a 4-week (28-day) treatment period starting from Day 4. No statistical analysis provided for Subjects With DLTs.
Recommended Dose (RD) of OPB-31121 was defined as the highest dose at which Dose Limited Toxicity (DLT) occurred at an incidence of \< 30%. DLT was defined as adverse events related to OPB-31121 occurring until Day 32, and 1) Grade 4 neutrophil count decreased persisting for ≧ 8 days, or Grade 3 or 4 febrile neutropenia, or infection with neutrophil count decreased 2) Grade 4 Plt decreased, or Grade 3 Plt decreased persisting for ≧ 8 days 3) Grade 3 or 4 nausea, vomiting, or diarrhoea that occurred despite the use of an anti-emetic or anti-diarrheal agents 4) Grade 3 or more severe AEsa excluding the AEs presented above 1) to 3) 5) AEs requiring interruption of IMP administration for a period of ≧ 8 consecutive days 6) Same AEs causing interruption of IMP administration twice
Outcome measures
| Measure |
OPB-31121
n=7 Participants
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
OPB-31121: 100 mg/Day
n=4 Participants
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
OPB-31121: 200 mg/Day
n=7 Participants
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
OPB-31121: 400 mg/Day
n=5 Participants
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
|---|---|---|---|---|
|
Number of Participants Who Experienced Dose-Limiting Toxicities (DLTs)
|
0 participants
|
0 participants
|
1 participants
|
4 participants
|
SECONDARY outcome
Timeframe: From first dose of study medication up to 28 weeksPopulation: Efficacy population included all treated subjects who had received at least 1 dose of study drug. No statistical analysis provided for Best Overall Responders.
Overall response was evaluated based on the Response Evaluation Criteria in Solid Tumors (RECIST guideline) - mRECIST 1.0.
Outcome measures
| Measure |
OPB-31121
n=23 Participants
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
OPB-31121: 100 mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
OPB-31121: 200 mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
OPB-31121: 400 mg/Day
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle
|
|---|---|---|---|---|
|
Best Overall Response
CR or PR
|
0 participants
|
—
|
—
|
—
|
|
Best Overall Response
SD ≧ 8w
|
6 participants
|
—
|
—
|
—
|
|
Best Overall Response
PD
|
9 participants
|
—
|
—
|
—
|
|
Best Overall Response
NE
|
8 participants
|
—
|
—
|
—
|
Adverse Events
OPB-31121: 50 mg/Day
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
OPB-31121: 100 mg/Day
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
OPB-31121: 200 mg/Day
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
OPB-31121: 400 mg/Day
Serious events: 2 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
OPB-31121: 50 mg/Day
n=7 participants at risk
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
OPB-31121: 100 mg/Day
n=4 participants at risk
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
OPB-31121: 200 mg/Day
n=7 participants at risk
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
OPB-31121: 400 mg/Day
n=5 participants at risk
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
|---|---|---|---|---|
|
General disorders
Gait disturbance
|
0.00%
0/7 • From first study medication to end-of-trial examination
|
0.00%
0/4 • From first study medication to end-of-trial examination
|
0.00%
0/7 • From first study medication to end-of-trial examination
|
20.0%
1/5 • Number of events 1 • From first study medication to end-of-trial examination
|
|
Hepatobiliary disorders
Jaundice
|
0.00%
0/7 • From first study medication to end-of-trial examination
|
0.00%
0/4 • From first study medication to end-of-trial examination
|
0.00%
0/7 • From first study medication to end-of-trial examination
|
20.0%
1/5 • Number of events 1 • From first study medication to end-of-trial examination
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
0.00%
0/7 • From first study medication to end-of-trial examination
|
0.00%
0/4 • From first study medication to end-of-trial examination
|
0.00%
0/7 • From first study medication to end-of-trial examination
|
20.0%
1/5 • Number of events 1 • From first study medication to end-of-trial examination
|
Other adverse events
| Measure |
OPB-31121: 50 mg/Day
n=7 participants at risk
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
OPB-31121: 100 mg/Day
n=4 participants at risk
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
OPB-31121: 200 mg/Day
n=7 participants at risk
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
OPB-31121: 400 mg/Day
n=5 participants at risk
OPB-31121: 50, 100, 200 and 400 mg/day oral once daily (QD) in a 4 week cycle.
|
|---|---|---|---|---|
|
Blood and lymphatic system disorders
Leukopenia
|
0.00%
0/7 • From first study medication to end-of-trial examination
|
0.00%
0/4 • From first study medication to end-of-trial examination
|
0.00%
0/7 • From first study medication to end-of-trial examination
|
60.0%
3/5 • Number of events 3 • From first study medication to end-of-trial examination
|
|
Gastrointestinal disorders
Nausea
|
57.1%
4/7 • Number of events 4 • From first study medication to end-of-trial examination
|
100.0%
4/4 • Number of events 4 • From first study medication to end-of-trial examination
|
100.0%
7/7 • Number of events 7 • From first study medication to end-of-trial examination
|
100.0%
5/5 • Number of events 5 • From first study medication to end-of-trial examination
|
|
Gastrointestinal disorders
Vomiting
|
57.1%
4/7 • Number of events 4 • From first study medication to end-of-trial examination
|
75.0%
3/4 • Number of events 3 • From first study medication to end-of-trial examination
|
100.0%
7/7 • Number of events 7 • From first study medication to end-of-trial examination
|
100.0%
5/5 • Number of events 5 • From first study medication to end-of-trial examination
|
|
Gastrointestinal disorders
Diarrhea
|
28.6%
2/7 • Number of events 2 • From first study medication to end-of-trial examination
|
100.0%
4/4 • Number of events 4 • From first study medication to end-of-trial examination
|
85.7%
6/7 • Number of events 6 • From first study medication to end-of-trial examination
|
80.0%
4/5 • Number of events 4 • From first study medication to end-of-trial examination
|
|
Gastrointestinal disorders
Fatigue/malaise
|
71.4%
5/7 • Number of events 5 • From first study medication to end-of-trial examination
|
25.0%
1/4 • Number of events 1 • From first study medication to end-of-trial examination
|
57.1%
4/7 • Number of events 4 • From first study medication to end-of-trial examination
|
40.0%
2/5 • Number of events 2 • From first study medication to end-of-trial examination
|
|
Investigations
Neutrophil count decreased
|
14.3%
1/7 • Number of events 1 • From first study medication to end-of-trial examination
|
25.0%
1/4 • Number of events 1 • From first study medication to end-of-trial examination
|
14.3%
1/7 • Number of events 1 • From first study medication to end-of-trial examination
|
0.00%
0/5 • From first study medication to end-of-trial examination
|
|
Investigations
Platelet count decreased
|
14.3%
1/7 • Number of events 1 • From first study medication to end-of-trial examination
|
25.0%
1/4 • Number of events 1 • From first study medication to end-of-trial examination
|
14.3%
1/7 • Number of events 1 • From first study medication to end-of-trial examination
|
0.00%
0/5 • From first study medication to end-of-trial examination
|
|
Metabolism and nutrition disorders
Anorexia
|
14.3%
1/7 • Number of events 1 • From first study medication to end-of-trial examination
|
50.0%
2/4 • Number of events 2 • From first study medication to end-of-trial examination
|
42.9%
3/7 • Number of events 3 • From first study medication to end-of-trial examination
|
100.0%
5/5 • Number of events 5 • From first study medication to end-of-trial examination
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
0.00%
0/7 • From first study medication to end-of-trial examination
|
0.00%
0/4 • From first study medication to end-of-trial examination
|
57.1%
4/7 • Number of events 4 • From first study medication to end-of-trial examination
|
40.0%
2/5 • Number of events 2 • From first study medication to end-of-trial examination
|
Additional Information
Leader of Department of "Small Global" Clinical Development
Otsuka Pharmaceutical Co., Ltd
Phone: +81-3-6361-7366
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place