Idiopathic Thrombocytopenic Purpura
Conditions
Brief summary
The purpose of this study is to investigate the efficacy, safety and pharmacokinetics of R788 compared with placebo, and to investigate the safety and efficacy of long term dosing of R788 in patients with chronic idiopathic thrombocytopenic purpura.
Interventions
DRUGR788
Oral administration
DRUGPlacebo
Oral administration
Sponsors
Kissei Pharmaceutical Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
20 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Japanese patients * Patients diagnosed with idiopathic thrombocytopenic purpura at least 6 months before acquisition of consent * Patients with a platelet count averages \<30000/μL during screening period. Each platelet count should not exceed 35000/μL. * Patients who have used and failed or who were intolerant at least 1 typical regimen for the treatment of ITP before informed consent (with or without splenectomy)
Exclusion criteria
* Patients with thrombocytopenia associated with other disease * Patients with autoimmune hemolytic anemia * Patients with poorly controlled hypertension * Patients with a history or active coagulopathy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Achievement Rate of Stable Platelet Response | 24 weeks (Period I) | The percentage of subjects who achieved stable platelet response (defined as a platelet count of ≥50000/μL at 4 or more of the 6 visits from Weeks 14 to 24) |
Other
| Measure | Time frame | Description |
|---|---|---|
| Duration of Platelet Response | R788 treatment period (maximum duration of exposure was 1184 days) | Period from the first measurement day on which a platelet count of ≥50000/μL for at least 28 consecutive days was achieved to the first measurement day on which platelet count fell below 50000/μL for at least 28 consecutive days |
Countries
Japan
Participant flow
Recruitment details
Seventy-two subjects were screened. Of these, 34 subjects considered eligible for the study were randomized to receive the investigational products. The number of subjects in Period I were 22 in the R788 group and 12 in the placebo group. The number of subjects who completed Period I was 10 in the R788 group and 4 in the placebo group.
Participants by arm
| Arm | Count |
|---|---|
| R788 Group R788 will be administered orally for 24 weeks in the Period I. | 22 |
| Placebo Group R788 placebo will be administered orally for 24 weeks in the Period I. | 12 |
| Total | 34 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Period I (Double-blind Period) | Adverse Event | 3 | 0 |
| Period I (Double-blind Period) | Lack of Efficacy | 9 | 8 |
| R788 Treatment Period | Adverse Event | 5 | 0 |
| R788 Treatment Period | Lack of Efficacy | 5 | 0 |
| R788 Treatment Period | Withdrawal by Subject | 1 | 0 |
Baseline characteristics
| Characteristic | R788 Group | Placebo Group | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 10 Participants | 5 Participants | 15 Participants |
| Age, Categorical Between 18 and 65 years | 12 Participants | 7 Participants | 19 Participants |
| Age, Continuous | 59.6 years STANDARD_DEVIATION 15.4 | 60.8 years STANDARD_DEVIATION 15.4 | 60.0 years STANDARD_DEVIATION 15.2 |
| Baseline platelet count | 19000 cells/μL | 18000 cells/μL | 18000 cells/μL |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 22 Participants | 12 Participants | 34 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| History of splenectomy | 5 Participants | 2 Participants | 7 Participants |
| Line of Therapy 2 | 10 Participants | 4 Participants | 14 Participants |
| Line of Therapy ≥3 | 12 Participants | 8 Participants | 20 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 22 Participants | 12 Participants | 34 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 0 Participants | 0 Participants | 0 Participants |
| Region of Enrollment Japan | 22 Participants | 12 Participants | 34 Participants |
| Sex: Female, Male Female | 18 Participants | 8 Participants | 26 Participants |
| Sex: Female, Male Male | 4 Participants | 4 Participants | 8 Participants |
| Time since ITP diagnosis | 12 years | 12 years | 12 years |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 22 | 0 / 12 | 0 / 33 |
| other Total, other adverse events | 15 / 22 | 7 / 12 | 32 / 33 |
| serious Total, serious adverse events | 2 / 22 | 1 / 12 | 8 / 33 |
Outcome results
Primary
Achievement Rate of Stable Platelet Response
The percentage of subjects who achieved stable platelet response (defined as a platelet count of ≥50000/μL at 4 or more of the 6 visits from Weeks 14 to 24)
Time frame: 24 weeks (Period I)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| R788 Group | Achievement Rate of Stable Platelet Response | 36.4 Percentage of participants |
| Placebo Group | Achievement Rate of Stable Platelet Response | 0.0 Percentage of participants |
p-value: 0.0395% CI: [3.1, 59.3]Fisher Exact
Other Pre-specified
Duration of Platelet Response
Period from the first measurement day on which a platelet count of ≥50000/μL for at least 28 consecutive days was achieved to the first measurement day on which platelet count fell below 50000/μL for at least 28 consecutive days
Time frame: R788 treatment period (maximum duration of exposure was 1184 days)
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| R788 Group | Duration of Platelet Response | 392 days |