Purpura, Thrombocytopenic, Idiopathic
Conditions
Brief summary
BI 655064 will be administered subcutaneously once weekly in patients with immune thrombocytopenic purpura (ITP) for up to 12 weeks.
Interventions
DRUGBI 655064
subcutaneous injection
Sponsors
Boehringer Ingelheim
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Male and female chronic immune ITP patients
Exclusion criteria
Any treatment of ITP excluded except for a stable dose of corticosteroids (up to 20 mg)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients With Response in Platelet Count | Up to 12 weeks. | This outcome measure presents number of patients with a response in platelet count. Response was defined as: \[A\] An increase in platelet count by greater than 20 x 10\^9/L from baseline at any time-point between Week 1 and Week 12, and \[B\] Platelet count above 50 x 10\^9/L at any time point between Week 1 and Week 12 with no rescue therapy. Baseline was defined as the platelet count at Visit 2 before administration of BI 655064. In case the patient fulfilled only one of the conditions, he/she was considered a non-responder. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Subjects With Drug-related Adverse Events | From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks. | Number of participants with investigator defined drug-related adverse events (AEs) is reported. |
| Number of Patients Reaching the Cut-off Point for Immune Thrombocytopenic Purpura [ITP] | Up to 12 weeks. | This outcome measure presents the number of patients reaching the cut-off point for ITP, which was defined as: \[A\] Platelet count ≥ 100 x 10\^9/L at any time point between Week 1 and Week 12. |
Countries
United States
Participant flow
Recruitment details
This was an open label, multiple dose with dose escalation for non-responders, multiple injection study in patients with chronic primary immune thrombocytopenic purpura.
Pre-assignment details
All subjects were screened for eligibility prior to participation in the trial. Subjects attended a specialist site which ensured that they (the subjects) strictly met all inclusion and none of the exclusion criteria. Subjects were not to be allocated to a treatment group if any of the entry criteria were violated.
Participants by arm
| Arm | Count |
|---|---|
| BI 655064 120 mg / 180 mg The patients were administered 120 mg BI 655064 solution for subcutaneous injection q1w (once a week) subcutaneously for 4 weeks.
Patients who showed an increase in platelet count above or equal to 100 x 10\^9/L continued treatment with 120 mg BI 655064 solution for subcutaneous injection q1w for additional 8 weeks, followed by 12 weeks of follow-up.
Patients whose platelet count stayed below 100 x 10\^9/L continued treatment for 2 weeks with 180 mg BI 655064 solution for subcutaneous injection q1w followed by 120 mg BI 655064 solution for subcutaneous injection q1w for additional 6 weeks, followed by 12 weeks of follow-up. | 6 |
| Total | 6 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 4 |
Baseline characteristics
| Characteristic | BI 655064 120 mg / 180 mg |
|---|---|
| Age, Continuous | 66.0 Years STANDARD_DEVIATION 9.5 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 5 Participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 6 |
| other Total, other adverse events | 6 / 6 |
| serious Total, serious adverse events | 0 / 6 |
Outcome results
Primary
Number of Patients With Response in Platelet Count
This outcome measure presents number of patients with a response in platelet count. Response was defined as: \[A\] An increase in platelet count by greater than 20 x 10\^9/L from baseline at any time-point between Week 1 and Week 12, and \[B\] Platelet count above 50 x 10\^9/L at any time point between Week 1 and Week 12 with no rescue therapy. Baseline was defined as the platelet count at Visit 2 before administration of BI 655064. In case the patient fulfilled only one of the conditions, he/she was considered a non-responder.
Time frame: Up to 12 weeks.
Population: Treated Set (TS): Included all patients who were dispensed trial medication and were documented to have taken at least one dose of trial medication.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| BI 655064 120 mg / 180 mg | Number of Patients With Response in Platelet Count | No | 5 Participants |
| BI 655064 120 mg / 180 mg | Number of Patients With Response in Platelet Count | Yes | 1 Participants |
Secondary
Number of Patients Reaching the Cut-off Point for Immune Thrombocytopenic Purpura [ITP]
This outcome measure presents the number of patients reaching the cut-off point for ITP, which was defined as: \[A\] Platelet count ≥ 100 x 10\^9/L at any time point between Week 1 and Week 12.
Time frame: Up to 12 weeks.
Population: Treated Set (TS): Included all patients who were dispensed trial medication and were documented to have taken at least one dose of trial medication.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| BI 655064 120 mg / 180 mg | Number of Patients Reaching the Cut-off Point for Immune Thrombocytopenic Purpura [ITP] | No | 6 Participants |
| BI 655064 120 mg / 180 mg | Number of Patients Reaching the Cut-off Point for Immune Thrombocytopenic Purpura [ITP] | Yes | 0 Participants |
Secondary
Number of Subjects With Drug-related Adverse Events
Number of participants with investigator defined drug-related adverse events (AEs) is reported.
Time frame: From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Population: Treated Set (TS): Included all patients who were dispensed trial medication and were documented to have taken at least one dose of trial medication.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BI 655064 120 mg / 180 mg | Number of Subjects With Drug-related Adverse Events | 1 Participants |