Immune Thrombocytopenic Purpura
Conditions
Keywords
ITP
Brief summary
It is known that intravenous immunoglobulins can induce hemolysis, but the mechanism is not known in detail. The primary objective of this study was to investigate the specificity of antigens on red blood cells in patients with chronic immune thrombocytopenic purpura (ITP) who have shown signs of clinically relevant hemolysis following treatment with the intravenous immunoglobin Privigen®. The study was to explore potential mechanisms of hemolysis by analysis of the specificity of the antibodies possibly involved. To distinguish between clinically non-relevant hemolysis and a relevant intravascular hemolysis, an independent adjudication by a committee was performed for each patient with signs of hemolysis determined in the laboratory or in the clinic. This study was requested as a post-marketing commitment study by the United States Food and Drug Administration (FDA). By September 2014, no case of clinically significant intravascular hemolysis was found, and the FDA agreed to halt the study and analyze all hemolysis-relevant endpoints using FDA criteria for hemolysis in addition to analyses planned in the protocol. The study was not restarted.
Interventions
BIOLOGICALIgPro10
IgPro10 will be administrated by IV infusion either a single dose of 1 g/kg bw on 1 day or 2 doses of 1 g/kg bw on 2 days (2 g/kg bw total dose) dependent on the response to the first treatment.
Sponsors
CSL Behring
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosis of chronic ITP * Age of 18 to 65 years * Platelet count of ≤ 30 x 10\^9/L at screening
Exclusion criteria
* Planned splenectomy throughout the study period * Treatment with immunoglobulin G for intravenous administration (IVIG) or anti-D immunoglobulin within 3 weeks prior to screening * Use of drugs that have any pharmacological effect on the blood clotting system within 3 weeks prior to screening * Known allergy or other severe reactions to blood products including intolerability to previous IVIG * Known hyperprolinemia * Red blood cell transfusion or erythropoietin treatment within the last 14 days
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Set of Antibodies Most Frequently Bound to Red Blood Cells (RBCs) in Subjects Experiencing Clinically Significant Intravascular Hemolysis | Within 3 days of infusion | The occurrence of clinically significant intravascular hemolysis was determined by an independent Adjudication Committee. No subject experienced clinically significant intravascular hemolysis; therefore, the primary safety endpoint could not be analyzed. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Responder Rate | Within 6 days after the first infusion | The responder rate is the percentage of subjects who have a platelet response (defined as a platelet count increase at least once to ≥ 50 x 10\^9/L after the first IgPro10 administration). |
Countries
Bulgaria, Romania, Serbia
Participant flow
Pre-assignment details
Screening occurred within 6 days before treatment with IgPro10 (Privigen). Subjects who met all of the inclusion criteria and none of the exclusion criteria could be enrolled into the study. Of 58 eligible subjects, one withdrew consent before treatment and 57 subjects received at least 1 dose of Privigen.
Participants by arm
| Arm | Count |
|---|---|
| IgPro10 IgPro10 was administered by IV infusion either as a single dose of 1 g/kg bw on 1 day or 2 doses of 1 g/kg bw on 2 days (2 g/kg bw total dose) dependent on the response to the first IgPro10 dose. | 57 |
| Total | 57 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Physician Decision | 1 |
Baseline characteristics
| Characteristic | IgPro10 |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 2 Participants |
| Age, Categorical Between 18 and 65 years | 55 Participants |
| Age, Continuous | 43.5 years STANDARD_DEVIATION 13.1 |
| Sex: Female, Male Female | 37 Participants |
| Sex: Female, Male Male | 20 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 19 / 57 |
| serious Total, serious adverse events | 1 / 57 |
Outcome results
Primary
Set of Antibodies Most Frequently Bound to Red Blood Cells (RBCs) in Subjects Experiencing Clinically Significant Intravascular Hemolysis
The occurrence of clinically significant intravascular hemolysis was determined by an independent Adjudication Committee. No subject experienced clinically significant intravascular hemolysis; therefore, the primary safety endpoint could not be analyzed.
Time frame: Within 3 days of infusion
Secondary
Responder Rate
The responder rate is the percentage of subjects who have a platelet response (defined as a platelet count increase at least once to ≥ 50 x 10\^9/L after the first IgPro10 administration).
Time frame: Within 6 days after the first infusion
Population: Full analysis set: all subjects who received at least 1 IgPro10 infusion.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| IgPro10 | Responder Rate | 74 percentage of participants |