Immune Thrombocytopenic Purpura
Conditions
Keywords
Chronic Immune Thrombocytopenic Purpura, Chronic Idiopathic Thrombocytopenic Purpura, Werlhofs Disease, Autoimmune Thrombocytopenia, Immunglobulin Intravenous, Chronic ITP, Platelet count, Thrombocytopenia
Brief summary
The purpose of this study is to evaluate the efficacy, tolerability and safety of IgPro10 in the treatment of patients with chronic immune thrombocytopenic purpura (ITP). The main efficacy parameter is the proportion of patients responding to treatment by an increase of platelet count to ≥ 50 x 10\^9/L.
Interventions
BIOLOGICALImmunoglobulin Intravenous (Human)
A dose of 1 g IgG per kg body weight (bw) administered on two consecutive days resulting in the total treatment dosage of 2 g IgG per kg bw.
Sponsors
CSL Behring
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Diagnosis of chronic ITP defined by: Failure to find other causes of thrombocytopenia; Platelet count ≤ 150 x 10\^9/L over 6 months or response to a previous treatment with subsequent decrease in platelet count even if duration of chronic ITP is less than 6 months * Platelet counts ≤ 20 x 10\^9/L Key
Exclusion criteria
* Planned splenectomy throughout the study period * Treatment with IVIG or anti-D immunoglobulin within 3 weeks prior to screening * Treatment with immunosuppressive or other immunomodulatory drugs within 3 weeks prior to screening * Treatment with intravenous steroids within 10 days prior to screening * Change of oral steroid treatment within 15 days prior to screening * Patients with known or suspected hypersensitivity to immunoglobulins or previous severe side effects to immunoglobulin therapy * Abnormal results in the following laboratory parameters: Hemoglobin \< 10 g/dL; Total bilirubin \> 1.5 x upper normal limit; ALAT \> 2.5 x upper normal limit; ASAT \> 2.5 x upper normal limit; Creatinine \> 1.5 x upper normal limit; Urea \> 1.5 x upper normal limit * Positive direct Coombs test * Patients with one of the following concomitant diseases Clinical active SLE Known or suspected HIV infection Acute hepatitis Clinically active chronic hepatitis Lymphoproliferative disease Heart failure Grade III or IV according to the New York Heart Association classification * Any other concomitant disease that has influence on the clotting system (i.e. hemophilia)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Platelet Response | 7 days | The platelet response rate is defined as the percentage of subjects responding to treatment with an increase of platelet count from ≤ 20 x 10\^9/L to ≥ 50 x 10\^9/L within the specified time frame. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Maximum Platelet Level | 29 days | Maximum absolute platelet count achieved over the duration of the study. |
| Regression of Hemorrhage (Skin) | up to 29 days | Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal. |
| Regression of Hemorrhage (Oral Cavity) | 29 days | Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal. |
| Duration of Platelet Response | up to 29 days | The number of days the platelet count remained ≥ 50 x 10\^9/L. |
| Regression of Hemorrhage (Nose) | 29 days | Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal. |
| Regression of Hemorrhage (Internal) | 29 days | Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal. |
| Time to Platelet Response | 29 days | Median time to reach a platelet count ≥ 50 x 10\^9/L. |
| Regression of Hemorrhage (Genitourinary Tract) | 29 days | Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal. |
Countries
Germany, Italy, Poland, Russia, Ukraine, United Kingdom
Participant flow
Recruitment details
The study was performed as a multicenter study at 17 centers, 6 in Poland, 4 in the Ukraine, 4 in Russia, 1 in Germany, 1 in Italy and 1 in the United Kingdom (UK).
Pre-assignment details
One enrolled subject was withdrawn prior to receiving treatment due to a non-fatal adverse event. This subject was not included in the analyses.
Participants by arm
| Arm | Count |
|---|---|
| IgPro10 All subjects treated with IgPro10 | 57 |
| Total | 57 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Therapy failure | 1 |
Baseline characteristics
| Characteristic | IgPro10 |
|---|---|
| Age Continuous | 38 years STANDARD_DEVIATION 15 |
| Body Mass Index | 25.69 kg/m^2 STANDARD_DEVIATION 4.56 |
| Race/Ethnicity, Customized Caucasian | 57 participants |
| Sex: Female, Male Female | 34 Participants |
| Sex: Female, Male Male | 23 Participants |
| Weight | 74.0 kg STANDARD_DEVIATION 15.5 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 52 / 57 |
| serious Total, serious adverse events | 3 / 57 |
Outcome results
Primary
Platelet Response
The platelet response rate is defined as the percentage of subjects responding to treatment with an increase of platelet count from ≤ 20 x 10\^9/L to ≥ 50 x 10\^9/L within the specified time frame.
Time frame: 7 days
Population: Intention to treat (ITT) analysis. The ITT population comprised all subjects who received at least once study medication.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| IgPro10 | Platelet Response | 80.7 Percent of participants |
Secondary
Duration of Platelet Response
The number of days the platelet count remained ≥ 50 x 10\^9/L.
Time frame: up to 29 days
Population: Analyzed for responders in the ITT population, i.e., only subjects with at least one platelet measurement ≥ 50 x 10\^9/L after start of treatment
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| IgPro10 | Duration of Platelet Response | 15.4 days |
Secondary
Maximum Platelet Level
Maximum absolute platelet count achieved over the duration of the study.
Time frame: 29 days
Population: ITT analysis. The ITT population comprised all subjects who received at least once study medication.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| IgPro10 | Maximum Platelet Level | 154 10^9/L |
Secondary
Regression of Hemorrhage (Genitourinary Tract)
Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal.
Time frame: 29 days
Population: The number of participants analyzed represents the number of subjects in the ITT population with genitourinary tract bleeding at baseline and respective post-baseline assessment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| IgPro10 | Regression of Hemorrhage (Genitourinary Tract) | 7 participants |
Secondary
Regression of Hemorrhage (Internal)
Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal.
Time frame: 29 days
Population: The number of participants analyzed represents the number of subjects in the ITT population with internal bleeding at baseline and respective post-baseline assessment.
Secondary
Regression of Hemorrhage (Nose)
Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal.
Time frame: 29 days
Population: The number of participants analyzed represents the number of subjects in the ITT population with nose bleeding at baseline and respective post-baseline assessment.
Secondary
Regression of Hemorrhage (Oral Cavity)
Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal.
Time frame: 29 days
Population: The number of participants analyzed represents the number of subjects in the ITT population with oral cavity bleeding at baseline and respective post-baseline assessment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| IgPro10 | Regression of Hemorrhage (Oral Cavity) | 11 participants |
Secondary
Regression of Hemorrhage (Skin)
Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal.
Time frame: up to 29 days
Population: The number of participants analyzed represents the number of subjects in the ITT population with skin bleeding at baseline and respective post-baseline assessment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| IgPro10 | Regression of Hemorrhage (Skin) | 31 participants |
Secondary
Time to Platelet Response
Median time to reach a platelet count ≥ 50 x 10\^9/L.
Time frame: 29 days
Population: ITT analysis. The ITT population comprised all subjects who received at least once study medication.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| IgPro10 | Time to Platelet Response | 2.5 days |