Idiopathic Thrombocytopenic Purpura
Conditions
Keywords
IVIG, ITP
Brief summary
Idiopathic (immune) thrombocytopenic purpura (ITP) is an autoimmune disorder characterized by platelet destruction and thrombocytopenia (peripheral blood platelet count \< 150 x 10\^9/L). IVIG therapy is useful in patients in whom the platelet count has to be raised either due to bleeding signs, or where bleeding is predicted (e.g., surgery or parturition). The primary goal of treatment is to maintain the platelet count at a hemostatic level. This study will test the safety and efficacy of IGIV3I Grifols 10% in the treatment of patients with chronic ITP.
Interventions
BIOLOGICALIGIV3I Grifols 10%
IGIV3I Grifols 10% 1 g/kg/day given on two consecutive days, Day 1 and Day 2, for a total dose of 2 g/kg over two days.
Sponsors
Instituto Grifols, S.A.
Grifols Biologicals, LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Diagnosis of chronic ITP * Platelet count ≤ 20 x 10\^9/L * When administered corticosteroids at any time within 3 weeks before screening visit, the subject must have completed at least 3 weeks (21 days) of therapy at a stable and constant dose and schedule prior to screening visit * When administered azathioprine (immunosuppressant) at any time within 3 months before screening visit, the subject must have received a stable dose and schedule for at least 3 months prior to screening visit * When administered vinca alkaloids (eg., vincristine) at any time within 2 weeks before screening visit, the subject must have received a stable dose and schedule for at least 2 weeks prior to screening visit * When administered attenuated androgens (eg, danazol) at any time within 8 weeks before screening visit, the subject must have received a stable dose and schedule for at least 8 weeks prior to screening visit. * Females of childbearing potential must test negative for pregnancy Key
Exclusion criteria
* History or clinical evidence of medical conditions (other than ITP) felt to be the underlying cause of the thrombocytopenia * Diagnosis of secondary immune thrombocytopenia * History of severe (eg, anaphylactic) reactions to blood or any blood- derived product * History of intolerance to any component of the IP, such as sorbitol * Suffering serious and/or life-threatening hemorrhage/bleeding defined as: * Any intracranial or central nervous system bleeding * Any hemorrhagic event in which the subject is at risk of death at the time of the event * Females who are pregnant or nursing an infant child * Known to have immunoglobulin A (IgA) deficiency * Known to abuse alcohol, opiates, psychotropic agents or other chemicals or drugs, or has done so within 12 months of the screening visit * Documented diagnosis of thrombotic complications to polyclonal IVIG therapy in the past * Unstable or uncontrolled disease, or condition, related to, or impacting, cardiac function: unstable angina, congestive heart failure, uncontrolled arterial hypertension * Is anemic (hemoglobin \< 9 g/dL) * Renal impairment (ie, serum creatinine \> 1.5 x upper limit of normal \[ULN\]) * Aspartate aminotransferase or alanine aminotransferase levels \> 2.5 x ULN * Known to have a positive test for either HCV or HIV (HIV 1/2) * Splenectomy within the prior 8 weeks to the screening visit * currently receiving any treatment for ITP except corticosteroids, azathioprine, vinca alkaloids or danazol * Received an immune serum globulin (ISG) product within the prior 3 weeks (21 days) to the screening visit * Received any alkylating agent (eg, cyclophosphamide) within 5 weeks prior to the screening visit * Received rituximab within the prior 3 months to the screening visit * Was currently receiving, or received, any therapeutic drug or device that was not approved by a Regulatory Authority (US or Canadian) for any indication within the prior 12 weeks to the screening visit
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Response Rate | 8 days | Defined by the percentage of treated patients in whom platelet counts increase from ≤ 20 x 10\^9/L to ≥ 50 x 10\^9/L by Day 8 ± 1 \[where the day of the first infusion is Day 1\] |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to Platelet Count Recovery | 30 days | Defined by the number of days elapsed from Day 1 (the day of the first infusion of the IP) to the day when the platelet count is first known to be ≥ 50 x 10\^9/L at any moment during the clinical follow-up period ending on Day 30 ± 1 |
| Duration of Response | 30 days | Defined by the number of consecutive days for which the platelet count remains ≥ 50 x 10\^9/L at any moment during the clinical follow-up period ending on Day 30 ± 1. |
| Regression of Hemorrhage/Bleedings | 15 days | Defined by the percentage of treated patients with hemorrhage/bleedings at Day 1 (i.e., the day of the first infusion, pre-infusion) who improve their diathesis during the clinical follow-up period ending on Day 15 ± 1. |
Countries
Canada, India, Puerto Rico, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| IGIV3I Grifols 10% (All Subjects) Intravenous immunoglobulin
IGIV3I Grifols 10%: IGIV3I Grifols 10% 1 g/kg/day given on two consecutive days, Day 1 and Day 2, for a total dose of 2 g/kg over two days. | 64 |
| Total | 64 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Disease progression | 5 |
| Overall Study | Required platelet transfusion | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | IGIV3I Grifols 10% (All Subjects) |
|---|---|
| Age, Continuous | 33.4 years STANDARD_DEVIATION 18.5 |
| Race/Ethnicity, Customized Asian | 32 Participants |
| Race/Ethnicity, Customized Black/African American | 2 Participants |
| Race/Ethnicity, Customized Hispanic/Latino | 6 Participants |
| Race/Ethnicity, Customized White | 24 Participants |
| Sex: Female, Male Female | 43 Participants |
| Sex: Female, Male Male | 21 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 64 |
| other Total, other adverse events | 59 / 64 |
| serious Total, serious adverse events | 4 / 64 |
Outcome results
Primary
Response Rate
Defined by the percentage of treated patients in whom platelet counts increase from ≤ 20 x 10\^9/L to ≥ 50 x 10\^9/L by Day 8 ± 1 \[where the day of the first infusion is Day 1\]
Time frame: 8 days
Population: Modified ITT Population
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| IGIV3I Grifols 10% (All Subjects) | Response Rate | 81.3 percentage of responders |
Secondary
Duration of Response
Defined by the number of consecutive days for which the platelet count remains ≥ 50 x 10\^9/L at any moment during the clinical follow-up period ending on Day 30 ± 1.
Time frame: 30 days
Population: Number of participants analyzed is based on the number of responding patients (52/64 \[81.3%\]) in the Modified ITT Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| IGIV3I Grifols 10% (All Subjects) | Duration of Response | 10.8 days | Standard Deviation 7.69 |
Secondary
Regression of Hemorrhage/Bleedings
Defined by the percentage of treated patients with hemorrhage/bleedings at Day 1 (i.e., the day of the first infusion, pre-infusion) who improve their diathesis during the clinical follow-up period ending on Day 15 ± 1.
Time frame: 15 days
Population: Number of participants analyzed is based on the number of patients with hemorrhage/bleeding at Day 1 in the Modified ITT Population
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| IGIV3I Grifols 10% (All Subjects) | Regression of Hemorrhage/Bleedings | 90.9 percent of subjects with regression |
Secondary
Time to Platelet Count Recovery
Defined by the number of days elapsed from Day 1 (the day of the first infusion of the IP) to the day when the platelet count is first known to be ≥ 50 x 10\^9/L at any moment during the clinical follow-up period ending on Day 30 ± 1
Time frame: 30 days
Population: Number of participants analyzed is based on the number of responding patients (52/64 \[81.3%\]) in the Modified ITT Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| IGIV3I Grifols 10% (All Subjects) | Time to Platelet Count Recovery | 1.7 days | Standard Deviation 0.85 |