Immune Thrombocytopenic Purpura
Conditions
Brief summary
Octagam is a solvent/detergent-treated human normal immunoglobulin (IGIV) solution for intravenous administration. Octagam 5% is currently registered in about 80 countries. This study evaluated the efficacy and safety of Octagam 10% in Idiopathic Thrombocytopenic Purpura (ITP) in adults. As Octagam 10% is essentially similar to Octagam 5%, it is expected that Octagam 10% is as efficacious and safe (in respect to viral safety) as Octagam 5%.
Detailed description
The primary objective of the study was to investigate the efficacy of Octagam® 10% in correcting platelet count. The blood count as well as laboratory chemistry were checked repeatedly up to day 21. The secondary objective of the study was to investigate the safety of Octagam® 10%. Safety was assessed by monitoring vital signs, evaluating adverse events, assessing laboratory parameters, and by viral safety testing.
Interventions
DRUGOctagam 10%
Octagam 10% was supplied as a ready-to-use solution in glass bottles.
Sponsors
Octapharma
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
Key Inclusion Criteria: * Diagnosis of Idiopathic Thrombocytopenic Purpura (ITP) according to standard criteria. * Platelet count ≤ 20 x 10\^9/L. Key
Exclusion criteria
* Chronic refractory ITP patients. * Thrombocytopenia secondary to other diseases, or drug related thrombocytopenia. * Administration of IGIV, anti-D, or other platelet enhancing drugs within 30 days before enrollment. * Administration of thrombocyte concentrates within 72 hours before baseline. * Experimental treatment (eg, rituximab) within 3 months before enrollment. * Prophylactic preoperative treatment for elective splenectomy. * Severe liver or kidney disease. * Pregnant or nursing female. * History of hypersensitivity to blood or plasma derived products. * Emergency operation. * Live viral vaccination within the last month prior to study entry. * Known IgA deficiency and antibodies against IgA.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With a Clinical Response | Day 2 to Day 7 | A clinical response is defined as an increase in platelet count to ≥ 50\*10\^9/L on any day from Day 2 to Day 7. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to Achieve a Clinical Response | Day 2 to Day 7 | A clinical response is defined as an increase in platelet count to ≥ 50\*10\^9/L on any day from Day 2 to Day 7. |
| Maximum Platelet Count | Day 2 to the end of the study (Day 63) | Platelet count was assessed on Days 2 through 7 and on Days 14, 21, and 63. The maximum measured platelet count is reported. |
| Duration of the Clinical Response | Day 2 to the end of the study (Day 63) | The duration of the clinical response was the number of days that the platelet count remained ≥ 50\*10\^9/L. Platelet count was assessed on Days 2 through 7 and on Days 14, 21, and 63. A conservative method was used to calculate the duration of the clinical response. For example, if the platelet count was ≥ 50\*10\^9/L on Day 7 and dropped below 50\*10\^9/L at Day 14, Day 7 was used as the last day to calculate the duration of the clinical response. The same procedure was used if the platelet count dropped below 50\*10\^9/L at Day 21 from Day 14 or Day 63 from Day 21. |
| Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7 | Day 7 | The investigator evaluated the severity of bleeding using the following rating scale: None (definitely no haemorrhage of any kind), Minor (few petechiae \[≤ 100 total\] and/or ≤ 5 small bruises \[≤ 3 cm diameter\], no mucosal bleeding), Mild (many petechiae \[\> 100 total\] and/or \> 5 large bruises \[\> 3 cm diameter\], no mucosal bleeding), Moderate (overt mucosal bleeding \[epistaxis, gum bleeding, oropharyngeal blood blisters, menorrhagia, gastrointestinal bleeding, etc\] that does not require immediate medical attention or intervention). |
Countries
Austria
Participant flow
Pre-assignment details
One participant was incorrectly enrolled in the study and was not included in the efficacy analyses but was included in the safety population.
Participants by arm
| Arm | Count |
|---|---|
| Octagam 10% 1 g/kg/Day Participants received Octagam 10% (human normal immunoglobulin) 1 g/kg intravenously once a day for 2 days. | 116 |
| Total | 116 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 5 |
| Overall Study | Incorrectly Enrolled in the Study | 1 |
Baseline characteristics
| Characteristic | Octagam 10% 1 g/kg/Day |
|---|---|
| Age, Continuous | 47.7 Years STANDARD_DEVIATION 19.1 |
| Sex: Female, Male Female | 74 Participants |
| Sex: Female, Male Male | 42 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 79 / 116 |
| serious Total, serious adverse events | 14 / 116 |
Outcome results
Primary
Percentage of Participants With a Clinical Response
A clinical response is defined as an increase in platelet count to ≥ 50\*10\^9/L on any day from Day 2 to Day 7.
Time frame: Day 2 to Day 7
Population: Full analysis set: All participants who received at least 1 dose of study medication, satisfied all major entry criteria, and had at least 1 post-baseline measurement of platelet count.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Octagam 10% 1 g/kg/Day | Percentage of Participants With a Clinical Response | 80.0 Percentage of participants |
Secondary
Duration of the Clinical Response
The duration of the clinical response was the number of days that the platelet count remained ≥ 50\*10\^9/L. Platelet count was assessed on Days 2 through 7 and on Days 14, 21, and 63. A conservative method was used to calculate the duration of the clinical response. For example, if the platelet count was ≥ 50\*10\^9/L on Day 7 and dropped below 50\*10\^9/L at Day 14, Day 7 was used as the last day to calculate the duration of the clinical response. The same procedure was used if the platelet count dropped below 50\*10\^9/L at Day 21 from Day 14 or Day 63 from Day 21.
Time frame: Day 2 to the end of the study (Day 63)
Population: Full analysis set: All participants who received at least 1 dose of study medication, satisfied all major entry criteria, and had at least 1 post-baseline measurement of platelet count. Only participants with a clinical response were included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Octagam 10% 1 g/kg/Day | Duration of the Clinical Response | 24.1 Days | Standard Deviation 23.88 |
Secondary
Maximum Platelet Count
Platelet count was assessed on Days 2 through 7 and on Days 14, 21, and 63. The maximum measured platelet count is reported.
Time frame: Day 2 to the end of the study (Day 63)
Population: Full analysis set: All participants who received at least 1 dose of study medication, satisfied all major entry criteria, and had at least 1 post-baseline measurement of platelet count.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Octagam 10% 1 g/kg/Day | Maximum Platelet Count | 221.6 *10^9/L | Standard Deviation 142.66 |
Secondary
Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7
The investigator evaluated the severity of bleeding using the following rating scale: None (definitely no haemorrhage of any kind), Minor (few petechiae \[≤ 100 total\] and/or ≤ 5 small bruises \[≤ 3 cm diameter\], no mucosal bleeding), Mild (many petechiae \[\> 100 total\] and/or \> 5 large bruises \[\> 3 cm diameter\], no mucosal bleeding), Moderate (overt mucosal bleeding \[epistaxis, gum bleeding, oropharyngeal blood blisters, menorrhagia, gastrointestinal bleeding, etc\] that does not require immediate medical attention or intervention).
Time frame: Day 7
Population: Full analysis set: All participants who received at least 1 dose of study medication, satisfied all major entry criteria, and had at least 1 post-baseline measurement of platelet count.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Octagam 10% 1 g/kg/Day | Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7 | None | 80.9 Percentage of participants |
| Octagam 10% 1 g/kg/Day | Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7 | Minor | 13.0 Percentage of participants |
| Octagam 10% 1 g/kg/Day | Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7 | Mild | 2.6 Percentage of participants |
| Octagam 10% 1 g/kg/Day | Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7 | Moderate | 0.9 Percentage of participants |
| Octagam 10% 1 g/kg/Day | Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7 | Missing | 2.6 Percentage of participants |
Secondary
Time to Achieve a Clinical Response
A clinical response is defined as an increase in platelet count to ≥ 50\*10\^9/L on any day from Day 2 to Day 7.
Time frame: Day 2 to Day 7
Population: Full analysis set: All participants who received at least 1 dose of study medication, satisfied all major entry criteria, and had at least 1 post-baseline measurement of platelet count. Only participants with a clinical response were included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Octagam 10% 1 g/kg/Day | Time to Achieve a Clinical Response | 2.1 Days | Standard Deviation 1.08 |