Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia
- Conditions
- Primary Thrombocytopenia
- Interventions
- Drug: NewGam
- Registration Number
- NCT01349790
- Lead Sponsor
- Octapharma
- Brief Summary
NewGam is a newly developed human normal immunoglobulin solution for intravenous administration (IGIV). This study will evaluate the safety and efficacy of NewGam 10% in patients with primary immune thrombocytopenia.
- Detailed Description
The primary objective of the study is to assess the efficacy of NewGam in correcting the platelet count. The secondary objective of the study is to evaluate the safety of NewGam. Safety will be assessed by monitoring vital signs, physical examination, evaluation of adverse events (AE) and laboratory parameters, and viral safety testing.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 40
- Age of ≥ 18 and ≤ 65 years.
- Confirmed diagnosis of chronic primary immune thrombocytopenia (ITP) (diagnosed with a threshold platelet count < 100x10^9/L) of at least 12 months duration.
- Platelet count of no more than 20x10^9/L with or without bleeding manifestations.
- Freely given written informed consent from patient.
- Women of childbearing potential must have a negative result on a pregnancy test (human chorionic gonadotropin [HCG]-based assay) and need to practice contraception using a method of proven reliability for the duration of the study.
-
Thrombocytopenia secondary to other diseases (such as acquired immunodeficiency syndrome [AIDS] or systemic lupus erythematosus [SLE]), or drug-related thrombocytopenia.
-
Administration of intravenous immunoglobulin (IGIV), anti-D immunoglobulin or thrombopoietin receptor agonists, or other platelet enhancing drugs (including immunosuppressive or other immunomodulatory drugs) within 3 weeks before enrollment, except for:
- Long-term corticosteroid therapy when the dose has been stable during the preceding 3 weeks and no dosage change is planned until study Day 22.
- Long-term azathioprine, cyclophosphamide, or attenuated androgen therapy when the dose has been stable during the preceding 3 months and no dosage change is planned until study Day 22.
-
Unresponsive to previous treatment with IGIV or anti-D immunoglobulin.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description NewGam NewGam Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
- Primary Outcome Measures
Name Time Method Percentage of Responders Day 1 to Day 8 A responder is a study participant with an increase in platelets to ≥ 50x10\^9/L within 7 days after the first infusion, ie, by study Day 8.
- Secondary Outcome Measures
Name Time Method Duration of a Complete Response Day 1 to Day 22 The duration of a complete response was defined as the time from when a complete response was achieved until the platelet count fell below 50x10\^9/L.
Percentage of Alternative Responders Day 1 to Day 22 An alternative responder is a study participant with an increase in platelets to ≥ 30x10\^9/L and to at least double the baseline platelet count, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.
Percentage of Complete Responders Day 1 to Day 22 A complete responder is a study participant with an increase in platelets to ≥ 100x10\^9/L, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.
Percentage of Alternative Responders Who Lost the Response Day 1 to Day 22 An alternative responder who lost the response is a study participant who met the criterion for an alternative response but who then deteriorated, ie, their platelet count decreased to \< 30x10\^9/L, their platelet count decreased to less than double the baseline count, or bleeding occurred.
Percentage of Complete Responders Who Lost the Response Day 1 to Day 22 A complete responder who lost the response is a study participant who met the criterion for a complete response but who then deteriorated, ie, their platelet count decreased to \< 100x10\^9/L or bleeding occurred.
Platelet Count by Visit Day 1 to Day 22 The platelet count at each study visit are presented.
Time to a Response Day 1 to Day 8 A study participant had a response if their platelets increased to ≥ 50x10\^9/L within 7 days after the first infusion, ie, by study Day 8.
Time to an Alternative Response Day 1 to Day 22 A study participant had a response if their platelets increased to ≥ 30x10\^9/L and to at least double the baseline platelet count, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.
Time to a Complete Response Day 1 to Day 22 A study participant had a complete response if their platelets increased to ≥ 100x10\^9/L, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.
Duration of a Response Day 1 to Day 22 The duration of a response was defined as the time from when a response was achieved until the platelet count fell below 50x10\^9/L.
Duration of an Alternative Response Day 1 to Day 22 The duration of an alternative response was defined as the time from when an alternative response was achieved until the platelet count fell below 50x10\^9/L.
Maximum Platelet Count Day 1 to Day 22 The maximum platelet count achieved during the study is presented.
Percentage of Responders Who Achieved a Normal Platelet Count Day 1 to Day 22 The percentage of responders who achieved a normal platelet count is presented.
Bleeding Intensity Day 1 to Day 22 The percentage of participants with various intensities of overall bleeding, epistaxis (bleeding of the nose), oral bleeding, and skin bleeding graded as none, minor, mild, moderate, or severe at Baseline and Day 22 are reported.
Percentage of Participants Who Achieved a Platelet Count > 30x10^9/L Day 1 to Day 2 The percentage of participants who achieved a platelet count \> 30x10\^9/L within 1 and 2 days after infusion is reported.
Trial Locations
- Locations (1)
Abdulgabar Salama
🇩🇪Berlin, Germany