Chronic immune thrombocytopenic purpura (ITP) MedDRA version: 15.0 Level: PT Classification code 10021245 Term: Idiopathic thrombocytopenic purpura System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Interventions
Trade Name: Privigen®
Product Name: Privigen®
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human Normal Immunoglobulin G (IgG > 98% purity)
Other descriptive name: HUMAN NORMAL IMMU
Sponsors
CSL Behring LLC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Diagnosis of chronic ITP defined by: ?- Failure to find other causes of thrombocytopenia, ?- Platelet count of = 150 x 109/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; • Age of 18 to 65 years; • Platelet count of = 30 x 109/L at screening; • Written informed consent for study participation obtained before undergoing any study specific procedures. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 150 F.1.3 Elderly (>=65 years) F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Planned splenectomy throughout the study period; • Treatment with IVIG or anti-D immunoglobulin within 3 weeks prior to screening; • Drugs that have any pharmacological effect on the blood clotting system within 3 weeks prior to screening (i.e., clotting factors, heparin, coumarin derivates, acetylsalicylic acid in high dose, and thrombopoietin receptor agonists [e.g., eltrombopag and romiplostim]); • Known allergic or other severe reactions to blood products including intolerability to previous IVIG (i.e. aseptic meningitis, recurrent severe headache, hypersensitivity, intravascular hemolysis); • Known hyperprolinemia; • Abnormal results in the following laboratory parameters at screening: ?- Hemoglobin the upper limit of the normal range (ULN), ?- Serum free haptoglobin ULN, ?- Alanine aminotransferase (ALAT) > 2.5 x ULN, -? Aspartate aminotransferase (ASAT) > 2.5 x ULN, ?- Creatinine > 1.5 x ULN, ?- Reticulocyte count > 1.5 x ULN, ?- Iron < 50% of LLN, ferritin < 50 LLN, vitamin B12 < 50% of LLN; • Pregnancy, lactation, or planned pregnancy; • RBC transfusion or erythropoietin treatment within the last 14 days; • One of the following concomitant diseases: -? Clinically active systemic lupus erythematosus, ?- Known or suspected HIV infection, ?- Acute hepatitis, -? Clinically active chronic hepatitis, -? Lymphoproliferative disease, ?- Sickle cell disease, ??- New York Heart Association (NYHA) grade III or IV heart failure; • Febrile illness or acute infection between screening and Day 1 (to be checked on Day 1 prior to dosing); • Body temperature = 38°C at screening or prior to treatment on Day 1 (to be checked on Day 1 prior to dosing); • History of Evan’s syndrome; • Participation in another clinical study during the last 3 months; • Alcohol, drug, or medication abuse within one year before the study; • Re-entry of subjects previously treated in the present study; • Re-screening within 4 weeks after the preceding screening; • Suspected inability (e.g., language problems) or unwillingness to comply with study procedures; • Mental condition rendering the subject (or the subject’s legally acceptable representative[s]) unable to understand the nature, scope and possible consequences of the study) ; • Any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct of the study; • Employee at the study site, or spouse/partner or relative of any study staff (e.g., investigator, sub-investigators, or study nurse).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to investigate the specificity of in vivo antibody binding to red blood cells (RBCs) in 10 subjects with chronic ITP who have shown signs of hemolysis and who experience clinically significant intravascular hemolytic reactions following treatment with IgPro10. The study will explore potential mechanisms of hemolysis by analysis of the specificity of the antibodies possibly involved.;Secondary Objective: The secondary objectives are to assess other safety and efficacy parameters, including the rates of presumptive hemolytic events and clinically significant intravascular hemolytic reactions.;Primary end point(s): The primary endpoint is the set of antibodies most frequently bound to RBCs in subjects experiencing clinically significant intravascular hemolysis. The presumptive hemolysis cases will be adjudicated by an independent Adjudication Committee to determine if the hemolysis represents clinically significant intravascular hemolysis. Most frequent antibodies are defined as those antibodies that are identified at day 3 as binding specific red cell antigens (determined using an antigen panel) in at least 4 subjects (out of 10 with clinically significant hemolysis).;Timepoint(s) of evaluation of this end point: Continously | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Subjects with a platelet response (PR) defined as subjects having a platelet count increases at least once to = 50 x 109/L within 6 days after the first infusion (i.e., up to and including Day 7).;Timepoint(s) of evaluation of this end point: 6 days after the first infusion | — |
Countries
Bulgaria, Poland, Serbia, Turkey
Contacts
Public Contact(Babette von Hagen) Senior CRA
CSL Behring GmbH
Outcome results
None listed