AutoImmune Hemolytic Anemia MedDRA version: 20.0 Level: LLT Classification code 10003825 Term: Autoimmune hemolytic anemia System Organ Class: 100000004851
Conditions
Interventions
Trade Name: CINRYZE® (C1 esterase inhibitor [human])
Pharmaceutical Form: Concentrate and solvent for solution for infusion
Sponsors
Academic Medical Center
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Positive (=1+) monospecific antiglobulin test for C3b and/or C3d with/without positivity for IgM or IgG • Indication for a transfusion with at least 2 red packed cell concentrates based on the clinical assessment by the hematologist in charge • Hemoglobin value at least =65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: • History of arterial and/or venous thromboembolic events in the absence of an actual treatment with Vitamin K-antagonists • Concomitant use of therapeutic doses of heparin • Female patients who are pregnant or breast feeding or adults of reproductive potential who are not using effective birth control methods. If barrier contraceptives are being used, these must be continued throughout the trial by both sexes. Oral contraceptives only are not acceptable. • Patients with known HIV seropositivity or chronic active hepatitis • Patients who have any severe and/or uncontrolled medical condition or other conditions that could affect their participation in the study such as: – cerebrovascular accidents = 6 months before study drug start – uncontrolled hypertension
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): not applicable;Timepoint(s) of evaluation of this end point: not applicable | — |
Primary
| Measure | Time frame |
|---|---|
| Main Objective: to test whether co-administration of C1-inh concentrate in patients suffering from AIHA needing RBC transfusion • improves recovery of RBC transfusion • inhibits complement activation and deposition on RBC via the classical pathway of complement • is safe? ;Secondary Objective: to investigate whether co-administration of C1-inhibitor concentrate in patients suffering from AIHA needing RBC transfusion • attenuates the pro-inflammatory response in AIHA • affects the response to the basic treatment targeting autoantibody production ;Primary end point(s): • improves recovery of RBC transfusion • inhibits complement activation and deposition on RBC via the classical pathway of complement ;Timepoint(s) of evaluation of this end point: • At entry: laboratory tests within 4 hours before start of treatment, before and after every dose C1-inh as well as 48 hours, 60 hours and 72 hours after the first dose NB: in case that the time between study entry and start treatment exceeds 4 hours, laboratory tests performed at study entry must be repeated | — |
Countries
Netherlands
Contacts
Public ContactM. Spiering
Academic Medical Center
Outcome results
None listed