Cold agglutinin disease MedDRA version: 12.1 Level: LLT Classification code 10009868 Term: Cold type haemolytic anaemia
Conditions
Interventions
Trade Name: Soliris (R)
Pharmaceutical Form: Concentrate for solution for infusion
Sponsors
Universitätsklinikum Essen
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Diagnosis of CAD defined by the combination of chronic hemolysis, cold agglutinin titer (IgM or IgA) >64 at 4°C, positive direct antiglobulin test (DAT) when performed with polyspecific antiserum, negative (or only weakly positive) anti-IgG, and strongly positive with anti-C3d. 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 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: An active aggressive lymphoma requiring therapy or an active non-lymphatic malignant disease other than basal cell carcinoma or CIS of the cervix.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluation of the efficacy and safety of eculizumab in symptomatic or transfusion-dependent patients with untreated or refractory hemolytic cold agglutinin disease (CAD);Secondary Objective: Effect of eculizumab in hemolytic cold agglutinin disease on lactate dehydrogenase (LDH) change, transfusion avoidance, PRBC units transfused, change in hemoglobin levels, haptoglobin, hemopexin, free hemoglobin, reticulocytes, SF-36V2 QLQ, FACIT-F SCALE version 4, Six-Minute Walk Test, circulatory symptoms and thrombosis record from baseline to 26 weeks. Exploratory endpoints include: direct agglutination test (DAT), serum monoclonal immunoglobulin level, cold agglutinin (IgM or IgA) titer and thermal amplitude measures from randomization to 26 weeks, presence of a clonal B-cell lymphoproliferative disorder defined by monoclonal IgM? or IgA? band by serum electrophoresis and immunofixation, immunophenotyping of bone marrow, immunophenotyping of peripheral blood and blood pressure. ;Primary end point(s): Primary endpoint for this study is lactate dehydrogenase (LDH) change from baseline to 26 weeks. | — |
Countries
Germany
Outcome results
None listed