Therapy of Chronic Cold Agglutinin Disease With Eculizumab

• Status: Completed
• Trial end date: 2015-06-01
• Target enrollment: 0
• Participant gender: All

Summary

Evaluation of the efficacy and safety of eculizumab in symptomatic or transfusion-dependent patients with untreated or refractory hemolytic cold agglutinin disease(CAD)

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University Hospital, Essen
• Collaborator: Alexion Pharmaceuticals
• Treatments: Agglutinins; Cold agglutinins; Complement System Proteins; Eculizumab

Criteria

Inclusion Criteria:

• Individuals at least 18 years of age

• 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

• LDH level > 2 x upper limit of normal (ULN)

• Clinical symptoms requiring treatment, such as anemia, anemia-related symptoms or hemolysis-related symptoms

• Sufficient supplementation of iron, folic acid and vitamin B12 prior to treatment phase

• Patient must be willing and able to give written informed consent;

• Patients must be vaccinated against N. meningitidis at least 14 days prior treatment

• Patient must avoid conception during the trial using a method that is most appropriate for their physical state and culture

Exclusion Criteria:

• Active aggressive lymphoma requiring therapy or an active non-lymphatic malignant disease other than basal cell carcinoma or CIS of the cervix

• Liver disease with elevated LDH

• Absolute neutrophil count < 500/µL

• Presence or suspicion of active bacterial or viral infection, in the opinion of the Investigator, at treatment start or severe recurrent bacterial infections

• Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to screening period

• Pregnant, breast-feeding, or intending to conceive during the course of the study, including the Post-treatment Phase

• History of bone marrow/stem cell transplantation

• Inability to cooperate or any condition that, in the opinion of the investigator, could increase the patient's risk by participating in the study or confound the outcome of the study

• Hypersensitivity to eculizumab, to murine proteins or to one of the excipients