Therapy of Chronic Cold Agglutinin Disease With Eculizumab (DECADE)

University Hospital Essen

Status and phase

Completed

Phase 2

Conditions

Cold Agglutinin Disease

Treatments

Drug: Eculizumab

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT01303952

CAD01

2009-016966-97 (EudraCT Number)

Details and patient eligibility

About

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

Enrollment

13 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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