Therapy of Chronic Cold Agglutinin Disease With Eculizumab

Phase 2

Completed

• Conditions: Cold Agglutinin Disease
• Interventions: Drug: Eculizumab

• Registration Number: NCT01303952
• Lead Sponsor: University Hospital, Essen

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2011-01
• Study First Submitted: 2011-02-24
• Study First Submitted QC: 2011-02-24
• Study First Posted: 2011-02-25
• Primary Completion: 2014-09
• Study Completion: 2015-06
• Status Verified: 2017-07
• Last Update Submitted: 2017-07-17
• Last Update Posted: 2017-07-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 13

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Eculizumab	Eculizumab	-

Primary Outcome Measures

Name	Time	Method
LDH	From baseline to 26 weeks	Primary endpoint for this study is: lactate dehydrogenase (LDH) in U/l change from baseline to 26 weeks

Secondary Outcome Measures

Name	Time	Method
Hemopexin	From baseline to 26 weeks
Transfusion avoidance	From baseline to 26 weeks
SF-36v2 QLQ	From baseline to 26 weeks
Circulatory symptoms	From baseline to 26 weeks
Free hemoglobin	From baseline to 26 weeks
Reticulocytes	From baseline to 26 weeks
PRBC units transfused	From baseline to 26 weeks
Haptoglobin	From baseline to 26 weeks
Change in hemoglobin levels	From baseline to 26 weeks
Six-Minute Walk Test	From baseline to 26 weeks
FACIT-F SCALE version 4	From baseline to 26 weeks
Thrombosis record	From baseline to 26 weeks

Trial Locations

Locations (1)

Department of Hematology, University Hospital Essen; 🇩🇪 Essen, Germany