Therapy of Chronic Cold Agglutinin Disease With Eculizumab

Cold Agglutinin Disease Clinical Trial

— DECADE  

Official title:

Therapy for Chronic Cold Agglutinin Disease: A Prospective, Non-Randomized Multi-Center Study Demonstrating the Efficacy of Terminal Complement Inhibition in Patients With Cold Agglutinin Disease Using Eculizumab

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01303952
• Other study ID #: CAD01
• Secondary ID: 2009-016966-97
• Status: Completed
• Phase: Phase 2
• First received: February 24, 2011
• Last updated: July 17, 2017
• Start date: January 2011
• Est. completion date: June 2015

Study information

• Verified date: July 2017
• Source: University Hospital, Essen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 13
• Est. completion date: June 2015
• Est. primary completion date: September 2014
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Anemia, Hemolytic, Autoimmune
• Cold Agglutinin Disease

Intervention

Drug:
• Eculizumab
Weekly treatment with eculizumab (600 mg) via IV infusion for 4 doses followed the next week by eculizumab (900 mg) IV infusion and maintenance treatment with Eculizumab (900 mg) via IV infusion every other week for 21 weeks

Locations

Country	Name	City	State
Germany	Department of Hematology, University Hospital Essen	Essen

Sponsors (2)

Lead Sponsor	Collaborator
University Hospital, Essen	Alexion Pharmaceuticals

Country where clinical trial is conducted

Germany, 

References & Publications (2)

Röth A, Dührsen U. Cold agglutinin disease. Eur J Haematol. 2010 Jan 1;84(1):91. doi: 10.1111/j.1600-0609.2009.01320.x. Epub 2009 Jul 14. — View Citation

Röth A, Hüttmann A, Rother RP, Dührsen U, Philipp T. Long-term efficacy of the complement inhibitor eculizumab in cold agglutinin disease. Blood. 2009 Apr 16;113(16):3885-6. doi: 10.1182/blood-2009-01-196329. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	LDH	Primary endpoint for this study is: lactate dehydrogenase (LDH) in U/l change from baseline to 26 weeks	From baseline to 26 weeks
Secondary	Transfusion avoidance		From baseline to 26 weeks
Secondary	PRBC units transfused		From baseline to 26 weeks
Secondary	Change in hemoglobin levels		From baseline to 26 weeks
Secondary	Haptoglobin		From baseline to 26 weeks
Secondary	Hemopexin		From baseline to 26 weeks
Secondary	Free hemoglobin		From baseline to 26 weeks
Secondary	Reticulocytes		From baseline to 26 weeks
Secondary	SF-36v2 QLQ		From baseline to 26 weeks
Secondary	FACIT-F SCALE version 4		From baseline to 26 weeks
Secondary	Six-Minute Walk Test		From baseline to 26 weeks
Secondary	Circulatory symptoms		From baseline to 26 weeks
Secondary	Thrombosis record		From baseline to 26 weeks

External Links

•   DECADE Trial Flyer