Therapy of Chronic Cold Agglutinin Disease With Eculizumab (DECADE)

July 17, 2017 updated by: Alexander Roeth, MD, University Hospital, Essen

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

13

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Essen, Germany, 45122
        • Department of Hematology, University Hospital Essen

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Eculizumab
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

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Essen

Collaborators

Alexion Pharmaceuticals

Investigators

  • Principal Investigator: Alexander Roeth, MD, Department of Hematology, University Hospital Essen

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2011

Primary Completion (Actual)

September 1, 2014

Study Completion (Actual)

June 1, 2015

Study Registration Dates

First Submitted

February 24, 2011

First Submitted That Met QC Criteria

February 24, 2011

First Posted (Estimate)

February 25, 2011

Study Record Updates

Last Update Posted (Actual)

July 19, 2017

Last Update Submitted That Met QC Criteria

July 17, 2017

Last Verified

July 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CAD01
  • 2009-016966-97 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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