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Therapy of Chronic Cold Agglutinin Disease With Eculizumab (DECADE)

Primary Purpose

Cold Agglutinin Disease

Status
Completed
Phase
Phase 2
Locations
Germany
Study Type
Interventional
Intervention
Eculizumab
Sponsored by
University Hospital, Essen
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cold Agglutinin Disease focused on measuring cold agglutinin disease, hemolysis, eculizumab, terminal complement inhibition

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Department of Hematology, University Hospital Essen

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Eculizumab

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Transfusion avoidance
PRBC units transfused
Change in hemoglobin levels
Haptoglobin
Hemopexin
Free hemoglobin
Reticulocytes
SF-36v2 QLQ
FACIT-F SCALE version 4
Six-Minute Walk Test
Circulatory symptoms
Thrombosis record

Full Information

First Posted
February 24, 2011
Last Updated
July 17, 2017
Sponsor
University Hospital, Essen
Collaborators
Alexion
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1. Study Identification

Unique Protocol Identification Number
NCT01303952
Brief Title
Therapy of Chronic Cold Agglutinin Disease With Eculizumab
Acronym
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2017
Overall Recruitment Status
Completed
Study Start Date
January 2011 (Actual)
Primary Completion Date
September 2014 (Actual)
Study Completion Date
June 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University Hospital, Essen
Collaborators
Alexion

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cold Agglutinin Disease
Keywords
cold agglutinin disease, hemolysis, eculizumab, terminal complement inhibition

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
13 (Actual)

8. Arms, Groups, and Interventions

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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Alexander Roeth, MD
Organizational Affiliation
Department of Hematology, University Hospital Essen
Official's Role
Principal Investigator
Facility Information:
Facility Name
Department of Hematology, University Hospital Essen
City
Essen
ZIP/Postal Code
45122
Country
Germany

12. IPD Sharing Statement

Citations:
PubMed Identifier
19372265
Citation
Roth A, Huttmann A, Rother RP, Duhrsen 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. No abstract available.
Results Reference
background
PubMed Identifier
19614954
Citation
Roth A, Duhrsen 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. No abstract available.
Results Reference
background
PubMed Identifier
30291112
Citation
Roth A, Bommer M, Huttmann A, Herich-Terhurne D, Kuklik N, Rekowski J, Lenz V, Schrezenmeier H, Duhrsen U. Eculizumab in cold agglutinin disease (DECADE): an open-label, prospective, bicentric, nonrandomized phase 2 trial. Blood Adv. 2018 Oct 9;2(19):2543-2549. doi: 10.1182/bloodadvances.2018024190.
Results Reference
derived

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Therapy of Chronic Cold Agglutinin Disease With Eculizumab

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