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Therapy of Chronic Cold Agglutinin Disease With Eculizumab (DECADE)

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Alexion Pharmaceuticals
Information provided by (Responsible Party):
Alexander Roeth, MD, University Hospital, Essen
ClinicalTrials.gov Identifier:
NCT01303952
First received: February 24, 2011
Last updated: May 25, 2014
Last verified: May 2014
  Purpose

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


Condition Intervention Phase
Cold Agglutinin Disease
Drug: Eculizumab
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by University Hospital, Essen:

Primary Outcome Measures:
  • LDH [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]
    Primary endpoint for this study is: lactate dehydrogenase (LDH) in U/l change from baseline to 26 weeks


Secondary Outcome Measures:
  • Transfusion avoidance [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]
  • PRBC units transfused [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]
  • Change in hemoglobin levels [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]
  • Haptoglobin [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]
  • Hemopexin [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]
  • Free hemoglobin [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]
  • Reticulocytes [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]
  • SF-36v2 QLQ [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]
  • FACIT-F SCALE version 4 [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]
  • Six-Minute Walk Test [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]
  • Circulatory symptoms [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]
  • Thrombosis record [ Time Frame: From baseline to 26 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 15
Study Start Date: February 2011
Estimated Study Completion Date: August 2014
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01303952

Locations
Germany
Department of Hematology, University Hospital Essen
Essen, Germany, 45122
Sponsors and Collaborators
University Hospital, Essen
Alexion Pharmaceuticals
Investigators
Principal Investigator: Alexander Roeth, MD Department of Hematology, University Hospital Essen
  More Information

Additional Information:
Publications:
Responsible Party: Alexander Roeth, MD, MD, University Hospital, Essen
ClinicalTrials.gov Identifier: NCT01303952     History of Changes
Other Study ID Numbers: CAD01, 2009-016966-97
Study First Received: February 24, 2011
Last Updated: May 25, 2014
Health Authority: Germany: Paul-Ehrlich-Institut

Keywords provided by University Hospital, Essen:
cold agglutinin disease
hemolysis
eculizumab
terminal complement inhibition

Additional relevant MeSH terms:
Autoimmune Diseases
Hematologic Diseases
Immune System Diseases
Anemia, Hemolytic, Autoimmune
Anemia
Anemia, Hemolytic
Agglutinins
Cold agglutinins
Hemagglutinins
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on November 24, 2014