Trial record 14 of 638 for:    Alexander Disease

Therapy of Chronic Cold Agglutinin Disease With Eculizumab (DECADE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01303952
Recruitment Status : Completed
First Posted : February 25, 2011
Last Update Posted : July 19, 2017
Alexion Pharmaceuticals
Information provided by (Responsible Party):
Alexander Roeth, MD, 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)

Condition or disease Intervention/treatment Phase
Cold Agglutinin Disease Drug: Eculizumab Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Intervention Model: Single Group Assignment
Masking: None (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
Actual Study Start Date : January 2011
Primary Completion Date : September 2014
Study Completion Date : June 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Eculizumab
U.S. FDA Resources

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

Primary Outcome Measures :
  1. 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

Secondary Outcome Measures :
  1. Transfusion avoidance [ Time Frame: From baseline to 26 weeks ]
  2. PRBC units transfused [ Time Frame: From baseline to 26 weeks ]
  3. Change in hemoglobin levels [ Time Frame: From baseline to 26 weeks ]
  4. Haptoglobin [ Time Frame: From baseline to 26 weeks ]
  5. Hemopexin [ Time Frame: From baseline to 26 weeks ]
  6. Free hemoglobin [ Time Frame: From baseline to 26 weeks ]
  7. Reticulocytes [ Time Frame: From baseline to 26 weeks ]
  8. SF-36v2 QLQ [ Time Frame: From baseline to 26 weeks ]
  9. FACIT-F SCALE version 4 [ Time Frame: From baseline to 26 weeks ]
  10. Six-Minute Walk Test [ Time Frame: From baseline to 26 weeks ]
  11. Circulatory symptoms [ Time Frame: From baseline to 26 weeks ]
  12. Thrombosis record [ Time Frame: From baseline to 26 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01303952

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

Additional Information:
Responsible Party: Alexander Roeth, MD, MD, University Hospital, Essen Identifier: NCT01303952     History of Changes
Other Study ID Numbers: CAD01
2009-016966-97 ( EudraCT Number )
First Posted: February 25, 2011    Key Record Dates
Last Update Posted: July 19, 2017
Last Verified: July 2017

Keywords provided by Alexander Roeth, MD, University Hospital, Essen:
cold agglutinin disease
terminal complement inhibition

Additional relevant MeSH terms:
Hematologic Diseases
Autoimmune Diseases
Immune System Diseases
Anemia, Hemolytic, Autoimmune
Anemia, Hemolytic
Complement System Proteins
Cold agglutinins
Immunologic Factors
Physiological Effects of Drugs