Therapy of Chronic Cold Agglutinin Disease With Eculizumab

NCT ID: NCT01303952

Last Updated: 2017-07-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 13 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-01-31
• Study Completion Date: 2015-06-30

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)

Conditions

Cold Agglutinin Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Eculizumab

Group Type: EXPERIMENTAL

Eculizumab

Intervention Type: DRUG

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

Interventions

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

Intervention Type: DRUG

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: collaborator

University Hospital, Essen

OTHER

Sponsor Role: lead

Responsible Party

Alexander Roeth, MD

MD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Alexander Roeth, MD

Role: PRINCIPAL_INVESTIGATOR

Department of Hematology, University Hospital Essen

Locations

Department of Hematology, University Hospital Essen

Essen, , Germany

Countries

Germany

References

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.

Reference Type: BACKGROUND

PMID: 19372265 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 19614954 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 30291112 (View on PubMed)

Other Identifiers

2009-016966-97

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CAD01

Identifier Type: -

Identifier Source: org_study_id