Clinical Trial: Short Course of Bortezomib in Anemic Patients With Refractory Cold Agglutinin Disease

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Short Course of Bortezomib in Anemic Patients With Refractory Cold Agglutinin Disease

Brief Summary: This multicenter, phase II, open label study will enroll patients with chronic cold agglutinin disorder. A single course of Bortezomib will be given at the dose of 1,3 mg/sqm iv on days 1, 4, 8, 11.

Detailed Summary: Based on its activity in MM, single agent Bortezomib was tested in phase II trials in lymphoplasmacytic lymphoma, the disorder most frequently associated with CAD, and achieved 40-80% responses. These striking clinical responses indicate that proteasome activity is critical for the survival of immunoglobulin-secreting cells. The resolution of transfusion requirement in two patients with refractory CAD associated with IgMk monoclonal gammopathy has been reported after treatment with a short course of Bortezomib. It may be interesting to test the efficacy of Bortezomib in a larger series of patients with refractory CAD, idiopathic or associated with an otherwise asymptomatic B cell clonal disorder, and to evaluate the duration of clinical benefit.
Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

Current Primary Outcome:

  • Number of patients who become transfusion-free after Bortezomib therapy. [ Time Frame: After 3 months from study entry. ]
    Cumulative proportion of patients transfusion-free at three months after Bortezomib therapy.
  • Number of patients who have never been transfused with a >2g hemoglobin rise compared to baseline. [ Time Frame: After 3 months from study entry. ]


Original Primary Outcome:

  • Number of patients who become transfusion-free after Bortezomib therapy. [ Time Frame: After 30 months from study entry. ]
    Cumulative proportion of patients transfusion-free at three months after Bortezomib therapy.
  • Number of patients who have never been transfused with a >2g hemoglobin rise compared to baseline. [ Time Frame: After 30 months from study entry. ]


Current Secondary Outcome:

  • Number of CTC grade 3 and 4 adverse events. [ Time Frame: After 12 months from study entry. ]
    Evaluation of the incidence of CTC grade 3 and 4 adverse events during and after treatment.
  • Duration in months of transfusion independence. [ Time Frame: At 12 months from study entry. ]
  • Effect of treatment on the underlying clonal B cell disorder. [ Time Frame: At 3 months from study entry. ]


Original Secondary Outcome:

  • Number of CTC grade 3 and 4 adverse events. [ Time Frame: After 30 months from study entry. ]
    Evaluation of the incidence of CTC grade 3 and 4 adverse events during and after treatment.
  • Duration in months of transfusion independance. [ Time Frame: At 30 months from study entry. ]
  • Effect of treatment on the underlying cloncal B cell disorder. [ Time Frame: At 30 months from study entry. ]


Information By: Gruppo Italiano Malattie EMatologiche dell'Adulto

Dates:
Date Received: September 27, 2012
Date Started: December 2012
Date Completion: December 2017
Last Updated: February 17, 2017
Last Verified: February 2017