Clinical Trial: Open-Label Phase 1/2 Study of VELCADE for Injection in Patients With Light-chain (AL)-Amyloidosis

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An Open-Label Phase 1/2 Study of VELCADE for Injection in Subjects With Light-Chain (AL)-Amyloidosis

Brief Summary: This is a phase 1/2 open-label, dose-escalation study investigating single-agent therapy with VELCADE in patients with previously treated systemic AL-amyloidosis who require further treatment.

Detailed Summary:
Sponsor: Millennium Pharmaceuticals, Inc.

Current Primary Outcome:

• Maximum Tolerated Dose [ Time Frame: 5 weeks in once weekly (QW) dose cohorts and 3 weeks in twice weekly (BIW) dose cohorts ]

  Maximum Tolerated Dose (MTD) was defined as the highest dose level that has 0/1 out of 6 patients experiences Dose Limited Toxicity (DLT). MTD is defined separately for QW and BIQ dose cohorts.

  DLT was defined as adverse events occurring during Cycle 1 and: (1) related to VELCADE, (2) Grade 4 thrombocytopenia or neutropenia, (3) Grade 3 or higher nonhematologic toxicity.

• Subjects With Treatment Emergent Adverse Events [ Time Frame: from first study-related procedure to 30 days after last dose of study medication ]
  Treatment emergent adverse events observed during outcome measure time frame
• Subjects With Serious Treatment Emergent Adverse Events [ Time Frame: from first study-related procedure to 30 days after last dose of study medication ]
  Serious treatment emergent adverse events observed during outcome measure time frame
• Subjects Grade 3/4/5 Treatment Emergent Adverse Events [ Time Frame: from first study-related procedure to 30 days after last dose of study medication ]

  Grade 3/4/5 treatment emergent adverse events observed during outcome measure time frame.

  Grade is determined according to Common Terminology Criteria for Adverse Event (CTCAE) Version 3.0.

• Subj
  
  

  Original Primary Outcome:
  
  

  Current Secondary Outcome: Best Confirmed Hematologic Responders [ Time Frame: from first dose of study medication to end of study visit ]

  Hematologic response was determined by the investigator per the response criteria for immunoglobulin light chain amyloidosis by Gertz (2005). It include Complete and Partial Responders (CR+PR). CR requires serum and urine negative for a monoclonal protein by immunofixation and free light chain ratio normal. PR requires: 1. reduction in quantitative serum M-protein by 50% if baseline value is at least 0.5 g/dL, 2. if light chain is detected in the urine (with a consistent peak and >100 mg/ 24 hours), then 50% reduction is required, 3. if free light chain >10 mg/dL, reduction by 50% is required.
  
  
  

  Original Secondary Outcome:
  
  Information By: Millennium Pharmaceuticals, Inc.
  

  Dates:
  Date Received: March 1, 2006
  Date Started: June 2005
  Date Completion:
  Last Updated: June 19, 2012
  Last Verified: June 2012