Clinical Trial: CC-5013 With or Without Dexamethasone in Treating Patients With Primary Systemic Amyloidosis

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

Official Title: A Phase II Trial of the Immunomodulatory Drug CC-5013 for Patients With AL Amyloidosis

Brief Summary:

RATIONALE: Drugs such as CC-5013 and dexamethasone may be effective in treating primary systemic amyloidosis.

PURPOSE: This phase II trial is studying CC-5013 to see how well it works with or without dexamethasone in treating patients with primary systemic amyloidosis.


Detailed Summary:

OBJECTIVES:

Primary

  • Determine the tolerability of CC-5013 in patients with primary systemic (AL) amyloidosis.
  • Determine the objective hematologic response rate in patients treated with this drug.
  • Determine amyloid organ disease response in patients treated with this drug.

Secondary

  • Determine hematologic and amyloid organ disease response in patients who do not achieve a response to CC-5013 alone and are subsequently treated with CC-5013 and dexamethasone.
  • Determine the toxicity of CC-5013 in combination with dexamethasone in these patients.

OUTLINE: Patients receive oral CC-5013 once daily on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients not achieving a hematologic response continue to receive CC-5013 as before and also receive oral dexamethasone twice daily on days 1-4, 9-12, and 17-20 of every other 28-day course for up to 6 courses of combination therapy. Patients who maintain a hematologic response after 6 courses of combination therapy may receive CC-5013 alone in the absence of disease progression or unacceptable toxicity. Patients not achieving a hematologic response after the initiation of dexamethasone are removed from the study.

Patients are followed annually.

PROJECTED ACCRUAL: A total of 15-25 patients will be accrued for this study within 5-12.5 months.


Sponsor: Vaishali Sanchorawala

Current Primary Outcome:

  • Number of Patients Removed From Study Treatment Due to Toxicities [ Time Frame: 1 year ]
  • Number of Patients With Hematologic Response With Single-agent CC-5013 [ Time Frame: 3 months ]

    Complete response = Absence of detectable monoclonal protein in serum or urine by immunofixation electrophoresis, less than 5% plasma cells on bone marrow biopsy without clonal dominance of kappa or lambda isotype, and normal serum free light chain assay.

    Partial response= For patients with detectable and quantifiable monoclonal marrow plasmacytosis= a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells. For patients with a detectable monoclonal peak on serum or urine protein electrophoresis= a reduction in the peak height of 50% or more.

    For patients with quantifiable urinary kappa or lambda chain concentration= a 50% reduction in daily light chain excretion in 24 hour urine.

    For patients with an elevated serum free light chain assay, a reduction of 50% or more.



Original Primary Outcome:

Current Secondary Outcome: Number of Patients Who Received Both CC-5013 and Dexamethasone and Had a Hematologic Response [ Time Frame: 1 year ]

Original Secondary Outcome:

Information By: Boston Medical Center

Dates:
Date Received: September 7, 2004
Date Started: January 2004
Date Completion:
Last Updated: December 28, 2016
Last Verified: December 2016