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

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.
 
ClinicalTrials.gov Identifier: NCT00091260
Recruitment Status : Completed
First Posted : September 9, 2004
Results First Posted : February 20, 2017
Last Update Posted : February 20, 2017
Sponsor:
Collaborator:
Celgene Corporation
Information provided by (Responsible Party):
Vaishali Sanchorawala, Boston Medical Center

Tracking Information
First Submitted Date  ICMJE September 7, 2004
First Posted Date  ICMJE September 9, 2004
Results First Submitted Date  ICMJE September 9, 2016
Results First Posted Date  ICMJE February 20, 2017
Last Update Posted Date February 20, 2017
Study Start Date  ICMJE January 2004
Actual Primary Completion Date July 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 28, 2016)
  • 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 Measures  ICMJE Not Provided
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 28, 2016)
Number of Patients Who Received Both CC-5013 and Dexamethasone and Had a Hematologic Response [ Time Frame: 1 year ]
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE CC-5013 With or Without Dexamethasone in Treating Patients With Primary Systemic Amyloidosis
Official Title  ICMJE 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 Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Multiple Myeloma
Intervention  ICMJE
  • Drug: dexamethasone
    dexamethasone 20 mg daily (10 mg BID) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
    Other Name: dexamethasone acetate
  • Drug: lenalidomide
    15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone
    Other Name: revlimid; CC-5013
Study Arms  ICMJE Experimental: revlimid
lenalidomide 15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone 20 mg daily (10 mg BID) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
Interventions:
  • Drug: dexamethasone
  • Drug: lenalidomide
Publications * Lichtman EI, Seldin DC, Shelton A, Sanchorawala V. Single agent lenalidomide three times a week induces hematologic responses in AL amyloidosis patients on dialysis. Am J Hematol. 2014 Jul;89(7):706-8. doi: 10.1002/ajh.23722. Epub 2014 Apr 10.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 19, 2013)
82
Original Enrollment  ICMJE Not Provided
Actual Study Completion Date  ICMJE May 2015
Actual Primary Completion Date July 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

DISEASE CHARACTERISTICS:

  • Histologically confirmed primary systemic (AL) amyloidosis

    • Tissue amyloid deposits or positive fat aspirate
  • Meets 1 of the following criteria for AL type disease:

    • Serum or urine monoclonal protein by immunofixation electrophoresis
    • Plasmacytosis of bone marrow by monoclonal staining for kappa- or lambda-light chain isotype

PATIENT CHARACTERISTICS:

Age

  • 18 and over

Performance status

  • SWOG 0-2

Life expectancy

  • Not specified

Hematopoietic

  • White blood count> 3,000/mm^3
  • Hemoglobin > 8 g/dL
  • Platelet count > 100,000/mm^3
  • Absolute neutrophil count > 1,000/mm^3

Hepatic

  • Bilirubin ≤ 2 times upper limit of normal (ULN)
  • aspartate aminotransferase (AST) and Alanine Aminotransferase (ALT) ≤ 2 times ULN

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • Prior thalidomide for AL amyloidosis allowed

Chemotherapy

  • More than 4 weeks since prior cytotoxic chemotherapy

Endocrine therapy

  • Prior steroids for AL amyloidosis allowed

Radiotherapy

  • More than 4 weeks since prior radiotherapy

Surgery

  • Prior surgery allowed

Other

  • Recovered from all prior therapy

Exclusion Criteria:

  • No secondary or familial amyloidosis
  • No multiple myeloma, defined as ≥ 30% plasma cells in bone marrow biopsy specimen OR lytic bone lesions
  • No prior CC-5013

Renal

  • No dialysis

Cardiovascular

  • No symptomatic cardiac arrhythmia
  • No oxygen-dependent restrictive cardiomyopathy

Other

  • No untreated or uncontrolled infection
  • No other malignancy except basal cell skin cancer or carcinoma in situ of the cervix or breast
  • No other serious medical illness that would preclude study participation
  • No history of hypersensitivity reaction to thalidomide
  • HIV negative
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00091260
Other Study ID Numbers  ICMJE CDR0000385687
BUMC-H-23235 ( Other Identifier: Boston University Medical Center IRB )
CELGENE-RV-AMYL-PI-003 ( Other Grant/Funding Number: Celgene )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Vaishali Sanchorawala, Boston Medical Center
Study Sponsor  ICMJE Vaishali Sanchorawala
Collaborators  ICMJE Celgene Corporation
Investigators  ICMJE
Principal Investigator: David C. Seldin, MD, PhD Boston Medical Center
PRS Account Boston Medical Center
Verification Date December 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP