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

December 28, 2016 updated by: Vaishali Sanchorawala

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Interventional

Enrollment (Actual)

82

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02118
        • Cancer Research Center at Boston Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
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 Names:
  • dexamethasone acetate
Drug: lenalidomide
15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone
Other Names:
  • revlimid; CC-5013

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Patients Removed From Study Treatment Due to Toxicities
Time Frame: 1 year
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.
3 months

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vaishali Sanchorawala

Collaborators

Celgene Corporation

Investigators

  • Principal Investigator: David C. Seldin, MD, PhD, Boston Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2004

Primary Completion (Actual)

July 1, 2012

Study Completion (Actual)

May 1, 2015

Study Registration Dates

First Submitted

September 7, 2004

First Submitted That Met QC Criteria

September 8, 2004

First Posted (Estimate)

September 9, 2004

Study Record Updates

Last Update Posted (Actual)

February 20, 2017

Last Update Submitted That Met QC Criteria

December 28, 2016

Last Verified

December 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CDR0000385687
  • BUMC-H-23235 (Other Identifier: Boston University Medical Center IRB)
  • CELGENE-RV-AMYL-PI-003 (Other Grant/Funding Number: Celgene)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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