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

This study has been completed.
Sponsor:
Collaborator:
Celgene Corporation
Information provided by (Responsible Party):
Vaishali Sanchorawala, Boston Medical Center
ClinicalTrials.gov Identifier:
NCT00091260
First received: September 7, 2004
Last updated: December 28, 2016
Last verified: December 2016
  Purpose

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.


Condition Intervention Phase
Multiple Myeloma Drug: dexamethasone Drug: lenalidomide Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Trial of the Immunomodulatory Drug CC-5013 for Patients With AL Amyloidosis

Resource links provided by NLM:


Further study details as provided by Vaishali Sanchorawala, Boston Medical Center:

Primary Outcome Measures:
  • 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.



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

Enrollment: 82
Study Start Date: January 2004
Study Completion Date: May 2015
Primary Completion Date: July 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 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

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.

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00091260

Locations
United States, Massachusetts
Cancer Research Center at Boston Medical Center
Boston, Massachusetts, United States, 02118
Sponsors and Collaborators
Vaishali Sanchorawala
Celgene Corporation
Investigators
Principal Investigator: David C. Seldin, MD, PhD Boston Medical Center
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Vaishali Sanchorawala, Principal Investigator, Boston Medical Center
ClinicalTrials.gov Identifier: NCT00091260     History of Changes
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 )
Study First Received: September 7, 2004
Results First Received: September 9, 2016
Last Updated: December 28, 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Vaishali Sanchorawala, Boston Medical Center:
primary systemic amyloidosis

Additional relevant MeSH terms:
Multiple Myeloma
Amyloidosis
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Neoplasms
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Proteostasis Deficiencies
Metabolic Diseases
Dexamethasone acetate
Dexamethasone
Lenalidomide
BB 1101
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists

ClinicalTrials.gov processed this record on September 19, 2017