Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Celgene Corporation
Information provided by (Responsible Party):
David Seldin, MD, Boston Medical Center
ClinicalTrials.gov Identifier:
NCT00091260
First received: September 7, 2004
Last updated: July 21, 2014
Last verified: July 2014
  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 and Plasma Cell Neoplasm
Drug: dexamethasone
Drug: lenalidomide
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 Boston Medical Center:

Primary Outcome Measures:
  • Tolerability and objective hematologic response with CC-5013 every 3 months [ Time Frame: 3 months ] [ Designated as safety issue: Yes ]
  • Organ response with CC-5013 every 3 months [ Time Frame: 3 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Hematologic response in patients who do not achieve a response to CC-5013 alone and are subsequently treated with CC-5013 and dexamethasone every 3 months after dexamethasone is added [ Time Frame: 3 months ] [ Designated as safety issue: No ]
  • Organ response in patients who do not achieve a response to CC-5013 alone and are subsequently treated with CC-5013 and dexamethasone every 3 months after dexamethasone is added [ Time Frame: 3 months ] [ Designated as safety issue: No ]
  • Toxicity of CC-5013 and dexamethasone every 3 months after dexamethasone is added [ Time Frame: 3 months ] [ Designated as safety issue: Yes ]

Enrollment: 82
Study Start Date: January 2004
Estimated Study Completion Date: April 2017
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 Drug: lenalidomide

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
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  • No secondary or familial amyloidosis
  • No multiple myeloma, defined as ≥ 30% plasma cells in bone marrow biopsy specimen OR lytic bone lesions

PATIENT CHARACTERISTICS:

Age

  • 18 and over

Performance status

  • SWOG 0-2

Life expectancy

  • Not specified

Hematopoietic

  • WBC > 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)
  • AST and ALT ≤ 2 times ULN

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

PRIOR CONCURRENT THERAPY:

Biologic therapy

  • No prior CC-5013
  • 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
  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
David Seldin, MD
Celgene Corporation
Investigators
Principal Investigator: David C. Seldin, MD, PhD Boston Medical Center
  More Information

Additional Information:
No publications provided by Boston Medical Center

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: David Seldin, MD, Principal Investigator, Boston Medical Center
ClinicalTrials.gov Identifier: NCT00091260     History of Changes
Other Study ID Numbers: CDR0000385687, BUMC-2004-009, BUMC-H-23235, CELGENE-RV-AMYL-PI-003
Study First Received: September 7, 2004
Last Updated: July 21, 2014
Health Authority: United States: Federal Government
United States: Food and Drug Administration

Keywords provided by Boston Medical Center:
primary systemic amyloidosis

Additional relevant MeSH terms:
Amyloidosis
Multiple Myeloma
Neoplasms, Plasma Cell
Plasmacytoma
Blood Protein Disorders
Cardiovascular Diseases
Hematologic Diseases
Hemorrhagic Disorders
Hemostatic Disorders
Immune System Diseases
Immunoproliferative Disorders
Lymphoproliferative Disorders
Metabolic Diseases
Neoplasms
Neoplasms by Histologic Type
Paraproteinemias
Proteostasis Deficiencies
Vascular Diseases
BB 1101
Dexamethasone
Dexamethasone 21-phosphate
Dexamethasone acetate
Lenalidomide
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Anti-Inflammatory Agents
Antiemetics
Antineoplastic Agents
Antineoplastic Agents, Hormonal
Autonomic Agents

ClinicalTrials.gov processed this record on November 20, 2014