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

NCT ID: NCT00091260

Last Updated: 2017-02-20

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 82 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2004-01-31
• Study Completion Date: 2015-05-31

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.

Conditions

Multiple Myeloma

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

dexamethasone

Intervention Type: DRUG

dexamethasone 20 mg daily (10 mg BID) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.

lenalidomide

Intervention Type: DRUG

15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone

Interventions

dexamethasone

dexamethasone 20 mg daily (10 mg BID) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.

Intervention Type: DRUG

lenalidomide

15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone

Intervention Type: DRUG

Other Intervention Names

dexamethasone acetate; revlimid; CC-5013

Eligibility 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Celgene Corporation

INDUSTRY

Sponsor Role: collaborator

Vaishali Sanchorawala

OTHER

Sponsor Role: lead

Responsible Party

Vaishali Sanchorawala

Principal Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

David C. Seldin, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Boston Medical Center

Locations

Cancer Research Center at Boston Medical Center

Boston, Massachusetts, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 24668858 (View on PubMed)

Other Identifiers

BUMC-H-23235

Identifier Type: OTHER

Identifier Source: secondary_id

CELGENE-RV-AMYL-PI-003

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

CDR0000385687

Identifier Type: -

Identifier Source: org_study_id