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

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Primary Purpose

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

dexamethasone

lenalidomide

About this trial

This is an interventional treatment trial for Multiple Myeloma focused on measuring primary systemic amyloidosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Cancer Research Center at Boston Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

revlimid

Arm Description

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.

Outcomes

Primary Outcome Measures

Number of Patients Removed From Study Treatment Due to Toxicities

Number of Patients With Hematologic Response With Single-agent CC-5013

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

Full Information

NCT ID

NCT00091260

First Posted

September 7, 2004

Last Updated

December 28, 2016

Sponsor

Vaishali Sanchorawala

Collaborators

Celgene Corporation

1. Study Identification

Unique Protocol Identification Number

NCT00091260

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

December 2016

Overall Recruitment Status

Completed

Study Start Date

January 2004 (undefined)

Primary Completion Date

July 2012 (Actual)

Study Completion Date

May 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Vaishali Sanchorawala

Collaborators

Celgene Corporation

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Multiple Myeloma

Keywords

primary systemic amyloidosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

82 (Actual)

8. Arms, Groups, and Interventions

Arm Title

revlimid

Arm Type

Experimental

Arm Description

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.

Intervention Type

Drug

Intervention Name(s)

dexamethasone

Other Intervention Name(s)

dexamethasone acetate

Intervention Description

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

Intervention Name(s)

lenalidomide

Other Intervention Name(s)

revlimid; CC-5013

Intervention Description

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

Primary Outcome Measure Information:

Title

Number of Patients Removed From Study Treatment Due to Toxicities

Time Frame

1 year

Title

Number of Patients With Hematologic Response With Single-agent CC-5013

Description

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.

Time Frame

3 months

Secondary Outcome Measure Information:

Title

Number of Patients Who Received Both CC-5013 and Dexamethasone and Had a Hematologic Response

Time Frame

1 year

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

No

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

David C. Seldin, MD, PhD

Organizational Affiliation

Boston Medical Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

Cancer Research Center at Boston Medical Center

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02118

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

No

Citations:

PubMed Identifier

24668858

Citation

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.

Results Reference

derived

Multiple Myeloma

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial