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

Multiple Myeloma Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00091260
• Other study ID #: CDR0000385687
• Secondary ID: BUMC-H-23235CELG
• Status: Completed
• Phase: Phase 2
• First received: September 7, 2004
• Last updated: January 14, 2016
• Start date: January 2004
• Est. completion date: May 2015

Study information

• Verified date: January 2016
• Source: Boston Medical Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal GovernmentUnited States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 82
• Est. completion date: May 2015
• Est. primary completion date: July 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

• 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

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 Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyloidosis
• Multiple Myeloma

Intervention

Drug:
• dexamethasone
dexamethasone 20 mg daily (10 mg BID) on Days 1-4, 9-12, and 17-20 of every other 28-day cycle.
• lenalidomide
15 mg/day, for 21 days with 7 days rest (28 day cycle) with or without dexamethasone

Locations

Country	Name	City	State
United States	Cancer Research Center at Boston Medical Center	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Vaishali Sanchorawala	Celgene Corporation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Tolerability and objective hematologic response with CC-5013 every 3 months		3 months	Yes
Secondary	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		3 months	No
Secondary	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		3 months	No
Secondary	Toxicity of CC-5013 and dexamethasone every 3 months after dexamethasone is added		3 months	Yes
Secondary	Organ response with CC-5013 every 3 months		3 months	No