Clinical trial NCT00091260: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Multiple Myeloma
Keywords: primary systemic amyloidosis
Lead sponsor: Boston Medical Center
Sponsor class: OTHER
Collaborators: name: Celgene Corporation
First posted: 2004-09-09
Enrollment: 82
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Number of Patients Removed From Study Treatment Due to Toxicities
timeFrame: 1 year
measure: Number of Patients With Hematologic Response With Single-agent CC-5013
timeFrame: 3 months
Secondary outcomes:
measure: Number of Patients Who Received Both CC-5013 and Dexamethasone and Had a Hematologic Response
timeFrame: 1 year
Other outcomes:
Locations:
city: Boston
country: United States
facility: Cancer Research Center at Boston Medical Center
state: Massachusetts
Study officials:
affiliation: Boston Medical Center
name: David C. Seldin, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
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.