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

Sponsored by:	Boston Medical Center
Information provided by:	National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:	NCT00091260

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 II

Genetics Home Reference related topics: aceruloplasminemia hemophilia

MedlinePlus related topics: Cancer Multiple Myeloma

Drug Information available for: Dexamethasone Dexamethasone acetate Dexamethasone Sodium Phosphate Doxiproct plus Lenalidomide CC 5013

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label
Official Title:	A Phase II Trial of the Immunomodulatory Drug CC-5013 for Patients With AL Amyloidosis

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:

Tolerability and objective hematologic response with CC-5013 every 3 months [ Designated as safety issue: Yes ]
Organ response with CC-5013 every 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 [ 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 [ Designated as safety issue: No ]
Toxicity of CC-5013 and dexamethasone every 3 months after dexamethasone is added [ Designated as safety issue: Yes ]

Study Start Date:

January 2004

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

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

Boston Medical Center

Investigators

Principal Investigator:

David C. Seldin, MD, PhD

Boston Medical Center

More Information

Clinical trial summary from the National Cancer Institute's PDQ® database This link exits the ClinicalTrials.gov site

Study ID Numbers:	CDR0000385687, BUMC-2004-009, BUMC-H-23235, CELGENE-RV-AMYL-PI-003
Study First Received:	September 7, 2004
Last Updated:	July 23, 2008
ClinicalTrials.gov Identifier:	NCT00091260
Health Authority:	United States: Federal Government

Keywords provided by National Cancer Institute (NCI):

primary systemic amyloidosis

Study placed in the following topic categories:

Dexamethasone
Metabolic Diseases
Immunoproliferative Disorders
Blood Protein Disorders
Hematologic Diseases
Primary Amyloidosis
Blood Coagulation Disorders
Lenalidomide
Vascular Diseases
Paraproteinemias

Hemostatic Disorders
Multiple Myeloma
Amyloidosis
Hemorrhagic Disorders
Multiple myeloma
Metabolic disorder
Lymphoproliferative Disorders
Dexamethasone acetate
Neoplasms, Plasma Cell

Additional relevant MeSH terms:

Anti-Inflammatory Agents
Neoplasms by Histologic Type
Antineoplastic Agents, Hormonal
Immune System Diseases
Antineoplastic Agents
Physiological Effects of Drugs
Hormones, Hormone Substitutes, and Hormone Antagonists
Gastrointestinal Agents
Antiemetics

Hormones
Glucocorticoids
Pharmacologic Actions
Neoplasms
Autonomic Agents
Therapeutic Uses
Cardiovascular Diseases
Peripheral Nervous System Agents
Central Nervous System Agents

ClinicalTrials.gov processed this record on January 14, 2009