Second Autologous Stem Cell Transplant in Treating Patients With Persistent or Recurrent Primary Systemic (AL) Amyloidosis - Full Text View

Sponsor:	Boston Medical Center
Information provided by (Responsible Party):	Boston Medical Center
ClinicalTrials.gov Identifier:	NCT00075608

Purpose

RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that more plasma cells are killed. By reducing the number of plasma cells, the disease may progress more slowly.

PURPOSE: This phase II trial is studying how well autologous stem cell transplant works in treating patients with persistent or recurrent primary systemic (AL) amyloidosis.

Condition	Intervention	Phase
Multiple Myeloma and Plasma Cell Neoplasm	Biological: filgrastim Drug: melphalan Procedure: autologous bone marrow transplantation Procedure: peripheral blood stem cell transplantation	Phase II

Study Type:	Interventional
Study Design:	Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Phase II Trial of Second Autologous Transplantation in AL Amyloidosis

Resource links provided by NLM:

Genetics Home Reference related topics: aceruloplasminemia hemophilia

MedlinePlus related topics: Amyloidosis Bone Marrow Transplantation Cancer Multiple Myeloma

Drug Information available for: Filgrastim Lenograstim Granulocyte colony-stimulating factor Melphalan Sarcolysin Melphalan hydrochloride

U.S. FDA Resources

Further study details as provided by Boston Medical Center:

Primary Outcome Measures:

Feasibility and tolerability [ Time Frame: 3 months after treatment and annually ] [ Designated as safety issue: Yes ]
Response and durability of response [ Time Frame: 3 months after treatment and annually ] [ Designated as safety issue: No ]
Evaluate immune reconstitution [ Time Frame: 3 months after treatment and annually ] [ Designated as safety issue: No ]

Enrollment:	12
Study Start Date:	August 2001
Study Completion Date:	October 2011
Primary Completion Date:	October 2011 (Final data collection date for primary outcome measure)

Intervention Details:

16mcg/kg IV daily beginning three days prior to SCC through last day of SCC

Other Name: G-CSF

140-200 mcg/kg IV over two days

Other Name: alkeran

infusion of previously collected stem cells on Day 0

Detailed Description:

OBJECTIVES:

Determine the feasibility and tolerability of second autologous stem cell transplantation in patients with persistent or recurrent AL amyloidosis.
Determine the response rate and durability of response in patients treated with this regimen.
Determine immune reconstitution in patients treated with this regimen.

OUTLINE:

Mobilization: Patients receive filgrastim (G-CSF) subcutaneously (SC) once daily beginning before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection.
Preparative regimen: Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2.
Autologous stem cell transplantation: Autologous stem cells are reinfused on day 0.

Patients are followed at 6 months, 1 year, and then annually thereafter.

PROJECTED ACCRUAL: A total of 19 patients will be accrued for this study within 5-6 years.

Eligibility

Ages Eligible for Study:	18 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

DISEASE CHARACTERISTICS:

Histologically confirmed AL amyloidosis
- Persistent or recurrent disease after 1 course of prior high-dose chemotherapy
Previously treated with autologous stem cell transplantation
Significant initial improvement in organ function after prior high-dose melphalan, defined by at least 1 of the following:
- Complete hematologic remission (e.g., absence of monoclonal spike by immunofixation in serum and urine AND less then 5% plasma cells in bone marrow with no clonal predominance) OR partial hematologic response (e.g., any decrease in serum or urine monoclonal protein OR decrease in bone marrow plasmacytosis)
- Greater than 50% reduction in proteinuria with preservation of creatinine clearance
- Greater than 50% reduction in alkaline phosphatase OR at least 2 cm decrease in liver size by physical exam
- Subjective neurologic improvement, as confirmed by neurologist
- Cardiac stabilization of disease confirmed by echocardiography defined as less than 2 mm increase in mean wall thickness and/or less than 20 g increase in left ventricular mass
- Improvement in performance status* NOTE: *This criteria alone does not constitute significant improvement in organ function
No myelodysplastic syndromes
No abnormal bone marrow cytogenetics
Prior stem cell yield must have been ≥ 2 x 10^6 CD34+ cells/kg

PATIENT CHARACTERISTICS:

Age

18 to 65

Performance status

SWOG 0-2

Life expectancy

More than 6 months

Hematopoietic

See Disease Characteristics

Hepatic

See Disease Characteristics

Renal

See Disease Characteristics

Cardiovascular

See Disease Characteristics
LVEF ≥ 45% by MUGA or echocardiogram

Pulmonary

DLCO ≥ 50%

Other

Not pregnant or nursing
Fertile patients must use effective contraception
Acceptable toxicity from first transplantation, confirmed by the transplant team
HIV negative
No other concurrent malignancy except treated skin cancer

PRIOR CONCURRENT THERAPY:

Biologic therapy

See Disease Characteristics

Chemotherapy

See Disease Characteristics
No chemotherapy after first transplantation

Endocrine therapy

Not specified

Radiotherapy

Not specified

Surgery

Not specified

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00075608

Locations

United States, Massachusetts
Boston University Cancer Research Center
Boston, Massachusetts, United States, 02118

Sponsors and Collaborators

Boston Medical Center

Investigators

Principal Investigator:

Karen Quillen, MD

Boston Medical Center

More Information

Additional Information:

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

No publications provided

Responsible Party:	Boston Medical Center
ClinicalTrials.gov Identifier:	NCT00075608 History of Changes
Other Study ID Numbers:	CDR0000347379, BUMC-2001-0156
Study First Received:	January 9, 2004
Last Updated:	December 9, 2011
Health Authority:	United States: Food and Drug Administration

Keywords provided by Boston Medical Center:

primary systemic amyloidosis

Additional relevant MeSH terms:

Amyloidosis
Neoplasms
Multiple Myeloma
Neoplasms, Plasma Cell
Plasmacytoma
Proteostasis Deficiencies
Metabolic Diseases
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders

Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Melphalan
Lenograstim
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antineoplastic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on February 12, 2012