Phase II Trial of Second Autologous Transplantation in AL Amyloidosis
filgrastim
+ melphalan
+ autologous stem cell transplantation
Immunoglobulin Light-chain Amyloidosis+17
+ Amyloidosis
+ Blood Protein Disorders
Treatment Study
Summary
Study start date: August 1, 2001
Actual date on which the first participant was enrolled.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.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.12 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.From 18 to 65 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
Inclusion 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 * Prior stem cell yield must have been ≥ 2 x 10\^6 CD34+ cells/kg 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 PATIENT CHARACTERISTICS: Age * 18 to 65 Performance status * Southwest Oncology Group- 0-2 Life expectancy * More than 6 months Hematopoietic * See Disease Characteristics Hepatic * See Disease Characteristics Renal * See Disease Characteristics Cardiovascular * See Disease Characteristics * Left ventricular ejection fraction ≥ 45% by multiple gated acquisition scan or echocardiogram Pulmonary * diffusing capacity of lung for carbon monoxide ≥ 50% Exclusion Criteria: * No myelodysplastic syndromes * No abnormal bone marrow cytogenetics 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
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.One single intervention group is designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalStudy Objectives
Primary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 1 location
Boston University Cancer Research Center
Boston, United StatesOpen Boston University Cancer Research Center in Google Maps