High-Dose Melphalan Plus Peripheral Stem Cell Transplantation in Treating Patients With Primary Systemic Amyloidosis
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| ClinicalTrials.gov Identifier: NCT00003353 |
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Recruitment Status :
Completed
First Posted : September 13, 2004
Last Update Posted : August 23, 2013
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RATIONALE: High-dose chemotherapy may destroy the amyloid-producing cells in bone marrow. Peripheral stem cell transplantation
PURPOSE: Phase II trial to study the effectiveness of high dose melphalan plus peripheral stem cell transplantation in treating patients who have primary systemic amyloidosis.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Multiple Myeloma and Plasma Cell Neoplasm | Biological: filgrastim Drug: melphalan Procedure: bone marrow ablation with stem cell support Procedure: peripheral blood stem cell transplantation | Phase 2 |
OBJECTIVES: I. Assess the response rate and overall survival of patients with biopsy proven primary amyloidosis following treatment with myeloablative chemotherapy and hematopoietic stem cell reconstitution. II. Evaluate the toxicity of high dose melphalan in this patient population.
OUTLINE: Patients receive filgrastim (G-CSF) subcutaneously daily beginning on day 1 of the peripheral blood stem cell (PBSC) collection period and continuing until PBSC collection is completed. PBSC are collected beginning on day 5 of the collection period and continuing until the final target cell count is reached or for up to a maximum of 7 collections. If sufficient PBSCs are not harvested within a maximum of 7 collections, the patient is removed from the study. Within 30 days of PBSC collection, patients receive melphalan IV on day -1 of the infusion period and PBSC infusion on day 0. The infusion period continues until day 30. Patients receive G-CSF subcutaneously daily starting on day 1 and continuing until blood counts recover. Patients are followed every 3 months for 2 years, every 3 months for 3 additional years, and yearly thereafter.
PROJECTED ACCRUAL: A maximum of 33 patients will be accrued for this study over 2 years.
| Study Type : | Interventional (Clinical Trial) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase II Study of High-Dose Melphalan With Hematopoietic Stem Cell Reconstitution for Patients With Primary Systemic Amyloidosis |
| Study Start Date : | July 1998 |
| Actual Primary Completion Date : | December 2002 |
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| Ages Eligible for Study: | 18 Years to 70 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS: Histologically confirmed primary amyloidosis Must have presence of paraprotein in serum or urine determined by immunoelectrophoresis/immunofixation No primary amyloidosis manifested only by carpal tunnel syndrome or purpura No history of secondary, familial, or localized amyloidosis No evidence of overt multiple myeloma: Lytic bone disease or pathological fractures OR At least 30% plasma cells in bone marrow
PATIENT CHARACTERISTICS: Age: 18 to 70 Performance status: ECOG 0-2 Life expectancy: Not specified Hematopoietic: Absolute granulocyte count at least 1000/mm3 Platelet count at least 100,000/mm3 Hepatic: Bilirubin no greater than 2.0 mg/dL Alkaline phosphatase no greater than 1000 u/L or less than 4 times upper limit of normal Renal: Creatinine no greater than 2.0 mg/dL Cardiovascular: Confirmed by echocardiogram: Ejection fraction at least 50% Interventricular septal thickness no greater than 15 mm No New York Heart Association classification II-IV Pulmonary: DLCO at least 50% FVC at least 60% FEV1 at least 55% Other: Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception No active infection No other malignancy within the past 5 years except surgically treated carcinoma in situ of the cervix, nonmelanoma skin cancer, or indolent prostate cancer No known sensitivity to E. coli derivatives
PRIOR CONCURRENT THERAPY: Biologic therapy: Prior interferon allowed Chemotherapy: At least 4 weeks since prior melphalan Lifetime cumulative dose of melphalan no greater than 150 mg No greater than 2 prior courses of chemotherapy Endocrine therapy: Prior dexamethasone allowed Radiotherapy: Not specified Surgery: Not specified
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00003353
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| Study Chair: | Morie A. Gertz, MD | Mayo Clinic |
| ClinicalTrials.gov Identifier: | NCT00003353 |
| Other Study ID Numbers: |
CDR0000066334 E-4A97 |
| First Posted: | September 13, 2004 Key Record Dates |
| Last Update Posted: | August 23, 2013 |
| Last Verified: | August 2013 |
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primary systemic amyloidosis |
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Multiple Myeloma Neoplasms, Plasma Cell Plasmacytoma Immunoglobulin Light-chain Amyloidosis Amyloidosis Neoplasms by Histologic Type Neoplasms Hemostatic Disorders Vascular Diseases Cardiovascular Diseases Paraproteinemias Blood Protein Disorders Hematologic Diseases Hemorrhagic Disorders |
Lymphoproliferative Disorders Immunoproliferative Disorders Immune System Diseases Proteostasis Deficiencies Metabolic Diseases Melphalan Immunologic Factors Physiological Effects of Drugs Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Myeloablative Agonists Immunosuppressive Agents |