High-Dose Melphalan Followed by Peripheral Stem Cell Transplant in Treating Patients With Amyloidosis

NCT00002810 | Completed Phase 2

• 3 other identifiers: CDR0000064938TUHSC-2797NCI-V96-0951
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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 peripheral 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 giving high-dose melphalan together with peripheral stem cell transplant works in treating patients with primary amyloidosis or amyloidosis associated with multiple myeloma.

Conditions

Multiple Myeloma and Plasma Cell Neoplasm

Keywords

refractory multiple myeloma; stage I multiple myeloma; stage II multiple myeloma; stage III multiple myeloma; primary systemic amyloidosis

Outcome Measures

Primary Outcomes (2)

• Overall survival

• Time to clinical progression of amyloid symptoms

Interventions

filgrastim BIOLOGICAL

melphalan DRUG

bone marrow ablation with stem cell support PROCEDURE

peripheral blood stem cell transplantation PROCEDURE

Eligibility Criteria

• Age: 16 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

DISEASE CHARACTERISTICS: * Primary amyloidosis diagnosed by appropriate amyloid stains or electromicroscopy of abdominal fat, bone marrow, or other target tissues * Pathology reviewed by Temple University * Amyloidosis secondary to any stage of multiple myeloma allowed provided plasma cell concentration in bone marrow is less than 15% * No amyloidosis secondary to rheumatoid arthritis or chronic infection * No familial amyloidosis PATIENT CHARACTERISTICS: Age: * 16 to 65 Performance status: * Karnofsky 80-100% Hematopoietic: * Not specified Hepatic: * Liver function tests less than twice normal * No active liver disease Renal: * Creatinine clearance greater than 50 mL/min * Nephrotic syndrome allowed Cardiovascular: * Cardiac evaluation required in patients with left ventricular ejection fraction less than 45% by echocardiogram or MUGA * No poorly controlled hypertension Pulmonary: * FEV\_1 and DLCO greater than 50% of predicted, or pulmonary evaluation required * No chronic obstructive pulmonary disease Other: * No history of serious coagulopathy, hemorrhage, or bleeding * No active infection * No other serious comorbid disease (e.g., poorly controlled diabetes) * No pregnant women * Adequate contraception required of fertile women PRIOR CONCURRENT THERAPY: Biologic therapy: * Not specified Chemotherapy: * More than 12 monthly cycles of prior alkylating agent chemotherapy discouraged Endocrine therapy: * Corticosteroids discontinued at least 6 weeks prior to transplantation Radiotherapy: * No prior radiotherapy Surgery: * Not specified

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Temple University: lead

Study Sites (1)

Fox Chase-Temple Cancer Center CCOP Research Base

Philadelphia, Pennsylvania, 19111-2442, United States

Location

MeSH Terms

Conditions

Multiple Myeloma; Neoplasms, Plasma Cell; Immunoglobulin Light-chain Amyloidosis

Interventions

Filgrastim; Melphalan; Peripheral Blood Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Hematopoietic Stem Cell Transplantation; Stem Cell Transplantation; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative

Study Officials

• Kenneth F. Mangan, MD, FACP

  Fox Chase Cancer Center

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: November 1, 1999
• First Posted: January 27, 2003
• Study Start: May 1, 1996
• Primary Completion: May 1, 2006
• Study Completion: May 1, 2006
• Last Updated: October 1, 2010

Record last verified: 2010-09

Locations

US (1)