Tandem Autologous Stem Cell Transplantation in Treating Patients With Primary Systemic (AL) Amyloidosis
This study is ongoing, but not recruiting participants.
Sponsor:
Boston Medical Center
Information provided by (Responsible Party):
Boston Medical Center
ClinicalTrials.gov Identifier:
NCT00075621
First received: January 9, 2004
Last updated: July 6, 2012
Last verified: June 2012
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Purpose
RATIONALE: Autologous stem cell transplantation may be effective treatment for primary systemic (AL) amyloidosis.
PURPOSE: This phase II trial is studying how well tandem (two) autologous stem cell transplantation works in treating patients with primary systemic (AL) amyloidosis.
| Condition | Intervention | Phase |
|---|---|---|
|
Multiple Myeloma and Plasma Cell Neoplasm |
Drug: filgrastim Drug: melphalan Procedure: autologous peripheral blood stem cell transplantation |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase II Trial of Tandem Transplantation in AL Amyloidosis |
Resource links provided by NLM:
Drug Information available for:
Melphalan
Melphalan hydrochloride
Tandem
Filgrastim
Lenograstim
Granulocyte colony-stimulating factor
U.S. FDA Resources
Further study details as provided by Boston Medical Center:
Primary Outcome Measures:
- safety [ Time Frame: 100 days, 6 months, and annual ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- Efficacy [ Time Frame: one year ] [ Designated as safety issue: No ]
| Enrollment: | 62 |
| Study Start Date: | August 2000 |
| Estimated Study Completion Date: | May 2015 |
| Primary Completion Date: | June 2005 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
tandem transplant
All patients will have tandem transplants, unless Complete response is achieved after the first transplant.
|
Drug: filgrastim
16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection
Drug: melphalan
200 mg/kg over 2 days
Other Name: alkeran
Procedure: autologous peripheral blood stem cell transplantation
autologous peripheral blood stem cell transplantation
|
Detailed Description:
OBJECTIVES:
- Determine the tolerability of tandem autologous stem cell transplantation in patients with AL amyloidosis.
- Determine whether this regimen can convert a hematologic non-complete response (CR) to CR in these patients.
- Determine the overall survival of patients treated with this regimen.
OUTLINE:
- First transplantation: Patients receive filgrastim (G-CSF) subcutaneously once daily beginning 3 days before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection. Patients may undergo bone marrow harvest if an inadequate number of peripheral blood stem cells are collected.
Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2. Patients undergo autologous stem cell transplantation (ASCT) on day 0.
- Second transplantation: Within 6-12 months after the first ASCT, patients not achieving a complete response receive high-dose melphalan IV over 20 minutes on days -3 and -2 and a second ASCT on day 0.
Treatment continues in the absence of unacceptable toxicity.
Patients are followed at 3 and 6 months, 1 year, and then annually thereafter.
PROJECTED ACCRUAL: A total of 62 patients will be accrued for this study within 2-3 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, meeting 1 of the following criteria:
Plasma cell dyscrasia, evidenced by 1 of the following:
- Monoclonal protein in the serum or urine by immunofixation electrophoresis
- Plasmacytosis of the bone marrow with monoclonal staining for kappa or lambda light chain isotype
- Macroglossia with at least 1 other site having biopsy proven amyloidosis and absence of a mutant transthyretin is ruled out
- No senile, secondary, localized, dialysis-related, or familial amyloidosis
- No overt multiple myeloma (e.g., greater than 30% bone marrow plasmacytosis, extensive [more than 2] lytic lesions, hypercalcemia)
PATIENT CHARACTERISTICS:
Age
- 18 to 65
Performance status
- SWOG 0-2
Life expectancy
- At least 1 year
Hematopoietic
- Not specified
Hepatic
- Not specified
Renal
- Not specified
Cardiovascular
- LVEF ≥ 45% by MUGA or echocardiogram
- No myocardial infarction within the past 6 months
- No congestive heart failure
- No arrhythmia refractory to therapy
- No evidence of symptomatic transient ischemic attacks or strokes
Pulmonary
- DLCO ≥ 50%
Other
- Not pregnant or nursing
- Fertile patients must use effective contraception
- Able to tolerate 2 courses of high-dose therapy
- HIV negative
- No other malignancy within the past 5 years except adequately treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, or adequately treated stage I or II cancer currently in complete remission
PRIOR CONCURRENT THERAPY:
Biologic therapy
- Not specified
Chemotherapy
- Prior alkylating agent chemotherapy allowed provided there is no morphologic or cytogenetic evidence of myelodysplastic syndromes
- Prior total cumulative oral melphalan dose < 300 mg
Endocrine therapy
- Not specified
Radiotherapy
- Not specified
Surgery
- Not specified
Other
- At least 4 weeks since prior cytotoxic therapy and recovered
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00075621
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: | Vaishali Sanchorawala, MD | Boston Medical Center |
More Information
Additional Information:
No publications provided
| Responsible Party: | Boston Medical Center |
| ClinicalTrials.gov Identifier: | NCT00075621 History of Changes |
| Other Study ID Numbers: | CDR0000347381, BUMC-2000-0279 |
| Study First Received: | January 9, 2004 |
| Last Updated: | July 6, 2012 |
| Health Authority: | United States: Federal Government |
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 May 22, 2013