Clinical trial · Interventional
Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis
Phase II Trial of High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
RATIONALE: Giving melphalan and bortezomib before and after a stem cell transplant stops the growth of abnormal cells by stopping them from dividing or killing them. Giving colony-stimulating factors and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy and monoclonal antibody therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy. PURPOSE: This phase II trial is studying how well giving melphalan together with bortezomib followed by stem cell transplant works in treating patients with primary systemic amyloidosis.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Multiple Myeloma | Multiple Myeloma | CURATED_EXACT | 0.92 |
Interventions
Interventions (4)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| bortezomib | Drug | Bortezomib | ALIAS |
| filgrastim | Biological | Filgrastim | ALIAS |
| melphalan | Drug | Melphalan | ALIAS |
| Stem Cell Infusion | Procedure | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Stem Cell Transplant with Bortezomib and Melphalan
- description
- Mobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
- interventionNames
- Biological: filgrastim
- Drug: bortezomib
- Drug: melphalan
- Procedure: Stem Cell Infusion
Primary outcomes (1)
- measure
- Number of Participants With Hematologic Response
- timeFrame
- one year
- description
- complete and partial hematologic response defined as: Complete response: absence of detectable monoclonal protein in serum and urine, and bone marrow biopsy \<5% plasma cells with no clonal predominance of kappa or lambda isotype. Partial response: any one of the following 1. For patients with detectable and quantifiable marrow plasmacytosis, a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells. 2. For patients with a detectable monoclonal peak on serum protein electropheresis or urine protein electropheresis, a reduction in the peak height of 50% or more. 3. For patients with quantifiable urinary kappa or lambda chain concentration, a reduction in daily light chain excretion (concentration x 24-hr urine volume).
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 65 Years
Show eligibility criteria text
Inclusion criteria: DISEASE CHARACTERISTICS: * Histologically confirmed primary systemic amyloidosis based on the following criteria: * Amyloid light-chain disease * Deposition of amyloid material by congo red stain showing characteristic green birefringence * Monoclonal light chain protein (Bence Jones protein) in the serum or urine, immunohistochemical studies, or serum free light chain assay * Evidence of tissue involvement other than carpal tunnel syndrome (i.e., positive immunohistochemical staining of bone marrow demonstrating clonal plasma cells); tissue amyloid deposits with anti-kappa or anti-lambda anti-serum; evidence for a plasma cell dyscrasia by serum/urine or bone marrow; or overwhelmingly convincing clinical features (e.g., macroglossia) associated with other systemic manifestations PATIENT CHARACTERISTICS: * Southwest Oncology Group performance status 0-1 * Fertile patients must use effective contraception * Left ventricular ejection fraction ≥ 45% by Echocardiogram within the past 60 days * diffusion capacity of lung for carbon monoxide ≥ 50% PRIOR CONCURRENT THERAPY: * Prior chemotherapy with alkylating agent allowed provided there is no morphological or cytogenetic evidence of myelodysplastic syndromes * Prior total cumulative dose of oral melphalan \< 300 mg * At least 4 weeks since prior cytotoxic therapy and fully recovered Exclusion criteria: * No senile, secondary, localized, dialysis-related, or familial amyloidosis * No overt multiple myeloma (\> 30% of bone marrow plasmacytosis, extensive \[\> 2\] lytic lesions, or hypercalcemia) * Not pregnant or nursing * No myocardial infarction within the past 6 months, congestive heart failure, or arrhythmia refractory to therapy * No prior malignancy except for any of the following: * Adequately treated basal cell or squamous cell skin cancer * In situ cervical cancer * Adequately treated stage I or II cancer currently in complete remission * Any cancer from which the patient has been disease-free ≥ 5 years * No advanced (grade 3-4) pre-existing neuropathy * No HIV positivity
References
Publications (0)
Data not yet available