Phase 2
N=2
Bortezomib, Cyclophosphamide, and Dexamethasone in Treating Patients With Primary Systemic Light Chain Amyloidosis
Primary Systemic Amyloidosis
Bottom Line
View on ClinicalTrials.gov: NCT01072773 ↗Enrolled (actual)
2
Serious AEs
50.0%
Results posted
Dec 2012
Primary outcome: Primary: Number of Participants With a Confirmed Hematologic Response — 0; 0; 0 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- bortezomib (Drug); cyclophosphamide (Drug); dexamethasone (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Mayo Clinic
- Primary completion
- Mar 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With a Confirmed Hematologic Response |
0; 0; 0 | — |
| SECONDARY Number of Participants With Treatment Related Adverse Events. |
2; 0; 0 | — |
| SECONDARY Number of Participants With an Organ Response. |
— | — |
| SECONDARY Overall Survival |
1.45 | — |
| SECONDARY Time to Disease Progression |
1.45 | — |
| SECONDARY Duration of Response |
— | — |
Summary
RATIONALE: Bortezomib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as cyclophosphamide and dexamethasone, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving bortezomib together with combination chemotherapy may kill more cancer cells.
PURPOSE: This phase II trial is studying how well giving bortezomib, cyclophosphamide, and dexamethasone together works in treating patients with primary systemic light chain amyloidosis.
Eligibility Criteria
Inclusion Criteria
- Histochemical diagnosis of amyloidosis as based on detection by polarizing microscopy of green birefringent material in Congo red-stained tissue specimens
- Measurable disease of amyloid light chain amyloidosis as defined by at least ONE of the following: serum monoclonal protein >= 1.0 g by protein electrophoresis, > 200 mg of monoclonal protein in the urine on 24 hour electrophoresis, serum free light-chain >= 7.5 mg/dL with an abnormal kappa:lambda ratio
- Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) 0, 1 or 2
- Absolute neutrophil count >= 1000/uL
- Platelet >= 75000/uL
- Total bilirubin = 30ml/min
- Women of childbearing potential should have a negative serum or urine pregnancy test done = 0.5 g/day in a 24- hour urine collection
- Cardiac involvement is defined as the presence of a mean left ventricular wall thickness on echocardiogram greater than 12 mm in the absence of a history of hypertension or valvular heart disease, or in the presence of unexplained low voltage ( = 2 cm below costal margin) on physical exam or an alkaline phosphatase > 1.5 x ULN
- Peripheral nerve involvement is defined based on clinical history or abnormal sensory and/or motor findings on neurologic exam
- Autonomic nerve involvement is defined as orthostasis, symptoms of nausea or dysgeusia, gastric atony by gastric emptying scan, diarrhea or constipation
- Soft tissue and lymphatic involvement may be ascertained based on classic physical exam findings (macroglossia, shoulder pad sign, raccoon eyes, carpal tunnel syndrome, synovial enlargement, firm enlarged lymph nodes) or biopsy
- Voluntary written informed consent before performance of any study-related procedure not part of normal medical care, with the understanding that consent may be withdrawn by subject any time without prejudice to future medical care
- Willingness to return to Mayo Clinic enrolling institution for follow-up
Exclusion Criteria
- Melphalan or other myelosuppressive agents = 30%), and at least one of the following: bone lesions or hypercalcemia
- History of myocardial infarction = 0.1 ng/mL
Data sourced from ClinicalTrials.gov (NCT01072773). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.