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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Last refreshed on: 19 October 2017
Main ID:  NCT01072773
Date of registration: 17/02/2010
Prospective Registration: Yes
Primary sponsor: Mayo Clinic
Public title: Bortezomib, Cyclophosphamide, and Dexamethasone in Treating Patients With Primary Systemic Light Chain Amyloidosis
Scientific title: Phase II Study of Bortezomib, Cyclophosphamide and Dexamethasone in Patients With Primary Systemic Light Chain Amyloidosis
Date of first enrolment: March 2010
Target sample size: 2
Recruitment status: Completed
Study type:  Interventional
Study design:   
Phase:  Phase 2
Countries of recruitment
United States
Name:     Shaji Kumar, M.D.
Affiliation:  Mayo Clinic
Key inclusion & exclusion 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 < 3.0 mg/dL

- Aspartate aminotransferase (AST) =< 3 x upper limit of normal (ULN)

- Creatinine clearance >= 30ml/min

- Women of childbearing potential should have a negative serum or urine pregnancy test
done =< 7 days prior to registration, and should be willing to use an acceptable
method of birth control (i.e., a hormonal contraceptive, intra-uterine device,
diaphragm with spermicide, condom with spermicide, or abstinence) for the duration of
the study

- Male subject agrees to use an acceptable method for contraception for the duration of
the study

- Previously treated amyloidosis; no limit to prior therapy provided there is adequate
residual organ function

- Symptomatic organ involvement (heart, kidney, liver/GI tract, peripheral nervous
system or soft tissue); carpal tunnel syndrome skin purpura, or the presence of
vascular amyloid on a bone marrow biopsy alone are not sufficient to meet criteria for
"symptomatic organ involvement"

- Renal involvement is defined as proteinuria (predominantly albumin) > 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
(< 0.5 mV) on the electrocardiogram

- Hepatic involvement is defined as hepatomegaly (>= 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 =< 3 weeks prior to registration;
non-myelosuppressive agents like thalidomide, or high dose corticosteroids <= 1week
prior to registration

- Concurrent use of corticosteroids, but patients may be on chronic steroids (maximum
dose 20 mg/day prednisone equivalent) if they are being given for disorders other than
amyloid, i.e., adrenal insufficiency, rheumatoid arthritis, etc

- Any of the following because this study involves an agent whose genotoxic, mutagenic
and teratogenic effects on the developing fetus and newborn are unknown: pregnant
women and nursing women

- Other active malignancy =< 2 years prior to registration; EXCEPTIONS: Nonmelanotic
skin cancer or carcinoma-in-situ of the cervix; NOTE: If there is a history or prior
malignancy, they must not be receiving any specific treatment for their cancer

- Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment
of the investigator, would make the patient inappropriate for entry into this study or
interfere significantly with the proper assessment of safety and toxicity of the
prescribed regimens including psychiatric illness/social situations that would limit
compliance with study requirements

- Known to be HIV positive

- Receiving any other investigational agent which would be considered as a treatment for
the primary neoplasm

- Clinically overt multiple myeloma (monoclonal Bone Marrow Plasma Count > 30%), and at
least one of the following: bone lesions or hypercalcemia

- History of myocardial infarction =< 6 months, or requiring use of ongoing maintenance
drug therapy for life-threatening ventricular arrhythmias

- Grade 3 sensory or grade 1 painful peripheral neuropathy

- Known hypersensitivity to bortezomib, boron or mannitol

- Cardiac syncope, uncompensated New York Heart Association (NYHA) Class 3 or 4
congestive heart failure or troponin T > 0.1 ng/mL

Age minimum: 18 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Primary Systemic Amyloidosis
Drug: bortezomib
Drug: cyclophosphamide
Drug: dexamethasone
Primary Outcome(s)
Number of Participants With a Confirmed Hematologic Response [Time Frame: Duration of treatment (up to 12 cycles/months)]
Secondary Outcome(s)
Duration of Response [Time Frame: Duration of Study (up to 5 years)]
Number of Participants With an Organ Response. [Time Frame: Duration on treatment (up to 12 cycles/months)]
Number of Participants With Treatment Related Adverse Events. [Time Frame: Duration on treatment (up to 12 cycles/months)]
Overall Survival [Time Frame: Duration of Study (up to 5 years)]
Time to Disease Progression [Time Frame: Duration of Study (up to 5 years)]
Secondary ID(s)
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results available: Yes
Date Posted: 05/12/2012
Date Completed:
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