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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: 12 December 2020
Main ID:  NCT02489500
Date of registration: 09/03/2015
Prospective Registration: Yes
Primary sponsor: Boston Medical Center
Public title: Trial of High Dose Melphalan/Stem Cell Transplant With or Without Bortezomib VelRand
Scientific title: Phase III Trial of High-dose Melphalan and Stem Cell Transplantation Versus High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis
Date of first enrolment: June 2015
Target sample size: 3
Recruitment status: Terminated
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 3
Countries of recruitment
United States
Name:     Vaishali Sanchorawala, MD
Affiliation:  Boston Medical Center
Key inclusion & exclusion criteria

Inclusion Criteria:

1. Histological diagnosis of primary systemic (AL) amyloidosis based on:

- Deposition of amyloid material by Congo red stain showing characteristic apple
green birefringence,AND…

- evidence of a clonal plasma cell dyscrasia with monoclonal protein in the serum
or urine by immunofixation electrophoresis studies AND/OR abnormal serum free
light chain assay AND/OR clonal plasma cells in the bone marrow exam demonstrated
by immunohistochemistry, flow cytometry or in situ hybridization AND…

- evidence of organ involvement other than carpal tunnel syndrome. Patients with
senile, secondary, localized, dialysis-related or familial amyloidosis are not
eligible. Confirmation of tissue diagnosis at all sites of organ dysfunction is
encouraged, but not required.

2. Patients must be > 18 years of age.

3. Patients must have a performance status of 0-2 by Eastern Cooperative Oncology Group
(ECOG) criteria

4. Patients must have left ventricular ejection fraction (LVEF) > 45% by echocardiogram
within 60 days of enrollment

5. Pulmonary Function Tests must show diffusing capacity of lung for carbon monoxide
(DLCO) > 50%.

6. All patients must be informed of the investigational nature of this study and must
sign and give written informed consent in accordance with institutional and federal

Exclusion Criteria:

1. Patients with recent (< 6 months) myocardial infarction, congestive heart failure, New
York Heart Association (NYHA) class III/IV or arrhythmia which are refractory to
medical therapy are ineligible.

2. Prior chemotherapy with alkylating agent allowed only if no evidence of
Myelodysplastic Dysplastic Syndrome (MDS) morphologically or cytogenetically. Total
cumulative dose of oral melphalan must be < 300 mg. Patients should not have received
any cytotoxic therapy < 4 weeks prior to registration and should have fully recovered
from the effects of such therapy.

3. Patients must not have overt multiple myeloma (>30% bone marrow plasmacytosis and,
extensive (>2) lytic lesions and hypercalcemia).

4. No other prior malignancy is allowed except for the following: adequately treated
basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated
Stage I or II cancer from which the patient is currently in complete remission, or any
other cancer from which the patient has been disease-free for 5 years.

5. Patients must not be HIV positive.

6. Pregnant or nursing women may not participate. Women and men of reproductive potential
may not participate unless they have agreed to use an effective contraceptive method.

Age minimum: 18 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
AL Amyloidosis
Drug: Bortezomib
Drug: Melphalan
Drug: Neupogen
Procedure: Stem Cell Collection
Procedure: Stem cell infusion
Primary Outcome(s)
Number of Participants With Hematologic Response [Time Frame: 6 months]
Secondary Outcome(s)
Number of Participants With Organ Response [Time Frame: 5 years]
Overall Survival [Time Frame: 5 years]
Toxicities [Time Frame: 100 days]
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: 06/08/2018
Date Completed:
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