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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.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT01083316
Date of registration: 02/11/2009
Prospective Registration: Yes
Primary sponsor: Boston Medical Center
Public title: Bortezomib and Dexamethasone Followed by High-Dose Melphalan and Stem Cell Transplantation for Primary (AL) Amyloidosis
Scientific title: Phase II Trial of Induction Therapy With Bortezomib and Dexamethasone Followed by High-Dose Melphalan and Stem Cell Transplantation in Patients With AL Amyloidosis
Date of first enrolment: September 2009
Target sample size: 35
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT01083316
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Name:     Vaishali Sanchorawala, MD
Address: 
Telephone:
Email:
Affiliation:  Boston Medical Center
Key inclusion & exclusion criteria

Inclusion Criteria:

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

1. Deposition of amyloid material by congo red stain showing characteristic green
birefringence, and

2. monoclonal light chain protein in the serum or urine or immunohistochemical
studies or serum free light chain assay and

3. evidence of tissue involvement other than carpal tunnel syndrome, i.e. positive
immunohistochemical staining of bone marrow demonstrating clonal plasma cells; or
tissue amyloid deposits with anti-kappa or anti-lambda anti-serum; evidence for a
plasma cell dyscrasia (PCD) by serum/urine or bone marrow; or overwhelmingly
convincing clinical features e.g. macroglossia, associated with other systemic
manifestations.

Note: 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.

- Must be at least 18 years of age.

- Must have a performance status of 0-2 by Southwest Oncology Group criteria

- Must have left ventricular ejection fraction (LVEF) at least 45% by echocardiogram
within 60 days of enrollment

- 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 less than 300 mg. Patients should not have
received any cytotoxic therapy less than 4 weeks prior to registration and should have
fully recovered from the effects of such therapy.

- Pulmonary Function Tests must show Diffusing capacity of the lungs for carbon monoxide
(DLCO) at least 50%.

- 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 the subject at any time without prejudice to future medical care.

- Female subject is either post-menopausal or surgically sterilized or 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.

Exclusion Criteria:

- No overt multiple myeloma (over 30% bone marrow plasmacytosis, extensive (great than
2) lytic lesions, hypercalcemia).

- 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 3 years.

- No known to be HIV positive.

- No platelet count of less than or equal to 70,000 within 14 days before enrollment.

- No absolute neutrophil count of less than or equal to 1000 within 14 days before
enrollment.

- No greater than or equal to Grade 2 peripheral neuropathy within 14 days before
enrollment.

- No myocardial infarction within 6 months prior to enrollment or has New York Heart
Association (NYHA) Class III or IV heart failure (see section 8.4), uncontrolled
angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence
of acute ischemia or active conduction system abnormalities. Prior to study entry, any
ECG abnormality at Screening has to be documented by the investigator as not medically
relevant.

- No hypersensitivity to bortezomib, boron or mannitol.

- No pregnant or breast-feeding females. Confirmation that the subject is not pregnant
must be established by a negative serum beta-human chorionic gonadotropin (beta-hCG)
pregnancy test result obtained during screening. Pregnancy testing is not required for
post-menopausal or surgically sterilized women.

- Must not have received other investigational drugs with 14 days before enrollment

- No serious medical or psychiatric illness likely to interfere with participation in
this clinical study.



Age minimum: 18 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Amyloidosis
Intervention(s)
Drug: Bortezomib
Drug: Dexamethasone
Drug: Melphalan
Primary Outcome(s)
Number of Participants Proceeding to Transplant Following Induction [Time Frame: 2 months]
Number of Participants Surviving at 100 Days Post Transplant [Time Frame: 100 days]
Number of Participants With Disease Response [Time Frame: One year]
Secondary Outcome(s)
Number of Participants Surviving at 5 Years [Time Frame: 5 years]
Secondary ID(s)
H-28441
X05292
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Millennium Pharmaceuticals, Inc.
Ethics review
Results
Results available: Yes
Date Posted: 06/02/2017
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT01083316
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