Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00790647
• Date of registration: 12/11/2008
• Prospective Registration: No
• Primary sponsor: Boston Medical Center
• Public title: Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis
• Scientific title: Phase II Trial of High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis
• Date of first enrolment: June 2008
• Target sample size: 10
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00790647
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Vaishali Sanchorawala, MD
• Affiliation: Boston Medical Center

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Multiple Myeloma

Intervention(s)

Biological: filgrastim

Procedure: Stem Cell Infusion

Drug: melphalan

Drug: bortezomib

Primary Outcome(s)

Number of Participants With Hematologic Response [Time Frame: one year]

Secondary Outcome(s)

Number of Participants Surviving at 1 Year [Time Frame: one year from transplant]

Number of Participants Surviving at 100 Days From Transplant [Time Frame: 100 Days from transplant date]

Number of Participants Surviving at 2 Years [Time Frame: 2 years from transplant]

Secondary ID(s)

BUMC-H-27277

CDR0000618857

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 06/02/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00790647