Bortezomib, Melphalan, and Dexamethasone in Treating Patients With Primary Amyloidosis or Light Chain Deposition Disease

NCT ID: NCT00520767

Last Updated: 2023-12-22

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

35 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-09-30

Study Completion Date

2019-06-06

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

RATIONALE: Giving bortezomib together with melphalan and dexamethasone may be an effective treatment for primary amyloidosis and light chain deposition disease.

PURPOSE: This phase II trial is studying how well giving bortezomib together with melphalan and dexamethasone works in treating patients with primary amyloidosis or light chain deposition disease.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

OBJECTIVES:

Primary

* Determine the complete hematologic response rate at 12 months.

Secondary

* Determine the overall hematologic response rate.
* Determine the organ response rate.
* Determine time to treatment failure.
* Determine the overall survival.

OUTLINE: This is a multicenter study.

Patients receive oral melphalan on days 1-4, bortezomib IV on days 1, 8, 15, and 22, and dexamethasone orally or IV on days 1, 2, 8, 9, 15, 16, 22, and 23. Treatment repeats every 4-6 weeks for up to 20 courses in the absence of disease progression or unacceptable toxicity.

Blood, urine, and bone marrow aspirates are collected at baseline and periodically after treatment to permit the correlation of clinical results with measured molecular events. A single baseline peripheral blood DNA sample is collected for future association studies linking disease onset, progression, and response to administered therapy with single nucleotide polymorphisms. Blood plasma and urine samples are evaluated for proteomic markers associated with disease progression and therapeutic response. Peripheral blood RNA samples are evaluated for transcriptional response to treatment of peripheral blood lymphocytes. Bone marrow aspirates are collected to extract plasma cells by flow cytometry for gene expression profiling.

Quality of life is assessed at the beginning of each course.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Primary Systemic Amyloidosis Light Chain Deposition Disease

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

primary systemic amyloidosis light chain deposition disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Melphalan, Dexamethasone, Bortezomib,

Bortezomib 1.3 mg/m2 days 1, 8, 15, 22; Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23; Melphalan 9 mg/m2/day days 1-4

Group Type EXPERIMENTAL

bortezomib

Intervention Type DRUG

Bortezomib 1.3 mg/m2 days 1, 8, 15, 22

dexamethasone

Intervention Type DRUG

Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23

melphalan

Intervention Type DRUG

Melphalan 9 mg/m2/day days 1-4

microarray analysis

Intervention Type GENETIC

≤28 days prior to enrollment

flow cytometry

Intervention Type OTHER

Day 1 of cycles 6, 12, 18 and at end of study.

laboratory biomarker analysis

Intervention Type OTHER

≤28 days prior to enrollment

quality-of-life assessment

Intervention Type PROCEDURE

Start of each cycle

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

bortezomib

Bortezomib 1.3 mg/m2 days 1, 8, 15, 22

Intervention Type DRUG

dexamethasone

Dexamethasone 40 mg/d days 1, 2, 8, 9, 15, 16, 22, 23

Intervention Type DRUG

melphalan

Melphalan 9 mg/m2/day days 1-4

Intervention Type DRUG

microarray analysis

≤28 days prior to enrollment

Intervention Type GENETIC

flow cytometry

Day 1 of cycles 6, 12, 18 and at end of study.

Intervention Type OTHER

laboratory biomarker analysis

≤28 days prior to enrollment

Intervention Type OTHER

quality-of-life assessment

Start of each cycle

Intervention Type PROCEDURE

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Velcade Dexasone Decadron Diodex Hexadrol Maxidex Dexamethasone Sodium Phosphate Dexamethasone Acetate Alkeran® L-PAM L-Sarcolysin Phenylalanine Mustard

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* If not previously treated, patient is either not a candidate for autologous stem cell transplantation (ASCT) or has declined the option of ASCT

* Patients who have undergone prior ASCT and have subsequently progressed are eligible, provided other eligibility criteria are met
* No secondary or familial amyloidosis

PATIENT CHARACTERISTICS:

* ECOG performance status 0-3
* Creatinine \< 5 mg/dL
* Bilirubin \< 2.5 times upper limit of normal (ULN)
* ALT and AST \< 3 times ULN
* Absolute neutrophil count ≥ 1,000/mm³
* Platelet count ≥ 80,000/mm³
* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use effective contraception
* Peripheral sensory neuropathy \< grade 3
* No myocardial infarction within the past 6 months
* No New York Heart Association class III or IV heart failure
* No uncontrolled angina
* No severe uncontrolled ventricular arrhythmias
* No EKG\* evidence of acute ischemia or active conduction system abnormalities (not including 1st degree AV-block, Wenckebach type 2nd degree heart block, or left bundle branch block) NOTE: \*Prior to study entry, any EKG screening abnormality must be documented by the investigator as not medically relevant; there is no lower limit of LVEF below which patients are excluded from participation
* No hypersensitivity to bortezomib, boron, or any of the other agents utilized in this study
* No serious concurrent illness (e.g., stroke) within the past 30 days
* No psychiatric illness likely to interfere with study participation
* No untreated HIV infection

* Patients with asymptomatic HIV infection on active antiretroviral therapy are potentially eligible
* No diagnosis or treatment of another malignancy within the past 3 years, except completely resected basal cell or squamous cell carcinoma of the skin, an in situ malignancy, or low-risk prostate cancer after curative therapy

PRIOR CONCURRENT THERAPY:

* See Disease Characteristics
* No other investigational drugs within the past 14 days
Minimum Eligible Age

18 Years

Maximum Eligible Age

120 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

Barbara Ann Karmanos Cancer Institute

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Jeffrey Zonder

Principal Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Jeffrey A. Zonder, MD

Role: PRINCIPAL_INVESTIGATOR

Barbara Ann Karmanos Cancer Institute

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Rocky Mountain Cancer Centers/Rocky Mountain Blood & Marrow Transplant Program

Denver, Colorado, United States

Site Status

Boston University Cancer Research Center

Boston, Massachusetts, United States

Site Status

Barbara Ann Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status

Josephine Ford Cancer Center at Henry Ford Hospital

Detroit, Michigan, United States

Site Status

Providence Cancer Institute at Providence Hospital - Southfield Campus

Southfield, Michigan, United States

Site Status

Duke Comprehensive Cancer Center

Durham, North Carolina, United States

Site Status

UPMC Cancer Centers

Pittsburgh, Pennsylvania, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Related Links

Access external resources that provide additional context or updates about the study.

http://cancer.gov/clinicaltrials

Clinical trial summary from the National Cancer Institute's PDQ® database

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

P30CA022453

Identifier Type: NIH

Identifier Source: secondary_id

View Link

MILLENNIUM-WSU-2006-132

Identifier Type: -

Identifier Source: secondary_id

WSU-HIC-060907M1F

Identifier Type: OTHER

Identifier Source: secondary_id

2006-132

Identifier Type: -

Identifier Source: org_study_id