AzTrials
AzTrials

Primary Systemic AmyloidosisPhase 2NCT00520767

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

A Phase 2 study testing bortezomib, dexamethasone and melphalan in people with Primary Systemic Amyloidosis and Light Chain Deposition Disease, run by Barbara Ann Karmanos Cancer Institute at 7 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

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… Read more about this study

Who is it for?
Ages 18 to 120 years
What is being tested?
bortezomib, dexamethasone and melphalan
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
35 participants
Where
7 sitesUnited States
Study dates
Sep 2007to 6 Jun 2019
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 120 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

DISEASE CHARACTERISTICS:

  • Biopsy-proven diagnosis of 1 of the following:
  • Primary systemic amyloidosis
  • Histochemical diagnosis of amyloidosis determined by polarizing microscopy of green bi-refringent material in Congo red-stained tissue specimens or characteristic electron microscopy appearance
  • Light chain deposition disease
  • Measurable disease as defined by one or more of the following:
  • Serum monoclonal protein ≥ 0.5 g/dL by serum electrophoresis
  • Urine monoclonal protein > 200 mg/tv in a 24 hr urine electrophoresis
  • Serum immunoglobulin free-light chain ≥ 10 mg/dL AND abnormal serum immunoglobulin kappa lambda free light chain ratio
  • Must meet 1 of the following criteria:
  • Clonal population of plasma cells in the bone marrow (≤ 30%)
  • Immunohistochemical stain with anti-light chain anti-sera of amyloid fibrils
  • Must not meet the following diagnostic criteria for symptomatic* multiple myeloma:
  • Lytic lesions on skeletal survey
  • Plasmacytoma
  • Increase in bone marrow plasma cells ≥ 30% NOTE: *Patients who meet the International Myeloma Working Group definition of symptomatic multiple myeloma with symptoms attributable only to associated amyloidosis and who do not otherwise meet the criteria for diagnosis of smoldering myeloma are potentially eligible upon approval of the principal investigator.
  • 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

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

United States7 sites
  • Boston University Cancer Research Center

    Boston, Massachusetts, 02118

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

    Denver, Colorado, 80218

  • Barbara Ann Karmanos Cancer Institute

    Detroit, Michigan, 48201-1379

  • Josephine Ford Cancer Center at Henry Ford Hospital

    Detroit, Michigan, 48202

  • Duke Comprehensive Cancer Center

    Durham, North Carolina, 27710

  • UPMC Cancer Centers

    Pittsburgh, Pennsylvania, 15232

  • Providence Cancer Institute at Providence Hospital - Southfield Campus

    Southfield, Michigan, 48075

Other trials in United States

About this study

Official title: A Multicenter Phase II Trial of Bortezomib (Velcade), Melphalan, and Dexamethasone (V-MD) in Patients With Symptomatic AL-Amyloidosis or Light Chain Deposition Disease

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.

Read the detailed description

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.

Being tested
bortezomib, dexamethasone, melphalan, microarray analysis, flow cytometry
Keywords
primary systemic amyloidosis, light chain deposition disease
Medical subject headings
Immunoglobulin Light-chain Amyloidosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • bortezomibDrug

    Also called Velcade

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

  • dexamethasoneDrug

    Also called Dexasone, Decadron, Diodex, Hexadrol

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

  • melphalanDrug

    Also called Alkeran®, L-PAM, L-Sarcolysin, Phenylalanine Mustard

    Melphalan 9 mg/m2/day days 1-4

  • microarray analysisGenetic

    ≤28 days prior to enrollment

  • flow cytometryOther

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

  • laboratory biomarker analysisOther

    ≤28 days prior to enrollment

  • quality-of-life assessmentProcedure or surgery

    Start of each cycle

Study groups in NCT00520767
Study groupRoleWhat they receive
Melphalan, Dexamethasone, Bortezomib,ExperimentalBortezomib 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
Receives: Drug: bortezomib; Drug: dexamethasone; Drug: melphalan; Genetic: microarray analysis; Other: flow cytometry; Other: laboratory biomarker analysis; Procedure: quality-of-life assessment

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Complete Hematologic Response

    Measured: Up to 12 months

Secondary outcomes (4)
  1. Overall Survival

    time from day of registration until day of death.

    Measured: time from day of registration until 72 months.

  2. Time to Treatment Failure (TTF)

    Time from start of treatment until date of documented disease progression, removal from protocol due to toxicity, or death from any cause.

    Measured: start of treatment until 72 months

  3. Organ Response Rate (OrR)

    Measured: Beginning of cycles 4, 8, 12, 16 and 20, at follow up and end of study.

  4. Overall Hematologic Response Rate (OHR)

    Measured: Beginning of cycles 4, 8, 12, 16 and 20, at follow up and end of study.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Aug 2007 (estimated)
  2. Study startSep 2007
  3. Primary completionOct 2010
  4. Study completion6 Jun 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
35 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00520767
Sponsor's protocol ID
2006-132
Other IDs
P30CA022453, MILLENNIUM-WSU-2006-132, WSU-HIC-060907M1F
Status last verified
Dec 2023
First submitted
24 Aug 2007
Last updated
22 Dec 2023
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing bortezomib, dexamethasone and melphalan in people with Primary Systemic Amyloidosis and Light Chain Deposition Disease, run by Barbara Ann Karmanos Cancer Institute at 7 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 22 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 30 Mar 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Complete Hematologic Response (time frame: Up to 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Dec 2023; AzTrials last refreshed its copy on 7 Nov 2026.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in