AzTrials
AzTrials

Light Chain Deposition DiseasePhase 3NCT01078454

Melphalan and Dexamethasone With or Without Bortezomib in Treating Patients With Previously Untreated Systemic Light-Chain Amyloidosis

A Phase 3 study testing melphalan, dexamethasone and bortezomib in people with Light Chain Deposition Disease and Primary Systemic Amyloidosis, run by National Cancer Institute (NCI) at 209 sites in United States.

Completed

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

Official record
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Trial summary

This randomized phase III trial is studying melphalan and dexamethasone to see how well they work with or without bortezomib in treating patients with previously untreated systemic amyloidosis. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
melphalan, dexamethasone and bortezomib
Could I receive a placebo?
No, everyone receives an active treatment
How many people
11 participants
Where
209 sitesUnited States
Study dates
Nov 2010to 27 Nov 2012
Phase
Phase 3
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Histologically confirmed diagnosis of systemic light-chain amyloidosis
  • Histologic diagnosis of disease must be confirmed by pathology (positive Congo red stain with green birefringence on polarized light microscopy)
  • Genetic testing must be negative for transthyretin mutations associated with hereditary amyloidosis (required in patients who are African-American or who present with peripheral neuropathy as the dominant organ involvement)
  • Measurable disease, defined by >= 1 of the following:
  • Serum M-protein >= 1 g/dL by serum protein electrophoresis (SPEP)
  • Difference between involved and uninvolved free light chain be >4.0mg/dL provided the kappa to lambda free light chain (FLC) ratio is abnormal
Show all 38Show fewer
  • Symptomatic organ involvement* (heart, kidney, liver/gastrointestinal tract, peripheral nervous system, or soft tissue), defined as any of the following:
  • NOTE: *Carpal tunnel syndrome skin purpura or the presence of vascular amyloid on a bone marrow biopsy alone are not sufficient to meet criteria for "symptomatic organ involvement"
  • Renal involvement is defined as proteinuria (predominantly albumin) > 0.5 g/day by 24-hour urine collection
  • Cardiac involvement is defined as the presence of a mean left ventricular wall thickness of > 12 mm by echocardiogram in the absence of a history of hypertension or valvular heart disease or in the presence of unexplained low voltage (< 0.5 mV) by electrocardiogram
  • Hepatic involvement is defined as hepatomegaly or an alkaline phosphatase > 1.5 times upper limit of normal (ULN)
  • Peripheral nerve involvement is defined by clinical history or abnormal sensory and/or motor findings on neurologic exam
  • Gastrointestinal (GI) involvement is defined as gross GI bleeding or diarrhea (at least 4 stools per day over baseline); a positive GI biopsy is not sufficient to document clinical involvement
  • Autonomic nerve involvement is defined as orthostasis, symptoms of nausea or dysgeusia, gastric atony by gastric emptying scan, diarrhea, or constipation
  • Soft tissue and lymphatic involvement may be ascertained based on classic physical exam findings (macroglossia, shoulder pad sign, raccoon eyes, carpal tunnel syndrome, synovial enlargement, firm enlarged lymph nodes) or biopsy
  • Ineligible for autologous stem cell transplantation with melphalan 200 mg/m^2 or refuses to undergo transplantation
  • Eastern Cooperative Oncology Group (ECOG) performance status 0-2
  • Amyloid cardiac biomarker stage I or II disease
  • The amyloid cardiac staging system is based on NT-proBNP and troponin-T levels. If troponin T (cTnT) is not available at local institution then troponin I (cTnI) may be used. Thresholds for cTnT, cTnI, and NT-proBNP are < 0.035 ug/L, < 0.1 ug/L, and < 332 ng/L, respectively. Stage I patients have both troponin-T (or I) and NT-proBNP below the threshold. Stage II patients have either troponin-T (I) or NT-proBNP above the threshold. Stage III patients have troponin-T (or I) and simultaneous NT-proBNP above the threshold. Stage III patients are further classified as "better risk" if NT-proBNP is over 332 ng/L but less than 6000 ng/L
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • The absence of supine systolic blood pressure < 100 mmHg and difficult to manage symptomatic orthostatic hypotension
  • Absolute neutrophil count (ANC) > 1,500/mm^3
  • Platelet count > 140,000/mm^3
  • Hemoglobin > 10 g/dL
  • Total bilirubin < 2.5 mg/dL
  • Alkaline phosphatase < 5 times upper limit of normal (ULN)
  • Aspartate aminotransferase (AST) < 3 times ULN
  • Creatinine clearance > 30 mL/min
  • Bone marrow plasma cells < 30%
  • Human immunodeficiency virus (HIV)-positivity allowed provided the following criteria are met:
  • No history of acquired immunodeficiency syndrome (AIDS)-defining events including history of CD4 cell count < 200/mm^3
  • Current CD4 cell count >= 350/mm^3
  • Not receiving zidovudine or stavudine
  • No secondary amyloidosis
  • More than 3 weeks since radiotherapy
  • Enrollment of subjects who require radiotherapy (which must be localized in field size) should be deferred until the radiotherapy is completed and 3 weeks have elapsed since the last date of therapy
  • More than 14 days since prior and no concurrent participation in clinical trials with other investigational agents not included in this trial

Who cannot join

  • Pregnant or nursing
  • Clinically overt myeloma (hypercalcemia or lytic bone lesions)
  • Prior chemotherapy or radiotherapy for the treatment of myeloma or systemic light-chain amyloidosis
  • History of sustained ventricular tachycardias
  • Cardiac syncope
  • Uncompensated New York Heart Association (NYHA) class III or IV congestive heart failure
Show all 11Show fewer
  • Uncontrolled infection
  • Active malignancy within the past 5 years except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or adequately treated stage I cancer currently in complete remission
  • Serious medical or psychiatric illness likely to interfere with study participation, including recent myocardial infarction (within the past 6 months) or poorly controlled diabetes mellitus
  • Hypersensitivity to bortezomib, boron, or mannitol
  • Grade 2 or higher peripheral neuropathy

Trial locations and contacts

209 sites in 1 country.

209 sites in 1 country

United States209 sites

  • Oncare Hawaii Inc-Pali Momi

    ‘Aiea, Hawaii, 96701

  • Pali Momi Medical Center

    ‘Aiea, Hawaii, 96701

  • McFarland Clinic PC-William R Bliss Cancer Center

    Ames, Iowa, 50010

  • Cancer Care Center at Island Hospital

    Anacortes, Washington, 98221

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • Emory University/Winship Cancer Institute

    Atlanta, Georgia, 30322

  • The Medical Center of Aurora

    Aurora, Colorado, 80012

  • Saint Francis Hospital and Health Centers

    Beech Grove, Indiana, 46107

  • PeaceHealth Saint Joseph Medical Center

    Bellingham, Washington, 98225

  • Montana Cancer Consortium CCOP

    Billings, Montana, 59101

  • Saint Vincent Healthcare

    Billings, Montana, 59101

  • Hematology-Oncology Centers of the Northern Rockies PC

    Billings, Montana, 59102

  • Billings Clinic

    Billings, Montana, 59107-7000

  • Illinois CancerCare-Bloomington

    Bloomington, Illinois, 61701

  • Saint Joseph Medical Center

    Bloomington, Illinois, 61701

  • Tufts Medical Center

    Boston, Massachusetts, 02111

  • Boston Medical Center

    Boston, Massachusetts, 02118

  • Boulder Community Hospital

    Boulder, Colorado, 80301

  • Bozeman Deaconess Cancer Center

    Bozeman, Montana, 59715

  • Bozeman Deaconess Hospital

    Bozeman, Montana, 59715

  • Harrison HealthPartners Hematology and Oncology-Bremerton

    Bremerton, Washington, 98310

  • Highline Medical Center-Main Campus

    Burien, Washington, 98166

  • Fairview Ridges Hospital

    Burnsville, Minnesota, 55337

  • Saint James Community Hospital and Cancer Treatment Center

    Butte, Montana, 59701

  • Cooper Hospital University Medical Center

    Camden, New Jersey, 08103

  • Graham Hospital Association

    Canton, Illinois, 61520

  • Illinois CancerCare-Canton

    Canton, Illinois, 61520

  • Illinois CancerCare-Carthage

    Carthage, Illinois, 62321

  • Memorial Hospital

    Carthage, Illinois, 62321

  • Rocky Mountain Oncology

    Casper, Wyoming, 82609

  • Marshfield Clinic-Chippewa Center

    Chippewa Falls, Wisconsin, 54729

  • The Jewish Hospital

    Cincinnati, Ohio, 45236

  • Clackamas Radiation Oncology Center

    Clackamas, Oregon, 97015

  • Case Western Reserve University

    Cleveland, Ohio, 44106

  • MetroHealth Medical Center

    Cleveland, Ohio, 44109

  • Penrose-Saint Francis Healthcare

    Colorado Springs, Colorado, 80907

  • Arthur G. James Cancer Hospital and Solove Research Institute at Ohio State University Medical Center

    Columbus, Ohio, 43210

  • Mercy Hospital

    Coon Rapids, Minnesota, 55433

  • Geisinger Medical Center

    Danville, Pennsylvania, 17822-2001

  • Grandview Hospital

    Dayton, Ohio, 45405

  • Good Samaritan Hospital - Dayton

    Dayton, Ohio, 45406

  • Miami Valley Hospital

    Dayton, Ohio, 45409

  • Samaritan North Health Center

    Dayton, Ohio, 45415

  • Dayton CCOP

    Dayton, Ohio, 45420

  • Decatur Memorial Hospital

    Decatur, Illinois, 62526

  • Porter Adventist Hospital

    Denver, Colorado, 80210

  • Exempla Saint Joseph Hospital

    Denver, Colorado, 80218

  • Presbyterian - Saint Lukes Medical Center - Health One

    Denver, Colorado, 80218

  • Rose Medical Center

    Denver, Colorado, 80220

  • Colorado Cancer Research Program CCOP

    Denver, Colorado, 80224-2522

  • Wayne State University/Karmanos Cancer Institute

    Detroit, Michigan, 48201

  • Marshfield Clinic Cancer Center at Sacred Heart

    Eau Claire, Wisconsin, 54701

  • Fairview-Southdale Hospital

    Edina, Minnesota, 55435

  • Union Hospital of Cecil County

    Elkton, Maryland, 21921

  • Swedish Medical Center

    Englewood, Colorado, 80113

  • Green Bay Oncology - Escanaba

    Escanaba, Michigan, 49431

  • Eureka Hospital

    Eureka, Illinois, 61530

  • Illinois CancerCare-Eureka

    Eureka, Illinois, 61530

  • Blanchard Valley Hospital

    Findlay, Ohio, 45840

  • Atrium Medical Center-Middletown Regional Hospital

    Franklin, Ohio, 45005-1066

  • Unity Hospital

    Fridley, Minnesota, 55432

  • Illinois CancerCare Galesburg

    Galesburg, Illinois, 61401

  • Saint Mary's Hospital and Regional Medical Center

    Grand Junction, Colorado, 81502

  • Benefis Healthcare- Sletten Cancer Institute

    Great Falls, Montana, 59405

  • Great Falls Clinic

    Great Falls, Montana, 59405

  • North Colorado Medical Center

    Greeley, Colorado, 80631

  • Green Bay Oncology at Saint Vincent Hospital

    Green Bay, Wisconsin, 54301-3526

  • Saint Vincent Hospital

    Green Bay, Wisconsin, 54301

  • Green Bay Oncology Limited at Saint Mary's Hospital

    Green Bay, Wisconsin, 54303

  • Saint Mary's Hospital

    Green Bay, Wisconsin, 54303

  • Wayne Hospital

    Greenville, Ohio, 45331

  • Illinois CancerCare-Havana

    Havana, Illinois, 62644

  • Mason District Hospital

    Havana, Illinois, 62644

  • Northern Montana Hospital

    Havre, Montana, 59501

  • Geisinger Medical Center-Cancer Center Hazelton

    Hazleton, Pennsylvania, 18201

  • Saint Peter's Community Hospital

    Helena, Montana, 59601

  • Oncare Hawaii Inc-POB II

    Honolulu, Hawaii, 96813

  • Queen's Medical Center

    Honolulu, Hawaii, 96813

  • Straub Clinic and Hospital

    Honolulu, Hawaii, 96813

  • University of Hawaii

    Honolulu, Hawaii, 96813

  • OnCare Hawaii-Liliha

    Honolulu, Hawaii, 96817-3169

  • Kuakini Medical Center

    Honolulu, Hawaii, 96817

  • Oncare Hawaii Inc-Kuakini

    Honolulu, Hawaii, 96817

  • Kapiolani Medical Center for Women and Children

    Honolulu, Hawaii, 96826

  • Hutchinson Area Health Care

    Hutchinson, Minnesota, 55350

  • Indiana University Medical Center

    Indianapolis, Indiana, 46202

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242

  • Green Bay Oncology - Iron Mountain

    Iron Mountain, Michigan, 49801

  • Swedish Cancer Institute-Issaquah

    Issaquah, Washington, 98029

  • University of Mississippi Medical Center

    Jackson, Mississippi, 39216

  • Mayo Clinic in Florida

    Jacksonville, Florida, 32224-9980

  • Castle Medical Center

    Kailua, Hawaii, 96734

  • Borgess Medical Center

    Kalamazoo, Michigan, 49001

  • Bronson Methodist Hospital

    Kalamazoo, Michigan, 49007

  • West Michigan Cancer Center

    Kalamazoo, Michigan, 49007

  • Glacier Oncology PLLC

    Kalispell, Montana, 59901

  • Kalispell Medical Oncology

    Kalispell, Montana, 59901

  • Kalispell Regional Medical Center

    Kalispell, Montana, 59901

  • Columbia Basin Hematology and Oncology PLLC

    Kennewick, Washington, 99336

  • Kettering Medical Center

    Kettering, Ohio, 45429

  • Illinois CancerCare-Kewanee Clinic

    Kewanee, Illinois, 61443

  • Gundersen Lutheran

    La Crosse, Wisconsin, 54601

  • Saint Anthony Hospital

    Lakewood, Colorado, 80228

  • Beebe Medical Center

    Lewes, Delaware, 19958

  • Wilcox Memorial Hospital and Kauai Medical Clinic

    Lihue, Hawaii, 96766

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72205

  • Littleton Adventist Hospital

    Littleton, Colorado, 80122

  • Sky Ridge Medical Center

    Lone Tree, Colorado, 80124

  • Longmont United Hospital

    Longmont, Colorado, 80501

  • McKee Medical Center

    Loveland, Colorado, 80539

  • Illinois CancerCare-Macomb

    Macomb, Illinois, 61455

  • Mcdonough District Hospital

    Macomb, Illinois, 61455

  • Holy Family Memorial Hospital

    Manitowoc, Wisconsin, 54221

  • Minnesota Oncology Hematology PA-Maplewood

    Maplewood, Minnesota, 55109

  • Saint John's Hospital - Healtheast

    Maplewood, Minnesota, 55109

  • Bay Area Medical Center

    Marinette, Wisconsin, 54143

  • Marshfield Clinic

    Marshfield, Wisconsin, 54449

  • Providence Milwaukie Hospital

    Milwaukie, Oregon, 97222

  • Abbott-Northwestern Hospital

    Minneapolis, Minnesota, 55407

  • Hennepin County Medical Center

    Minneapolis, Minnesota, 55415

  • Marshfield Clinic-Minocqua Center

    Minocqua, Wisconsin, 54548

  • Montana Cancer Specialists

    Missoula, Montana, 59802

  • Saint Patrick Hospital - Community Hospital

    Missoula, Montana, 59802

  • Holy Family Medical Center

    Monmouth, Illinois, 61462

  • Illinois CancerCare-Monmouth

    Monmouth, Illinois, 61462

  • Skagit Valley Hospital

    Mount Vernon, Washington, 98274

  • D N Greenwald Center

    Mukwonago, Wisconsin, 53149

  • New Ulm Medical Center

    New Ulm, Minnesota, 56073

  • Christiana Care Health System-Christiana Hospital

    Newark, Delaware, 19718

  • Providence Newberg Medical Center

    Newberg, Oregon, 97132

  • Bromenn Regional Medical Center

    Normal, Illinois, 61761

  • Community Cancer Center Foundation

    Normal, Illinois, 61761

  • Illinois CancerCare-Community Cancer Center

    Normal, Illinois, 61761

  • Eastern Connecticut Hematology and Oncology Associates

    Norwich, Connecticut, 06360

  • Oconomowoc Memorial Hospital-ProHealth Care Inc

    Oconomowoc, Wisconsin, 53066-3896

  • Green Bay Oncology - Oconto Falls

    Oconto Falls, Wisconsin, 54154

  • Providence Willamette Falls Medical Center

    Oregon City, Oregon, 97045

  • Illinois CancerCare-Ottawa Clinic

    Ottawa, Illinois, 61350

  • Ottawa Regional Hospital and Healthcare Center

    Ottawa, Illinois, 61350

  • Parker Adventist Hospital

    Parker, Colorado, 80138

  • Pekin Cancer Treatment Center

    Pekin, Illinois, 61554

  • Illinois CancerCare-Pekin

    Pekin, Illinois, 61603

  • Methodist Medical Center of Illinois

    Peoria, Illinois, 61603

  • Proctor Hospital

    Peoria, Illinois, 61614

  • Illinois CancerCare-Peoria

    Peoria, Illinois, 61615

  • Illinois Oncology Research Association CCOP

    Peoria, Illinois, 61615

  • OSF Saint Francis Medical Center

    Peoria, Illinois, 61637

  • Illinois CancerCare-Peru

    Peru, Illinois, 61354

  • Illinois Valley Hospital

    Peru, Illinois, 61354

  • Fox Chase Cancer Center

    Philadelphia, Pennsylvania, 19111

  • Providence Portland Medical Center

    Portland, Oregon, 97213

  • Western Oncology Research Consortium

    Portland, Oregon, 97213

  • Adventist Medical Center

    Portland, Oregon, 97216

  • Providence Saint Vincent Medical Center

    Portland, Oregon, 97225

  • Harrison HealthPartners Hematology and Oncology-Poulsbo

    Poulsbo, Washington, 98370

  • Illinois CancerCare-Princeton

    Princeton, Illinois, 61356

  • Perry Memorial Hospital

    Princeton, Illinois, 61356

  • Saint Mary Corwin Medical Center

    Pueblo, Colorado, 81004

  • Marshfield Clinic at James Beck Cancer Center

    Rhinelander, Wisconsin, 54501

  • Marshfield Clinic-Rice Lake Center

    Rice Lake, Wisconsin, 54868

  • Reid Hospital and Health Care Services

    Richmond, Indiana, 47374

  • North Memorial Medical Health Center

    Robbinsdale, Minnesota, 55422

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • Metro-Minnesota CCOP

    Saint Louis Park, Minnesota, 55416

  • Park Nicollet Clinic - Saint Louis Park

    Saint Louis Park, Minnesota, 55416

  • Regions Hospital

    Saint Paul, Minnesota, 55101

  • United Hospital

    Saint Paul, Minnesota, 55102

  • Mayo Clinic in Arizona

    Scottsdale, Arizona, 85259

  • Harborview Medical Center

    Seattle, Washington, 98104

  • Minor and James Medical PLLC

    Seattle, Washington, 98104

  • Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

    Seattle, Washington, 98109

  • Group Health Cooperative-Seattle

    Seattle, Washington, 98112

  • Swedish Medical Center-First Hill

    Seattle, Washington, 98122-4307

  • The Polyclinic

    Seattle, Washington, 98122

  • University of Washington Medical Center

    Seattle, Washington, 98195

  • United General Hospital

    Sedro-Woolley, Washington, 98284

  • Saint Francis Regional Medical Center

    Shakopee, Minnesota, 55379

  • Saint Nicholas Hospital

    Sheboygan, Wisconsin, 53081

  • Welch Cancer Center

    Sheridan, Wyoming, 82801

  • Siouxland Hematology Oncology Associates

    Sioux City, Iowa, 51101

  • Mercy Medical Center-Sioux City

    Sioux City, Iowa, 51104

  • Saint Luke's Regional Medical Center

    Sioux City, Iowa, 51104

  • Providence Hospital

    Southfield, Michigan, 48075

  • Cancer Care Northwest - Spokane South

    Spokane, Washington, 99202

  • Evergreen Hematology and Oncology PS

    Spokane, Washington, 99218

  • Illinois CancerCare-Spring Valley

    Spring Valley, Illinois, 61362

  • Memorial Medical Center

    Springfield, Illinois, 62781-0001

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • Geisinger Medical Group

    State College, Pennsylvania, 16801

  • Marshfield Clinic Cancer Care at Saint Michael's Hospital

    Stevens Point, Wisconsin, 54481

  • Lakeview Hospital

    Stillwater, Minnesota, 55082

  • Green Bay Oncology - Sturgeon Bay

    Sturgeon Bay, Wisconsin, 54235

  • North Suburban Medical Center

    Thornton, Colorado, 80229

  • Upper Valley Medical Center

    Troy, Ohio, 45373

  • PeaceHealth Southwest Medical Center

    Vancouver, Washington, 98664

  • Compass Oncology Vancouver

    Vancouver, Washington, 98684

  • Ridgeview Medical Center

    Waconia, Minnesota, 55387

  • Waukesha Memorial Hospital - ProHealth Care

    Waukesha, Wisconsin, 53188

  • Wenatchee Valley Medical Center

    Wenatchee, Washington, 98801

  • Cleveland Clinic Florida - Weston

    Weston, Florida, 33331

  • Diagnostic and Treatment Center

    Weston, Wisconsin, 54476

  • Marshfield Clinic - Weston Center

    Weston, Wisconsin, 54476

  • Exempla Lutheran Medical Center

    Wheat Ridge, Colorado, 80033

  • Geisinger Wyoming Valley

    Wilkes-Barre, Pennsylvania, 18711

  • Rice Memorial Hospital

    Willmar, Minnesota, 56201

  • Clinton Memorial Hospital

    Wilmington, Ohio, 45177

  • Marshfield Clinic - Wisconsin Rapids Center

    Wisconsin Rapids, Wisconsin, 54494

  • Minnesota Oncology and Hematology PA-Woodbury

    Woodbury, Minnesota, 55125

  • Greene Memorial Hospital

    Xenia, Ohio, 45385

Other trials in United States

About this study

Official title: A Randomized Phase III Trial of Melphalan and Dexamethasone (MDex) Versus Bortezomib, Melphalan and Dexamethasone (BMDex) for Untreated Patients With Systemic Light-Chain (AL) Amyloidosis Ineligible for Autologous Stem-Cell Transplantation

This randomized phase III trial is studying melphalan and dexamethasone to see how well they work with or without bortezomib in treating patients with previously untreated systemic amyloidosis. Drugs used in chemotherapy, such as melphalan and dexamethasone, work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Bortezomib may stop the growth of plasma cells by blocking some of the enzymes needed for cell growth. It is not yet known whether giving melphalan together with dexamethasone is more effective with or without bortezomib in treating systemic amyloidosis.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To compare hematologic overall response (partial response [PR], very good PR, amyloid complete hematologic response [ACR], and stringent complete response [sCR]) after 3 courses of therapy in patients with previously untreated systemic light-chain amyloidosis treated with melphalan and dexamethasone with vs without bortezomib.

SECONDARY OBJECTIVES:

  1. To evaluate the ACR rate after 3 courses of therapy and at completion of therapy.

II. To evaluate organ response rates after 3 courses of therapy and at 6, 9, and 12 months.

III. To evaluate treatment-related mortality.

IV. To evaluate toxicity.

  1. To evaluate progression-free and overall survival.

VI. To evaluate PR or better at completion of therapy.

VII. To evaluate time to hematologic and organ response.

VIII. To evaluate the duration of hematologic and organ response.

IX. To assess quality of life (QOL) at baseline, at 3, 6, and 9 months during the therapy, at completion of therapy, and 3 and 6 months after therapy.

TERTIARY OBJECTIVES:

  1. To determine the prognostic impact of t(11;14) translocation and cyclin D1 overexpression on response and overall survival.

II. (Correlative) To compare sCR rates and to determine the impact of sCR on the outcomes.

III. (Correlative) To perform a descriptive analysis of amyloid typing and proteomic composition of amyloid tissues.

OUTLINE: This is a multicenter study. Patients are stratified according to amyloid cardiac stage (stage I/II vs. better risk stage III) and are randomized to 1 of 2 treatment arms.

ARM A (Mel-Dex): Patients receive melphalan 0.22 mg/kg orally (PO) and dexamethasone 40 mg PO on days 1-4 every 4 weeks. Treatment repeats every 4 weeks for up to 9 courses in the absence of disease progression or unacceptable toxicity.

ARM B (B-Mel-Dex): Patients receive melphalan 0.22 mg/kg PO and dexamethasone 40 mg PO on days 1-4 and bortezomib 1.3 mg/m2 intravenously (IV) on days 1, 4, 8, and 11 every 4 weeks. Treatment repeats every 4 weeks for 2 cycles. Patients then receive melphalan PO and dexamethasone PO on days 1-4 and bortezomib IV on days 1, 8, 15, and 22 every 5 weeks. Treatment repeats every 5 weeks for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

Blood, urine, bone marrow, and fat samples may be collected periodically for laboratory analysis. Health-related quality of life is assessed periodically before, during, and after therapy. After completion of study treatment, patients are followed up periodically for 5 years.

Keywords
Melphalan, Dexamethasone, Bortezomib, Systemic Light-chain (AL) Amyloidosis
Medical subject headings
Immunoglobulin Light-chain Amyloidosis, Amyloidosis

What participants receive

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

  • melphalanDrug

    Also called L-PAM, L-phenylalanine mustard, L-sarcolysin, Alkeran

    Given PO

  • dexamethasoneDrug

    Also called Decadron, Hexadrol, Dexameth, Dexone

    Given PO

  • bortezomibDrug

    Also called Velcade®, PS-341, MLN-341, LDP-341

    Given IV

Study groups in NCT01078454
Study groupRoleWhat they receive
ARM A (Mel-Dex)ExperimentalPatients receive melphalan 0.22 mg/kg orally (PO) and dexamethasone 40 mg PO on days 1-4 every 4 weeks. Treatment repeats every 4 weeks for up to 9 courses in the absence of disease progression or unacceptable toxicity.
Receives: Drug: melphalan; Drug: dexamethasone
ARM B (B-Mel-Dex)ExperimentalPatients receive melphalan 0.22 mg/kg PO and dexamethasone 40 mg PO on days 1-4 and bortezomib 1.3 mg/m^2 intravenously (IV) on days 1, 4, 8, and 11 every 4 weeks. Treatment repeats every 4 weeks for 2 cycles. Patients then receive melphalan PO and dexamethasone PO on days 1-4 and bortezomib IV on days 1, 8, 15, and 22 every 5 weeks. Treatment repeats every 5 weeks for up to 6 cycles in the absence of disease progression or unacceptable toxicity.
Receives: Drug: melphalan; Drug: dexamethasone; Drug: bortezomib

What is being measured

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

  1. Proportion of Patients With Hematologic Overall Response (Partial Response [PR]+ Very Good PR [VGPR]+ Amyloid Complete Response [ACR]+ Stringent Complete Response [sCR]) After 3 Months (3 Cycles) of Therapy

    sCR: ACR and no clonal cells in bone marrow (BM) ACR: Negative serum/urine immunofixation (IF), <5% plasma cells in BM, and normal serum FLC ratio VGPR: 1. PR and any of the following; 2. serum/urine M-protein detectable by IF but not measurable (NM) on electrophoresis (EP); (3) ≥90% reduction in serum M-component and urine M-protein <100 mg/24 hr if baseline serum measurable; (4) urine M-component <100 mg/24 hr and NM serum M-protein on serum protein EP if baseline urine measurable; (5) ≥90% drop in the difference between involved and uninvolved FLC levels if only FLC measurable PR: (1) ≥50% drop of serum M-protein and 24-hr urinary M-protein drop by ≥90% or to <200 mg/24 hr if baseline serum/urine measurable; or (2) ≥50% drop of serum M-protein if only serum measurable at baseline; or (3) 24-hr urinary M-protein drop by ≥90% or to <200 mg/24 hr if baseline urine measurable; or (4) ≥ 50% drop in the difference between involved and uninvolved FLC if only FLC measu

    Measured: Assessed at 3 months

Timeline

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

  1. First posted2 Mar 2010 (estimated)
  2. Study startNov 2010
  3. Primary completion12 Nov 2012
  4. Study completion27 Nov 2012

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 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
11 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01078454
Sponsor's protocol ID
NCI-2011-02010
Other IDs
NCI-2011-02010, E4A08, U10CA021115
Status last verified
Jan 2022
First submitted
27 Feb 2010
Last updated
1 Feb 2022
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing melphalan, dexamethasone and bortezomib in people with Light Chain Deposition Disease and Primary Systemic Amyloidosis, run by National Cancer Institute (NCI) at 209 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?

The sponsor lists 38 inclusion and 11 exclusion criteria. 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 1 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 209 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 26 Nov 2014). 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?

Proportion of Patients With Hematologic Overall Response (Partial Response [PR]+ Very Good PR [VGPR]+ Amyloid Complete Response [ACR]+ Stringent Complete Response [sCR]) After 3 Months (3 Cycles) of Therapy (time frame: Assessed at 3 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01078454.

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 1 Feb 2022.

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