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Multiple MyelomaPhase 3NCT01109004

Stem Cell Transplant With Lenalidomide Maintenance in Patients With Multiple Myeloma (BMT CTN 0702)

A Phase 3 study testing Lenalidomide and lenalidomide, bortezomib and dexamethasone in people with Multiple Myeloma, run by National Heart, Lung, and Blood Institute (NHLBI) at 54 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

The study is designed as a Phase III, multicenter trial of tandem autologous transplants plus maintenance therapy versus the strategy of single autologous transplant plus consolidation therapy with lenalidomide, bortezomib and dexamethasone (RVD) followed by maintenance therapy or single autologous transplant plus… Read more about this study

Who is it for?
Ages Up to 70 years
Conditions the study is for
Multiple Myeloma
What is being tested?
Lenalidomide and lenalidomide, bortezomib and dexamethasone
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
758 participants
Where
54 sitesUnited States
Study dates
May 2010to 3 Mar 2018
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 Up to 70 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Patients meeting the criteria for symptomatic multiple myeloma (MM).
  • Patients who are 70 years of age, or younger, at time of enrollment.
  • Patients who have received at least two cycles of any regimen as initial systemic therapy and are within 2 - 12 months of the first dose of initial therapy.
  • Cardiac function: left ventricular ejection fraction at rest greater than 40 percent.
  • Hepatic: bilirubin less than 1.5x the upper limit of normal and alanine aminotransferase (ALT) and aspartate aminotransferase (AST) less than 2.5x the upper limit of normal. (Patients who have been diagnosed with Gilbert's Disease are allowed to exceed the defined bilirubin value of 1.5x the upper limit of normal.)
  • Renal: Creatinine clearance of grater than or equal to 40 mL/min, estimated or calculated.
Show all 9Show fewer
  • Pulmonary: Diffusing capacity of the lung for carbon monoxide (DLCO), forced expiratory volume in one second (FEV1), or forced vital capacity (FVC) greater than 50 percent of predicted value (corrected for hemoglobin).
  • Patients with an adequate autologous graft defined as a cryopreserved PBSC graft containing greater than or equal to 4 x 10^6 CD34+ cells/kg patient weight. The graft may not be CD34+ selected or otherwise manipulated to remove tumor or other cells. The graft can be collected at the transplanting institution or by a referring center. The autograft must be stored so that there are two products each containing at least 2 x 10^6 CD34+ cells/kg patient weight.
  • Signed informed consent form.

Who cannot join

  • Patients who never fulfill the criteria for symptomatic MM.
  • Patients with purely non-secretory MM [absence of a monoclonal protein (M protein) in serum as measured by electrophoresis and immunofixation and the absence of Bence Jones protein in the urine defined by use of conventional electrophoresis and immunofixation techniques]. Patients with light chain MM detected in the serum by free light chain assay are eligible.
  • Patients with plasma cell leukemia.
  • Karnofsky performance score less than 70 percent.
  • Patients with greater than grade 2 sensory neuropathy (CTCAE).
  • Patients with uncontrolled bacterial, viral or fungal infections (currently taking medication and progression of clinical symptoms).
Show all 23Show fewer
  • Patients seropositive for the human immunodeficiency virus (HIV).
  • Myocardial infarction within 6 months prior to enrollment or has New York Heart Association (NYHA) Class III or IV heart failure, 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.
  • Patient has hypersensitivity to bortezomib, boron or mannitol.
  • Patient has received other investigational drugs with 14 days before enrollment.
  • Patients with prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ. Cancer treated with curative intent less than 5 years previously will not be allowed unless approved by the Protocol Officer or one of the Protocol Chairs. Cancer treated with curative intent greater than 5 years previously is allowed.
  • Female patients who are pregnant (positive B-HCG) or breastfeeding.
  • Females of childbearing potential (FCBP) or men who have sexual contact with FCBP unwilling to use contraceptive techniques during the length of lenalidomide maintenance therapy.
  • Prior allograft or prior autograft.
  • Patients who have received mid-intensity melphalan (greater than 50 mg IV) as part of prior therapy.
  • Patients unable or unwilling to provide informed consent.
  • Prior organ transplant requiring immunosuppressive therapy.
  • Patients with disease progression prior to enrollment.
  • Patients who have received lenalidomide as initial therapy for MM and have experienced toxicities resulting in treatment discontinuation.
  • Patients who experienced thromboembolic events while on full anticoagulation during prior therapy with lenalidomide or thalidomide.
  • Patients unwilling to take deep vein thrombosis (DVT) prophylaxis.
  • Patients who cannot undergo an intervention in any treatment arm due to a priori denial of medical costs coverage by third party payers.
  • Patients unable to unwilling to return to the transplant center for their assigned treatments.

Trial locations and contacts

54 sites in 1 country.

54 sites in 1 country

United States54 sites

  • University of Michigan Medical Center

    Ann Arbor, Michigan, 48105-2967

  • Blood and Marrow Transplant Program at Northside Hospital

    Atlanta, Georgia, 30342

  • Georgia Health Sciences University

    Augusta, Georgia, 30912

  • St. Lukes Mountain States Tumor Institute

    Boise, Idaho, 83712

  • DFCI, Brigham and Womens Hospital

    Boston, Massachusetts, 02114

  • DFCI, Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Roswell Park Cancer Center

    Buffalo, New York, 14263

  • University of North Carolina Hospital at Chapel Hill

    Chapel Hill, North Carolina, 27599-7305

  • Rush University Medical Center

    Chicago, Illinois, 60612

  • University of Illinois

    Chicago, Illinois, 60612

  • Jewish Hospital BMT Program

    Cincinnati, Ohio, 45236

  • University Hospitals of Cleveland

    Cleveland, Ohio, 44106-5061

  • Ohio State/Arthur G. James Cancer Hospital

    Columbus, Ohio, 43210

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390

  • Colorado Blood Cancer Institute

    Denver, Colorado, 80218

  • Karmanos Cancer Institute/BMT

    Detroit, Michigan, 48201

  • City of Hope National Medical Center

    Duarte, California, 91010-3000

  • Duke University Medical Center

    Durham, North Carolina, 27705

  • University of Florida College of Medicine

    Gainesville, Florida, 32610

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • Penn State College of Medicine, The Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033

  • Baylor College of Medicine/The Methodist Hospital

    Houston, Texas, 77030

  • University of Texas, MD Anderson CRC

    Houston, Texas, 77030

  • University of Kansas Hospital

    Kansas City, Kansas, 66160

  • Thompson Cancer Survival Center

    Knoxville, Tennessee, 37916

  • UCSD Medical Center

    La Jolla, California, 92093

  • North Shore University Hospital

    Lake Success, New York, 11042

  • University of Kentucky

    Lexington, Kentucky, 40536

  • University of Wisconsin Hospital & Clinics

    Madison, Wisconsin, 53792-5156

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53211

  • University of Minnesota

    Minneapolis, Minnesota, 55455

  • West Virginia University Hospital

    Morgantown, West Virginia, 26506

  • Sarah Cannon Blood & Marrow Transplant Program

    Nashville, Tennessee, 37203

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232-8210

  • Mount Sinai Medical Center

    New York, New York, 10029

  • Memorial Sloan-Kettering Cancer Center

    New York, New York, 10174

  • Christiana Care Health System

    Newark, Delaware, 19718

  • University of Oklahoma Medical Center

    Oklahoma City, Oklahoma, 73104

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198-7680

  • Florida Hospital Cancer Institute

    Orlando, Florida, 32804

  • Advocate Lutheran General Hospital

    Park Ridge, Illinois, 60068

  • University of Pennsylvania Cancer Center

    Philadelphia, Pennsylvania, 19104

  • Oregon Health & Science University

    Portland, Oregon, 97239-3098

  • University of Rochester Medical Center

    Rochester, New York, 14642

  • Texas Transplant Institute

    San Antonio, Texas, 78229

  • University of California, San Francisco

    San Francisco, California, 94143-0324

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington, 98109-1024

  • Louisiana State University Health Sciences Center

    Shreveport, Louisiana, 71130

  • Washington University, Barnes Jewish Hospital

    St Louis, Missouri, 63110

  • Stanford Hospital and Clinics

    Stanford, California, 94305

  • H. Lee Moffitt Cancer Center

    Tampa, Florida, 33624

  • Arizona Cancer Center

    Tucson, Arizona, 85724

  • Wichita CCOP

    Wichita, Kansas, 67214

  • Wake Forest University Health Sciences

    Winston-Salem, North Carolina, 27157

Other trials in United States

About this study

Official title: A Trial of Single Autologous Transplant With or Without Consolidation Therapy Versus Tandem Autologous Transplant With Lenalidomide Maintenance for Patients With Multiple Myeloma (BMT CTN 0702)

The study is designed as a Phase III, multicenter trial of tandem autologous transplants plus maintenance therapy versus the strategy of single autologous transplant plus consolidation therapy with lenalidomide, bortezomib and dexamethasone (RVD) followed by maintenance therapy or single autologous transplant plus maintenance therapy as part of upfront treatment of multiple myeloma (MM). Lenalidomide will be used as maintenance therapy for three years in all arms.

Read the detailed description

The primary objective of the randomized trial is to compare three-year progression-free survival (PFS) between the three treatment arms as a pairwise comparison. Mobilization therapy will not be specified for the study. Randomization to three treatment arms will be done prior to the first transplants. All patients will undergo a first autologous peripheral blood stem cell (PBSC) transplant with high-dose melphalan (200 mg/m^2 IV) given on Day -2. Upon recovery from the first transplant patients will receive either a second autologous PBSC transplant with the same conditioning regimen as the first transplant or consolidation therapy with RVD (lenalidomide 15 mg/day on Days 1-14, dexamethasone 40 mg on Days 1, 8 and 15, and bortezomib 1.3mg/m^2 on Days 1, 4, 8 and 11 of every 21 day cycle, patients will receive four cycles) or maintenance with lenalidomide (15 mg daily). All patients will also receive maintenance lenalidomide which will start after the second transplant, after the first autologous transplant or after consolidation therapy depending on the treatment arm. Maintenance therapy with lenalidomide will start at 10 mg daily for three months and increase to 15 mg daily. The duration of maintenance will be three years in all treatment arms.

Being tested
Lenalidomide, lenalidomide, bortezomib and dexamethasone
Keywords
Symptomatic Multiple Myeloma, Lenalidomide, Anti-Myeloma Agents, Hematologic Disorders, Maintenance Therapy, Progression, Autologous Transplant, RVD Consolidation
Medical subject headings
Multiple Myeloma, Hematologic Diseases, Disease Progression

What participants receive

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

  • LenalidomideDrug

    Also called Revlimid™

    All patients will undergo a first autologous peripheral blood stem cell (PBSC) transplant with high-dose melphalan (200 mg/m^2 IV) given on Day -2. Upon recovery from the first transplant patients will receive a second autologous PBSC transplant with the same conditioning regimen as the first transplant. All patients will also receive maintenance lenalidomide which will start after the second transplant. Maintenance therapy with lenalidomide will start at 10 mg daily for 3 months and increase to 15 mg daily. The duration of maintenance will be three years in all treatment arms.

  • lenalidomide, bortezomib and dexamethasoneDrug

    Also called Revlimid™, Velcade®, and Decadron

    All patients will undergo a first autologous peripheral blood stem cell (PBSC) transplant with high-dose melphalan (200 mg/m^2 IV) given on Day -2. Upon recovery from the first transplant patients will receive consolidation therapy with RVD (lenalidomide 15 mg/day on Days 1-14, dexamethasone 40mg on Days 1, 8 and 15, and bortezomib 1.3mg/m2 on Days 1, 4, 8 and 11 of every 21 day cycle, patients will receive four cycles). All patients will also receive maintenance lenalidomide which will start after consolidation therapy. Maintenance therapy with lenalidomide will start at 10 mg daily for 3 months and increase to 15 mg daily. The duration of maintenance will be three years in all treatment arms.

  • LenalidomideDrug

    Also called Revlimid™

    All patients will undergo a first autologous peripheral blood stem cell (PBSC) transplant with high-dose melphalan (200 mg/m^2 IV) given on Day -2. Upon recovery from the first transplant patients will receive maintenance with lenalidomide (15 mg daily). Maintenance lenalidomide will start after the first autologous transplant. Maintenance therapy with lenalidomide will start at 10 mg daily for 3 months and increase to 15 mg daily. The duration of maintenance will be three years in all treatment arms.

Study groups in NCT01109004
Study groupRoleWhat they receive
Tandem auto transplantActive comparatorInitial autologous transplant followed by a second autologous transplant and lenalidomide maintenance
Receives: Drug: Lenalidomide
RVD consolidationActive comparatorInitial autologous transplant followed by lenalidomide, bortezomib and dexamethasone (RVD) consolidation and lenalidomide maintenance
Receives: Drug: lenalidomide, bortezomib and dexamethasone
Lenalidomide maintenanceActive comparatorInitial autologous transplant followed by lenalidomide maintenance
Receives: Drug: Lenalidomide

What is being measured

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

  1. Percentage of Participants With Progression-free Survival (PFS)

    Progression-free survival is defined as survival without disease progression or initiation of non-protocol anti-myeloma therapy. To account for loss to follow-up of a few participants, the Kaplan-Meier estimator was used to estimate progression-free survival at 38 months post-randomization.

    Measured: 38 months post-randomization

Secondary outcomes (9)

  1. Percentage of Participants With Disease Progression

    Disease Progression is defined as progression of multiple myeloma, including one or more of the following:

    • A reappearance of serum monoclonal paraprotein, with a level of at least 0.5 g/dL
    • 24-hour urine protein electrophoresis with at least 200 mg paraprotein/24 hours
    • Abnormal free light chain levels of >10 mg/dl, only in patients without measurable paraprotein in the serum and urine
    • At least 10% plasma cells in a bone marrow aspirate or on trephine biopsy
    • Definite increase in the size of existing bone lesions or soft tissue plasmacytomas
    • Development of new bone lesions or soft tissue plasmacytomas
    • Development of hypercalcemia (corrected serum Ca >11.5 mg/dL or >2.8 mmol/L) not attributable to any other cause

    To account for loss to follow-up of a few participants, the cumulative incidence of TRM at 38 months post-randomization was estimated using the Aalen-Johansen estimator, treating death prior to disease progression as a competing risk.

    Measured: 38 months post-randomization

  2. Percentage of Participants With Overall Survival (OS)

    Overall survival is defined as survival of death from any cause. To account for loss to follow-up of a few participants, the Kaplan-Meier estimator was used to estimate overall survival at 38 months post-randomization.

    Measured: 38 months post-randomization

  3. Percentage of Participants With Treatment-related Mortality (TRM)

    TRM is defined as death prior to progression of multiple myeloma. To account for loss to follow-up of a few participants, the cumulative incidence of TRM at 38 months post-randomization was estimated using the Aalen-Johansen estimator, treating disease progression as a competing risk.

    Measured: Up to 38 months post-randomization

  4. Number of Participants With Treatment Response

    The number of participants with very good partial response (VGPR) or better [complete response (CR), near CR (nCR), and stringent CR (sCR)] according to the International Uniform Response Criteria will be calculated. The "Worse than VGPR" group includes PR, stable disease, and progressive disease.

    sCR requires, in addition to CR: Normal free light chain ratio (FLC), Absence of clonal cells in bone marrow CR requires, in addition to nCR: Absence of the original monoclonal paraprotein (PPN), Disappearance of soft tissue plasmacytomas nCR is defined as: < 5% plasma cells in a bone marrow aspirate, No increase in lytic bone lesions VGPR requires: Serum or urine PPN not detectable on electrophoresis OR >=90% reduction in serum PPN plus urine PPN <100 mg/24hrs, >= 50% reduction in the level of serum monoclonal PPN or reduction in 24 hour urinary monoclonal PPN either >= 90% or to <200 mg/24 hours in light chain disease, >= 50% reduction in the size of soft tissue plasmacytomas

    Measured: 1 and 2 years post-randomization

  5. FACT-G Total Score

    The Functional Assessment of Cancer Therapy-General (FACT-G) is a quality of life instrument that assesses the effects of cancer therapy on a patient's physical, social/family, emotional, and functional well-being. The assessment has 27 questions, each scored on a Likert scale from 0-4. The overall score is computed by adding scores of the questions and falls in the range 0-108, with higher scores indicating higher levels of overall well-being.

    Measured: Up to 3 years post-randomization

  6. FACT-BMT Score

    The Functional Assessment of Cancer Therapy-Bone Marrow Transplant scale (FACT-BMT) is a quality of life instrument that assesses the effects of bone marrow transplantation (BMT) on a patient's physical, social/family, emotional, and functional well-being while taking into consideration BMT-specific concerns. The assessment has 37 questions, each scored on a Likert scale from 0-4. The overall score is computed by adding scores of the questions and falls in the range 0-148, with higher scores indicating higher levels of overall well-being.

    Measured: Up to 3 years post-randomization

  7. FACT-BMT Trial Outcome Index

    The Functional Assessment of Cancer Therapy (FACT) Trial Outcome Index is a quality of life instrument that assesses the impact of bone marrow transplantation (BMT) on a patient's physical and functional well-being while taking into consideration BMT-specific concerns. The assessment has 24 questions, each scored on a Likert scale from 0-4. The overall score is computed by adding scores of the questions and falls in the range 0-96, with higher scores indicating higher levels of overall well-being.

    Measured: Up to 3 years post-randomization

  8. MOS SF-36 Physical Component Summary

    The Medical Outcome Study (MOS) SF-36 Physical Component Summary is a subscale of the SF-36 intended to measure physical well-being. It is scored on a scale of 0-100, with higher scores indicating higher levels of well-being.

    Measured: Up to 3 years post-randomization

  9. MOS SF-36 Mental Component Summary

    The Medical Outcome Study (MOS) SF-36 Mental Component Summary is a subscale of the SF-36 intended to measure mental well-being. It is scored on a scale of 0-100, with higher scores indicating higher levels of well-being.

    Measured: Up to 3 years post-randomization

Timeline

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

  1. First posted22 Apr 2010 (estimated)
  2. Study startMay 2010
  3. Primary completion15 Jan 2017
  4. Study completion3 Mar 2018

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
758 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01109004
Sponsor's protocol ID
BMTCTN0702
Other IDs
BMT CTN 0702, U01HL069294-05, 690
Status last verified
Dec 2021
First submitted
21 Apr 2010
Last updated
9 Dec 2021
Data monitoring committee
Yes
Individual data sharing
Yes: Results will be published in a manuscript and supporting information submitted to NIH BioLINCC (including data dictionaries, case report forms, data submission documentation, documentation for outcomes dataset, etc where indicated).

Publications

  • Stadtmauer EA, Pasquini MC, Blackwell B, Hari P, Bashey A, Devine S, Efebera Y, Ganguly S, Gasparetto C, Geller N, Horowitz MM, Koreth J, Knust K, Landau H, Brunstein C, McCarthy P, Nelson C, Qazilbash MH, Shah N, Vesole DH, Vij R, Vogl DT, Giralt S, Somlo G, Krishnan A. Autologous Transplantation, Consolidation, and Maintenance Therapy in Multiple Myeloma: Results of the BMT CTN 0702 Trial. J Clin Oncol. 2019 Mar 1;37(7):589-597. doi: 10.1200/JCO.18.00685. Epub 2019 Jan 17.

    PubMed 30653422

  • Pasquini MC, Wallace PK, Logan B, Kaur M, Tario JD, Howard A, Zhang Y, Brunstein C, Efebera Y, Geller N, Giralt S, Hari P, Horowitz MM, Koreth J, Krishnan A, Landau H, Somlo G, Shah N, Stadtmauer E, Vogl DT, Vesole DH, McCarthy PL, Hahn T. Minimal Residual Disease Status in Multiple Myeloma 1 Year After Autologous Hematopoietic Cell Transplantation and Lenalidomide Maintenance Are Associated With Long-Term Overall Survival. J Clin Oncol. 2024 Aug 10;42(23):2757-2768. doi: 10.1200/JCO.23.00934. Epub 2024 May 3.

    PubMed 38701390

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Lenalidomide and lenalidomide, bortezomib and dexamethasone in people with Multiple Myeloma, run by National Heart, Lung, and Blood Institute (NHLBI) at 54 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 9 inclusion and 23 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 9 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 54 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 11 Jun 2018). 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?

Percentage of Participants With Progression-free Survival (PFS) (time frame: 38 months post-randomization). 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 NCT01109004, which also lists 2 linked publications.

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 9 Dec 2021.

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