NCT01413178

Brief Summary

The goal of this clinical research study is to compare Busulfex (busulfan) with or without Alkeran (melphalan) to learn which study therapy may be better at helping to control MM in patients who will receive an autologous stem cell transplant. The safety of this combination therapy will also be studied. Melphalan and busulfan are designed to damage the DNA (genetic material) of cells, which may cause cancer cells to die.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
205

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Sep 2011

Longer than P75 for phase_3

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 8, 2011

Completed
2 days until next milestone

First Posted

Study publicly available on registry

August 10, 2011

Completed
2 months until next milestone

Study Start

First participant enrolled

September 30, 2011

Completed
7.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 10, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 10, 2019

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

April 21, 2020

Completed
Last Updated

April 21, 2020

Status Verified

April 1, 2020

Enrollment Period

7.4 years

First QC Date

August 8, 2011

Results QC Date

February 17, 2020

Last Update Submit

April 7, 2020

Conditions

Keywords

MyelomaMultiple MyelomaLight chain Multiple MyelomaMMAutologous Hematopoietic Stem Cell TransplantationBusulfanBusulfexMyleranMelphalanAlkeranG-CSFFilgrastimNeupogenTMQuestionnaireSurveyQuality of LifeQOL

Outcome Measures

Primary Outcomes (1)

  • Progression-Free Survival (PFS)

    Participants that are still alive and without Multiple Myeloma 3 years after Stem cell Transplantation.

    3 years after transplant

Secondary Outcomes (4)

  • Number of Participants With Complete Response (CR)

    Evaluated 90 days from transplant.

  • Treatment-Related Mortality (TRM) Between 2 Arms.

    100 days post treatment

  • Number of Participants That Had Grade 3-4 Toxicities.

    At day 90 post SCT (Stem Cell Transplantation)

  • Overall Survival (OS)

    From time of ASCT to 3 years

Study Arms (2)

Busulfan + Melphalan

EXPERIMENTAL

Busulfan test dose (32 mg/m\^2) on day -9 then 130 mg/m\^2 intravenous (IV) Days -7, -6, -5, and -4 + Melphalan 70 mg/m2 IV on Days -2 and -1. Stem Cell Transplant (SCT) Day 0.

Drug: BusulfanDrug: MelphalanOther: QuestionnaireDrug: G-CSFProcedure: Stem cell transplant

Melphalan

EXPERIMENTAL

High-dose Melphalan 200 mg/m2/day IV over 30 minutes on day -2. SCT Day 0.

Other: QuestionnaireDrug: G-CSFDrug: High Dose MelphalanProcedure: Stem cell transplant

Interventions

Test dose (32 mg/m\^2) on day -9 then 130 mg/m\^2 by vein or adjusted dose on Days -7, -6, -5, and -4.

Also known as: Busulfex, Myleran
Busulfan + Melphalan

70 mg/m2 by vein over 30 minutes minutes on Days -2 and -1.

Also known as: Alkeran
Busulfan + Melphalan

Quality of Life (QOL) questionnaire before starting the study drugs and then once every 4 weeks after the stem cell transplant, taking about 15 minutes to complete.

Also known as: Survey
Busulfan + MelphalanMelphalan
G-CSFDRUG

Approximately 5 mcg/kg/day subcutaneously beginning on Day +5.

Also known as: Filgrastim, NeupogenTM
Busulfan + MelphalanMelphalan

200 mg/m2 by vein over 30 minutes on Day -2.

Also known as: Alkeran
Melphalan

Stem cell infusion on Day 0.

Also known as: ABMT, autologous bone marrow transplantation, Peripheral Blood Progenitor Cells, PBPCs
Busulfan + MelphalanMelphalan

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patients with multiple myeloma in complete remission (CR), partial remission (PR), or very good partial remission (VGPR), or symptomatic stable disease (no evidence of progression) including patients with light chain MM detected in the serum by free light chain assay.
  • Patients with non-secretory multiple myeloma \[absence of a monoclonal protein (M protein) in serum as measured by electrophoresis (SPEP) and immunofixation (SIFE) and the absence of Bence Jones protein in the urine (UPEP) defined by use of conventional electrophoresis and immunofixation (UIFE) techniques\] but with measurable disease on imaging studies like MRI, CT scan or PET scan.
  • Who have received at least two cycles of initial systemic therapy and are within 2 to 12 months of the first dose. Mobilization therapy is not considered initial therapy.
  • years of age or younger.
  • Karnofsky performance score 70% or higher.
  • Cardiac function: left ventricular ejection fraction at rest \> 40% within 3 months of registration.
  • Hepatic function: bilirubin \< 2x the upper limit of normal and ALT and AST \< 2.5x the upper limit of normal.
  • Renal function: creatinine clearance of \>/= 40 mL/min, estimated or calculated.
  • Pulmonary function: DLCO, FEV1, FVC \>/= 50% of predicted value (corrected for hemoglobin) within 3 months of registration
  • Signed informed consent form.

You may not qualify if:

  • Patients with uncontrolled bacterial, viral or fungal infections (currently taking medication and progression of clinical symptoms).
  • Patients seropositive for the human immunodeficiency virus (HIV).
  • Patients with history of 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.
  • Patients participating in an investigational new drug protocol within 14 days before enrollment.
  • Female patients who are pregnant (positive b-HCG) or breastfeeding.
  • Prior stem cell transplantation allogeneic or autologous.
  • Prior organ transplant requiring immunosuppressive therapy.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Publications (1)

  • Bashir Q, Thall PF, Milton DR, Fox PS, Kawedia JD, Kebriaei P, Shah N, Patel K, Andersson BS, Nieto YL, Valdez BC, Parmar S, Rondon G, Delgado R, Hosing C, Popat UR, Oran B, Ciurea SO, Lin P, Weber DM, Thomas SK, Lee HC, Manasanch EE, Orlowski RZ, Williams LA, Champlin RE, Qazilbash MH. Conditioning with busulfan plus melphalan versus melphalan alone before autologous haemopoietic cell transplantation for multiple myeloma: an open-label, randomised, phase 3 trial. Lancet Haematol. 2019 May;6(5):e266-e275. doi: 10.1016/S2352-3026(19)30023-7. Epub 2019 Mar 22.

Related Links

MeSH Terms

Conditions

Neoplasms, Plasma CellMultiple Myeloma

Interventions

BusulfanMelphalanSurveys and QuestionnairesGranulocyte Colony-Stimulating FactorFilgrastimStem Cell Transplantation

Condition Hierarchy (Ancestors)

Neoplasms by Histologic TypeNeoplasmsHemostatic DisordersVascular DiseasesCardiovascular DiseasesParaproteinemiasBlood Protein DisordersHematologic DiseasesHemic and Lymphatic DiseasesHemorrhagic DisordersLymphoproliferative DisordersImmunoproliferative DisordersImmune System Diseases

Intervention Hierarchy (Ancestors)

Butylene GlycolsGlycolsAlcoholsOrganic ChemicalsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicHydrocarbonsSulfonic AcidsSulfur AcidsSulfur CompoundsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedPhenylalanineAmino Acids, AromaticAmino Acids, CyclicAmino AcidsAmino Acids, Peptides, and ProteinsData CollectionEpidemiologic MethodsInvestigative TechniquesHealth Care Evaluation MechanismsQuality of Health CareHealth Care Quality, Access, and EvaluationPublic HealthEnvironment and Public HealthColony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesProteinsBiological FactorsCell TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, Operative

Results Point of Contact

Title
Dr. Muzaffar H. Qazilbash / Stem Cell Transplantation and Cellular Therapy
Organization
U.T. MD Anderson Cancer Center

Study Officials

  • Muzaffar H. Qazilbash, MD

    M.D. Anderson Cancer Center

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 8, 2011

First Posted

August 10, 2011

Study Start

September 30, 2011

Primary Completion

March 10, 2019

Study Completion

March 10, 2019

Last Updated

April 21, 2020

Results First Posted

April 21, 2020

Record last verified: 2020-04

Locations