Busulfan Plus Melphalan Versus Melphalan

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Otsuka Pharmaceutical Development & Commercialization, Inc.
Information provided by (Responsible Party):
M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:
NCT01413178
First received: August 8, 2011
Last updated: May 23, 2014
Last verified: May 2014
  Purpose

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.


Condition Intervention Phase
Myeloma
Drug: Busulfan
Drug: Melphalan
Other: Questionnaire
Drug: G-CSF
Drug: High Dose Melphalan
Procedure: Stem cell transplant
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Randomized Trial to Compare Busulfan + Melphalan 140 mg/m2 With Melphalan 200 mg/m2 as Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation for Multiple Myeloma

Resource links provided by NLM:


Further study details as provided by M.D. Anderson Cancer Center:

Primary Outcome Measures:
  • Number of Participants with Complete Response (CR) [ Time Frame: Evaluated 90 days from transplant. ] [ Designated as safety issue: No ]
    Complete response (CR), evaluated 90 days from transplant, defined as (i) negative immunofixation of the multiple myeloma (MM) protein in urine and serum, (ii) disappearance of any soft tissue plasmacytomas, and (iii) less than 5% plasma MM cells in the bone marrow. International Myeloma Working Group uniform response criteria.


Estimated Enrollment: 190
Study Start Date: September 2011
Estimated Primary Completion Date: September 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Busulfan + Melphalan
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: Busulfan
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.
Other Names:
  • Busulfex
  • Myleran
Drug: Melphalan
70 mg/m2 by vein over 30 minutes minutes on Days -2 and -1.
Other Name: Alkeran
Other: Questionnaire
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.
Other Name: Survey
Drug: G-CSF
Approximately 5 mcg/kg/day subcutaneously beginning on Day +5.
Other Names:
  • Filgrastim
  • NeupogenTM
Procedure: Stem cell transplant
Stem cell infusion on Day 0.
Other Names:
  • ABMT
  • autologous bone marrow transplantation
  • Peripheral Blood Progenitor Cells
  • PBPCs
Experimental: Melphalan
High-dose Melphalan 200 mg/m2/day IV over 30 minutes on day -2. SCT Day 0.
Other: Questionnaire
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.
Other Name: Survey
Drug: G-CSF
Approximately 5 mcg/kg/day subcutaneously beginning on Day +5.
Other Names:
  • Filgrastim
  • NeupogenTM
Drug: High Dose Melphalan
200 mg/m2 by vein over 30 minutes on Day -2.
Other Name: Alkeran
Procedure: Stem cell transplant
Stem cell infusion on Day 0.
Other Names:
  • ABMT
  • autologous bone marrow transplantation
  • Peripheral Blood Progenitor Cells
  • PBPCs

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. 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.
  2. 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.
  3. 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.
  4. 70 years of age or younger.
  5. Karnofsky performance score 70% or higher.
  6. Cardiac function: left ventricular ejection fraction at rest > 40% within 3 months of registration.
  7. Hepatic function: bilirubin < 2x the upper limit of normal and ALT and AST < 2.5x the upper limit of normal.
  8. Renal function: creatinine clearance of >/= 40 mL/min, estimated or calculated.
  9. Pulmonary function: DLCO, FEV1, FVC > 50% of predicted value (corrected for hemoglobin) within 3 months of registration
  10. Signed informed consent form.

Exclusion Criteria:

  1. Patients with uncontrolled bacterial, viral or fungal infections (currently taking medication and progression of clinical symptoms).
  2. Patients seropositive for the human immunodeficiency virus (HIV).
  3. 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.
  4. Patients participating in an investigational new drug protocol within 14 days before enrollment.
  5. Female patients who are pregnant (positive b-HCG) or breastfeeding.
  6. Prior stem cell transplantation allogeneic or autologous.
  7. Prior organ transplant requiring immunosuppressive therapy.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01413178

Locations
United States, Texas
UT MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Otsuka Pharmaceutical Development & Commercialization, Inc.
Investigators
Principal Investigator: Muzaffar H. Qazilbash, MD UT MD Anderson Cancer Center
  More Information

Additional Information:
No publications provided

Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT01413178     History of Changes
Other Study ID Numbers: 2010-0071, NCI-2011-02760
Study First Received: August 8, 2011
Last Updated: May 23, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by M.D. Anderson Cancer Center:
Myeloma
Multiple Myeloma
Light chain Multiple Myeloma
MM
Autologous Hematopoietic Stem Cell Transplantation
Busulfan
Busulfex
Myleran
Melphalan
Alkeran
G-CSF
Filgrastim
NeupogenTM
Questionnaire
Survey
Quality of Life
QOL

Additional relevant MeSH terms:
Multiple Myeloma
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Neoplasms
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Busulfan
Melphalan
Lenograstim
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Antineoplastic Agents
Therapeutic Uses
Myeloablative Agonists
Adjuvants, Immunologic

ClinicalTrials.gov processed this record on August 18, 2014