Phase 3
Completed N=205
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
Source: ClinicalTrials.gov NCT01413178 ↗Enrolled (actual)
205
Serious AEs
52.0%
Results posted
Apr 2020
Primary outcomePrimary: Progression-Free Survival (PFS) — 72; 50 Participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Progression-Free Survival (PFS) |
72; 50 | — |
| SECONDARY Number of Participants With Complete Response (CR) |
12; 15 | — |
| SECONDARY Treatment-Related Mortality (TRM) Between 2 Arms. |
0; 0 | — |
| SECONDARY Number of Participants That Had Grade 3-4 Toxicities. |
5; 4; 3; 0; 2; 0 | — |
| SECONDARY Overall Survival (OS) |
91; 79 | — |
Eligibility Criteria
Inclusion Criteria
- 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.
- 70 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 /= 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.
Exclusion Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT01413178). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.