Clinical trial NCT02416206: High Dose Chemotherapy Using BeEAM for Autologous Transplant in Multiple Myeloma
Official title: A Phase II Trial of High-dose Bendamustine, Etoposide, Cytarabine, and Melphalan (BeEAM) in the Up-front Treatment of Multiple Myeloma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Multiple Myeloma
Keywords: Multiple myeloma
hematopoietic stem cell transplant
Lead sponsor: Northside Hospital, Inc.
Sponsor class: OTHER
Collaborators: name: Teva Pharmaceuticals USA
First posted: 2015-04-14
Enrollment: 65
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: High-dose chemotherapy and autologous stem cell transplantation (ASCT) as part of the up-front treatment of patients with multiple myeloma has been associated with improved disease-free and overall survival in multiple large randomized controlled trials. Following 3-6 cycles of standard induction therapy with biologic agents, consolidation with high dose Melphalan and ASCT has become the standard-of-care approach for fit myeloma patients up to 70 years of age. Single-agent high-dose Melphalan (200mg/m2) is currently the standard-of-care preparative regimen prior to autologous transplant in Myeloma. Historical studies utilizing Busulfan- or Total Body Irradiation-based preparative regimens have yielded similar results to single-agent Melphalan with higher toxicity.

Detailed description: Myeloma patients, following up-front induction therapy, will receive an ASCT following a high-dose bendamustine-based preparative regimen (BeEAM). The primary endpoint of this trial will be the rate of CR at day 100 post-transplant. Experience from the literature, as well as results from our institution, suggests that following ASCT for the upfront treatment of myeloma, the rate of CR at day 100 post-transplant is approximately 45%. It is hoped that under this protocol, this rate will be at least 65%. Thus we statistically formalize this study by testing the null hypothesis that p, the CR rate is 0.65 or more versus the alternative hypothesis that p is less than 0.45. A sample size of 65 pts gives 90% power with an alpha=0.05, using the formula for a one sample binomial (two-sided) test of a proportion.

Eligibility criteria: Inclusion Criteria:

* Age between 18 - 70 years
* Karnofsky status ≥ 70%
* Diagnosis of Multiple Myeloma
* Within 9 months of the start of induction chemotherapy and no evidence of relapse or progression.
* Availability of Cryopreserved peripheral blood stem cells with a CD34 dose of at least 2x106/kg.

Exclusion Criteria:

* Poor cardiac function: left ventricular ejection fraction \<40%
* Poor pulmonary function: FEV1, FVC, or DLCO \<40% predicted
* Poor liver function: bilirubin \>2.5 mg/dl (not due to hemolysis, Gilbert's or primary malignancy), AST/ALT \> 3X ULN
* Poor renal function: Creatinine \>2.0 mg/dl or creatinine clearance \< 40 mL/min (calculated creatinine clearance is permitted)
* Ongoing or active systemic infection, active hepatitis B or C virus infection, or known human immunodeficiency virus (HIV) positive.
* Women of childbearing potential who currently are pregnant or who are not practicing adequate contraception
* Patients who have any debilitating medical or psychiatric illness which would preclude their giving informed consent or their receiving optimal treatment and follow-up.

Primary outcomes:
measure: To Estimate the Response at Day 100 Following Transplant (Rate of CR)
timeFrame: Day 100
Secondary outcomes:
measure: Number of Patients With Overall Survival Post-transplant
timeFrame: 3 years
measure: Number of Patients With Progression-free Survival
timeFrame: 3 years
measure: Number of Patients Who Relapsed After Transplant
timeFrame: 3 years
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Blood and Marrow Transplant Group of Georgia
state: Georgia
Study officials:
affiliation: Northside Hospital
name: Scott R Solomon, MD
role: PRINCIPAL_INVESTIGATOR
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