Clinical trial NCT00378222: Autologous Transplantation for Multiple Myeloma
Official title: A Prospective, Randomized Study of Single Versus Double Autologous Stem Cell Transplantation for Multiple Myeloma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Multiple Myeloma
Keywords: Myeloma previously untreated
Single autologous transplantation
Double autologous transplantation
Complete response
Lead sponsor: University of Bologna
Sponsor class: OTHER
First posted: 2006-09-19
Enrollment: 324
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The present study was designed in an attempt to prospectively evaluate in a randomized fashion whether further cytotoxic dose intensification, as delivered with two sequential autologous stem-cell transplantations, improved the outcome of younger patients with newly diagnosed multiple myeloma in comparison with a single autologous transplantation.

Detailed description: Following demonstration that single autologous transplantation for the treatment of younger patients with newly diagnosed multiple myeloma prolonged overall survival in comparison with conventional chemotherapy, double autologous transplantation was tested, initially in refractory myeloma and subsequently to include also patients with newly diagnosed disease. To explore the role of double autologous stem-cell transplantation as part of up-front therapy for multiple myeloma, in 1996 we launched a prospective, randomized trial comparing a single course of stem-cell-supported melphalan with the same regimen followed, after three to six months, by a second autologous transplantation in support of melphalan and busulfan. The study was designed to detect a 15 percent increase in complete or near complete response rate with double transplants compared to a single transplantation. With a 2-sided significance level α = 0.05 and a power 1-β = 0.80, 162 patients were required in each treatment arm of the study to detect a statistically significant increase in complete or near complete response rate from 30% in the single-transplant arm to 45% in the double-transplant arm. Primary study endpoint was the complete or near complete response rate. Secondary study endpoints were relapse-free survival, event-free survival and overall survival. The recruitment target was 324 patients.

Eligibility criteria: Inclusion Criteria:

* Previously untreated
* Younger than 61 years
* Symptomatic myeloma
* Measurable disease
* Fit to receive high-dose chemotherapy

Exclusion Criteria:

* M-GUS
* Solitary plasmacytoma
* Plasma cell leukemia
* AL Amyloidosis

Primary outcomes:
measure: Complete or near Complete Response Rate
Secondary outcomes:
measure: Relapse-free Survival
measure: Event-free Survival
measure: Overall Survival
Other outcomes:
Locations:
Study officials:
affiliation: Institute of Hematology and Medical Oncology - University of Bologna
name: Michele Cavo, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Cavo M et al. Superiority of Double over Single Autologous Stem Cell Transplantation as First-Line Therapy for Multiple Myeloma. Blood (ASH Annual Meeting Abstracts) 2004 104: Abstract 536