Lenalidomide and Low Dose Dexamethasone Induction Therapy Followed by Low Dose Melphalan, Prednisone, Lenalidomide and Bortezomib Sequential Maintenance Therapy for Newly Diagnosed High-risk Multiple Myeloma
Study design: A total of 35 patients will be accrued to this Phase II trial over a period of
about 3 years. The primary objective is to estimate the proportion of patients who are still
progression-free at 2 years. Two years will be as measured from date of registration to the
trial Progression will include disease progression as well as death due to any cause. The
Garban et al. trial found a 2-year progression-free rate of about 0.60 in 212 similar
patients. If a 2-year rate of 0.60 were to be observed in this trial, we would consider the
treatment a success. A rate of 0.60 has an exact 80% confidence interval of 0.48 - 0.71.
In section 5.1 are given the probabilities of observing a 2-year progression-free rate ≥
0.60 under different assumptions about the true rate.
Analyses: The proportion of patients progression-free at the times of full restaging (i.e.,
at 6, 12, 18, 24, 36, 48, 60, 72 months) will be calculated with 80% confidence intervals
and descriptively compared to the rates in the Garban et al. paper. Time-to-progression will
be estimated with the Kaplan-Meier method. The percentage of patients achieving a sCR, CR,
VGPR or PR, will be tabulated, and the sCR+CR+VGPR+PR disease rate will be estimated with
exact 80% confidence interval. Duration of response among patients achieving a sCR, CR,
VGPR or PR, will be defined as the length of the interval from initial response to
progression; duration of response will be tabulated. All toxicities will be tabulated by
type and grade.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Evaluate the efficacy as measured by the progression free survival at 2 years
David Rizzieri, MD
United States: Institutional Review Board
|Duke University Medical Center||Durham, North Carolina 27710|