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NCT01125293 · OUTCOME

Very-good-partial-response-or-better Rate [Phase II]

Everolimus, Bortezomib and/or Rituximab in Patients With Relapsed/Refractory Waldenstrom's Macroglobulinemia · Source last updated 2021-04-23

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
NUMBER
Unit
percentage of participants
Interval / dispersion
95% Confidence Interval
Time frame
Up to 6 cycles (Day 168)

What was measured

Very-good-partial-response-or-better rate is the percentage of participants with complete response (CR) or very good partial response (VGPR) on to the combination of everolimus/bortezomib/rituximab. The combination regimen was received for up to 6 cycles. CR: * Absence of serum monoclonal IgM protein by immunofixation * Normal serum IgM level * Complete resolution of extramedullary disease, i.e., lymphadenopathy and splenomegaly if present at baseline * Morphologically normal bone marrow aspirate and trephine biopsy VGPR: * Monoclonal IgM protein is detectable ≥90% reduction in serum IgM level from baseline\* * Complete resolution of extramedullary disease, i.e.

Groups in this outcome

Phase II

Combination of everolimus \& rituximab with bortezomib for 6 cycles Everolimus 10 mg: Taken orally on a daily basis x 28 days (1 cycle) Rituximab 375 mg/Kg: Given intravenously on days 1, 8, 15 and 22 of Cycle 1 and Cycle 4 only (1 cycle = 28 days) Bortezomib 1.6 mg/m\^2: Given intravenously on days 1, 8 and 15 of every cycle (1 cycle = 28 days)

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
Phase II23

Reported measurements

Source class 1
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Phase II4.3Not reported019Not reported

Source statistical analyses

Group order, estimate direction, methods and comments are retained. No treatment ranking is inferred.

  1. group Description
    In a two-stage Simon design, a VGPR or better rate of at least 18% is considered promising versus a 5% or less rate. In stage 1, if 1 or fewer of 23 evaluable participants achieve VGPR, the regimen is considered non-promising else continue with 24 more patients enrolled. If \</=4 of 47 evaluable patients have VGPR or better, the regimen is considered non-promising. If \>/=5, then the regimen is considered promising for further study. With this design, there is 90% power and 1-sided 10% alpha.
    group Ids
    1. OG000
    non Inferiority Type
    OTHER
    other Analysis Description
    The study did not continue to stage 2 given 1 VGPR or better response was observed in 23 evaluable participants in stage 1.
    p Value
    0.69
    statistical Method
    Two stage design, exact method
Complete source fields
analyses
  1. group Description
    In a two-stage Simon design, a VGPR or better rate of at least 18% is considered promising versus a 5% or less rate. In stage 1, if 1 or fewer of 23 evaluable participants achieve VGPR, the regimen is considered non-promising else continue with 24 more patients enrolled. If \</=4 of 47 evaluable patients have VGPR or better, the regimen is considered non-promising. If \>/=5, then the regimen is considered promising for further study. With this design, there is 90% power and 1-sided 10% alpha.
    group Ids
    1. OG000
    non Inferiority Type
    OTHER
    other Analysis Description
    The study did not continue to stage 2 given 1 VGPR or better response was observed in 23 evaluable participants in stage 1.
    p Value
    0.69
    statistical Method
    Two stage design, exact method
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        0
        upper Limit
        19
        value
        4.3
denoms
  1. counts
    1. group Id
      OG000
      value
      23
    units
    Participants
description
Very-good-partial-response-or-better rate is the percentage of participants with complete response (CR) or very good partial response (VGPR) on to the combination of everolimus/bortezomib/rituximab. The combination regimen was received for up to 6 cycles. CR: * Absence of serum monoclonal IgM protein by immunofixation * Normal serum IgM level * Complete resolution of extramedullary disease, i.e., lymphadenopathy and splenomegaly if present at baseline * Morphologically normal bone marrow aspirate and trephine biopsy VGPR: * Monoclonal IgM protein is detectable ≥90% reduction in serum IgM level from baseline\* * Complete resolution of extramedullary disease, i.e.
dispersion Type
95% Confidence Interval
groups
  1. description
    Combination of everolimus \& rituximab with bortezomib for 6 cycles Everolimus 10 mg: Taken orally on a daily basis x 28 days (1 cycle) Rituximab 375 mg/Kg: Given intravenously on days 1, 8, 15 and 22 of Cycle 1 and Cycle 4 only (1 cycle = 28 days) Bortezomib 1.6 mg/m\^2: Given intravenously on days 1, 8 and 15 of every cycle (1 cycle = 28 days)
    id
    OG000
    title
    Phase II
param Type
NUMBER
reporting Status
POSTED
time Frame
Up to 6 cycles (Day 168)
title
Very-good-partial-response-or-better Rate [Phase II]
type
PRIMARY
unit Of Measure
percentage of participants

Download exact source JSON → · Snapshot ctgov-results-25a395029948b04d52b1253f

Preserved source evidence · Not medical advice