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NCT02303821 · OUTCOME

Phase 2: Percentage of Participants With Complete Remission (CR) After Induction Therapy

Study of Carfilzomib in Combination With Induction Chemotherapy in Children With Relapsed or Refractory Acute Lymphoblastic Leukemia · Source last updated 2025-06-04

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 Day 50 (28-day cycle of induction therapy + recovery window from Day 29 to Day 45 [Day 36 to Day 50 for infants])

What was measured

CR was defined as: 1. Attainment of M1 bone marrow status (less than 5% blasts in a bone marrow aspirate and at least 200 cells counted) with no evidence of circulating blasts or extramedullary disease. 2. Recovery of peripheral counts: * Absolute neutrophil count (ANC) greater than or equal to 1000/µL * Platelet count greater than or equal to 100000/µL. * Assessed between days 29 and 45 Data was adjusted as inverse probability of treatment weight (IPTW) for the average treatment effect of the treated (IPTW-ATTW).

Analysis population: Phase 2: Participants enrolled in 20140106, in the PAS, who received at least 1 dose of carfilzomib.

Groups in this outcome

Phase 2: B-cell (20/56 mg/m^2)

Eligible participants with relapsed or refractory ALL (B-cell, after having received a targeted B-cell therapy) were treated with VXLD plus carfilzomib 20/56 mg/m\^2 for 1 cycle (induction), followed by assessment of treatment response. Participants who did not show progression during induction underwent a bone marrow and extramedullary disease evaluation after completion of induction therapy. Participants without disease progression after induction could at the investigator's discretion, be treated with 1 28-day consolidation cycle of BFM therapy (cyclophosphamide, cytarabine, 6-mercaptopurine, PEG-asparaginase, vincristine), and IT therapy plus carfilzomib.

Phase 2: T-cell (20/56 mg/m^2)

Eligible participants with relapsed or refractory ALL (T-cell) were treated with VXLD plus carfilzomib 20/56 mg/m\^2 for 1 cycle (induction), followed by assessment of treatment response. Participants who did not show progression during induction underwent a bone marrow and extramedullary disease evaluation after completion of induction therapy. Participants without disease progression after induction could at the investigator's discretion, be treated with 1 28-day consolidation cycle of BFM therapy (cyclophosphamide, cytarabine, 6-mercaptopurine, PEG-asparaginase, vincristine), and IT therapy plus carfilzomib.

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 2: B-cell (20/56 mg/m^2)61
Phase 2: T-cell (20/56 mg/m^2)44

Reported measurements

Source class 1
Values in Percentage of Participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Phase 2: B-cell (20/56 mg/m^2)14.8Not reported5.923.7Not reported
Phase 2: T-cell (20/56 mg/m^2)13.6Not reported3.523.8Not reported

Source statistical analyses

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

  1. group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    OTHER
    other Analysis Description
    The primary objective of this endpoint was to compare the percentage of participants achieving CR after the end of induction therapy in study 20140106 with the percentage of participants achieving CR in an external control arm selected from an observational study (Amgen 20180065). In the external control arm, 7.8% of B-Cell participants (95% confidence interval \[CI\]: 1.0%, 14.7%) achieved CR, with a treatment difference odds ratio of 2.04 (95% CI: 0.54, 7.66). For T-Cell participants, 9.1% (95% CI: 0.7%, 17.5%) achieved CR, with an odds ratio of 1.58 (95% CI: 0.47, 5.31).
Complete source fields
analyses
  1. group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    OTHER
    other Analysis Description
    The primary objective of this endpoint was to compare the percentage of participants achieving CR after the end of induction therapy in study 20140106 with the percentage of participants achieving CR in an external control arm selected from an observational study (Amgen 20180065). In the external control arm, 7.8% of B-Cell participants (95% confidence interval \[CI\]: 1.0%, 14.7%) achieved CR, with a treatment difference odds ratio of 2.04 (95% CI: 0.54, 7.66). For T-Cell participants, 9.1% (95% CI: 0.7%, 17.5%) achieved CR, with an odds ratio of 1.58 (95% CI: 0.47, 5.31).
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        5.9
        upper Limit
        23.7
        value
        14.8
      2. group Id
        OG001
        lower Limit
        3.5
        upper Limit
        23.8
        value
        13.6
denoms
  1. counts
    1. group Id
      OG000
      value
      61
    2. group Id
      OG001
      value
      44
    units
    Participants
description
CR was defined as: 1. Attainment of M1 bone marrow status (less than 5% blasts in a bone marrow aspirate and at least 200 cells counted) with no evidence of circulating blasts or extramedullary disease. 2. Recovery of peripheral counts: * Absolute neutrophil count (ANC) greater than or equal to 1000/µL * Platelet count greater than or equal to 100000/µL. * Assessed between days 29 and 45 Data was adjusted as inverse probability of treatment weight (IPTW) for the average treatment effect of the treated (IPTW-ATTW).
dispersion Type
95% Confidence Interval
groups
  1. description
    Eligible participants with relapsed or refractory ALL (B-cell, after having received a targeted B-cell therapy) were treated with VXLD plus carfilzomib 20/56 mg/m\^2 for 1 cycle (induction), followed by assessment of treatment response. Participants who did not show progression during induction underwent a bone marrow and extramedullary disease evaluation after completion of induction therapy. Participants without disease progression after induction could at the investigator's discretion, be treated with 1 28-day consolidation cycle of BFM therapy (cyclophosphamide, cytarabine, 6-mercaptopurine, PEG-asparaginase, vincristine), and IT therapy plus carfilzomib.
    id
    OG000
    title
    Phase 2: B-cell (20/56 mg/m^2)
  2. description
    Eligible participants with relapsed or refractory ALL (T-cell) were treated with VXLD plus carfilzomib 20/56 mg/m\^2 for 1 cycle (induction), followed by assessment of treatment response. Participants who did not show progression during induction underwent a bone marrow and extramedullary disease evaluation after completion of induction therapy. Participants without disease progression after induction could at the investigator's discretion, be treated with 1 28-day consolidation cycle of BFM therapy (cyclophosphamide, cytarabine, 6-mercaptopurine, PEG-asparaginase, vincristine), and IT therapy plus carfilzomib.
    id
    OG001
    title
    Phase 2: T-cell (20/56 mg/m^2)
param Type
NUMBER
population Description
Phase 2: Participants enrolled in 20140106, in the PAS, who received at least 1 dose of carfilzomib.
reporting Status
POSTED
time Frame
Up to Day 50 (28-day cycle of induction therapy + recovery window from Day 29 to Day 45 [Day 36 to Day 50 for infants])
title
Phase 2: Percentage of Participants With Complete Remission (CR) After Induction Therapy
type
PRIMARY
unit Of Measure
Percentage of Participants

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Preserved source evidence · Not medical advice