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NCT02303821 · OUTCOME

Phase 2: Event Free Survival (EFS)

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
MEDIAN
Unit
Months
Interval / dispersion
95% Confidence Interval
Time frame
Up to approximately 2 years

What was measured

EFS was defined as time from initiation of therapy until treatment failure (defined as failure to reach at least a CRi after consolidation or after induction in participants that did not receive consolidation), relapse, or death from any cause. CRi was defined as: a. Attainment of M1 bone marrow status with no evidence of circulating blasts or extramedullary disease b. ANC and platelet counts not fulfilling criteria for CRh, CRp, or CR. Data was IPTW adjusted. Medians were estimated using the Kaplan-Meier (KM) method.

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 Months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Phase 2: B-cell (20/56 mg/m^2)1.18Not reported0.952.24Not reported
Phase 2: T-cell (20/56 mg/m^2)1.20Not reported0.951.48Not 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 objective of this endpoint was to compare EFS in study 20140106 with EFS in an external control arm selected from an observational study (Amgen 20180065). The median duration in months in the external control arm was 3.62 (95% CI: 1.55, 5.36) for B-cell participants, with a treatment difference hazard ration of 1.435 (95% CI: 0.976, 2.111). For T-Cell participants the median in months was 2.93 (95% CI: 0.95, 5.10) with a treatment difference hazard ratio of 1.404 (95% CI: 0.869, 2.270).
Complete source fields
analyses
  1. group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    OTHER
    other Analysis Description
    The objective of this endpoint was to compare EFS in study 20140106 with EFS in an external control arm selected from an observational study (Amgen 20180065). The median duration in months in the external control arm was 3.62 (95% CI: 1.55, 5.36) for B-cell participants, with a treatment difference hazard ration of 1.435 (95% CI: 0.976, 2.111). For T-Cell participants the median in months was 2.93 (95% CI: 0.95, 5.10) with a treatment difference hazard ratio of 1.404 (95% CI: 0.869, 2.270).
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        0.95
        upper Limit
        2.24
        value
        1.18
      2. group Id
        OG001
        lower Limit
        0.95
        upper Limit
        1.48
        value
        1.20
denoms
  1. counts
    1. group Id
      OG000
      value
      61
    2. group Id
      OG001
      value
      44
    units
    Participants
description
EFS was defined as time from initiation of therapy until treatment failure (defined as failure to reach at least a CRi after consolidation or after induction in participants that did not receive consolidation), relapse, or death from any cause. CRi was defined as: a. Attainment of M1 bone marrow status with no evidence of circulating blasts or extramedullary disease b. ANC and platelet counts not fulfilling criteria for CRh, CRp, or CR. Data was IPTW adjusted. Medians were estimated using the Kaplan-Meier (KM) method.
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
MEDIAN
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 approximately 2 years
title
Phase 2: Event Free Survival (EFS)
type
SECONDARY
unit Of Measure
Months

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Preserved source evidence · Not medical advice