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NCT02440464 · OUTCOME

Percentage of Participants With Treatment-Related Mortality (TRM)

Allogeneic Hematopoietic Stem Cell Transplantation With Ixazomib for High Risk Multiple Myeloma (BMT CTN 1302) · Source last updated 2023-01-04

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
90% Confidence Interval
Time frame
12 months and 21 months post-randomization

What was measured

Treatment-related mortality is defined as death occurring in a patient from causes other than disease relapse or progression. The cumulative incidence of treatment-related mortality (TRM) post-randomization is estimated for each treatment arm using the Aalen-Johansen estimator, with progression treated as a competing risk. Participants who were event-free at two years post-transplant are censored at that time. Confidence intervals for values of 0% were not calculated.

Analysis population: All randomized participants were included in the analysis.

Groups in this outcome

Ixazomib Maintenance

Allogeneic HSCT and Fludarabine/Melphalan/Bortezomib conditioning followed by Ixazomib maintenance Allogeneic HSCT: Eligible patients with a Human Leukocyte Antigen (HLA)-matched related or unrelated donor will undergo allogeneic hematopoietic stem cell transplant at Day 0. For GVHD prophylaxis, patients will be given Tacrolimus from Day -3 until at least 6 months following initiation, at an intravenous daily dose of 0.015 mg/kg. This will be combined with an intravenous administration of Methotrexate at 5 mg/m2 on Days +1, +3, +6, and +11. Fludarabine: Patients will receive conditioning treatment before and after HSCT. Fludarabine will be given at 30 mg/m2 intravenously on Day -6 through Day -3. Melphalan: Melphalan will be given at 70 mg/m2 intravenously on Days -4 and -3. Bortezomib: Bortezomib will be administered at 1.3 mg/m2 intravenously on Day -3. Ixazomib: Between 60 and 120 days following HSCT, patients randomized to the experimental arm will receive Ixazomib maintenance. Maintenance will begin at 3-mg oral doses on Days 1, 8, and 15 of a 28-day cycle. This will increase to 4-mg doses on the same cycle schedule, following successful completion of 3 cycles at the previous dosage, for a total of 12 cycles.

Placebo

Allogeneic HSCT and Fludarabine/Melphalan/Bortezomib conditioning followed by placebo maintenance. Allogeneic HSCT: Eligible patients with a Human Leukocyte Antigen (HLA)-matched related or unrelated donor will undergo allogeneic hematopoietic stem cell transplant at Day 0. For GVHD prophylaxis, patients will be given Tacrolimus from Day -3 until at least 6 months following initiation, at an intravenous daily dose of 0.015 mg/kg. This will be combined with an intravenous administration of Methotrexate at 5 mg/m2 on Days +1, +3, +6, and +11. Fludarabine: Patients will receive conditioning treatment before and after HSCT. Fludarabine will be given at 30 mg/m2 intravenously on Day -6 through Day -3. Melphalan: Melphalan will be given at 70 mg/m2 intravenously on Days -4 and -3. Bortezomib: Bortezomib will be administered at 1.3 mg/m2 intravenously on Day -3. Placebo: Between 60 and 120 days following HSCT, patients randomized to the control group will be given 3 mg of placebo orally on Days 1, 8, and 15 of a 28-day cycle. This will increase to 4-mg doses on the same cycle schedule following successful completion of 3 cycles of placebo maintenance at the 3-mg dose. This will continue for a total of 12 cycles.

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

Analysis denominator · Participants

GroupSource count
Ixazomib Maintenance21
Placebo22

Reported measurements

12 Months Post-randomization
Values in percentage of participants · Interval/dispersion: 90% Confidence Interval
GroupValueSpreadLowerUpperComment
Ixazomib Maintenance0Not reportedNANABecause there were no events, the confidence interval was not calculated.
Placebo0Not reportedNANABecause there were no events, the confidence interval was not calculated.
21 Months Post-randomization
Values in percentage of participants · Interval/dispersion: 90% Confidence Interval
GroupValueSpreadLowerUpperComment
Ixazomib Maintenance0Not reportedNANABecause there were no events, the confidence interval was not calculated.
Placebo4.5Not reported0.516.5Not reported

Source statistical analyses

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

  1. group Description
    The null hypothesis is that there is no difference in the proportions of participants with treatment-related mortality between the Ixazomib and Placebo arms during 21 months post-randomization, with progression treated as a competing risk. Cumulative incidences are compared between treatment arms using Gray's test.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.173
    p Value Comment
    Statistical significance was determined using a pre-specified one-sided threshold of 0.05.
    statistical Method
    Gray's Test
Complete source fields
analyses
  1. group Description
    The null hypothesis is that there is no difference in the proportions of participants with treatment-related mortality between the Ixazomib and Placebo arms during 21 months post-randomization, with progression treated as a competing risk. Cumulative incidences are compared between treatment arms using Gray's test.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.173
    p Value Comment
    Statistical significance was determined using a pre-specified one-sided threshold of 0.05.
    statistical Method
    Gray's Test
classes
  1. categories
    1. measurements
      1. comment
        Because there were no events, the confidence interval was not calculated.
        group Id
        OG000
        lower Limit
        NA
        upper Limit
        NA
        value
        0
      2. comment
        Because there were no events, the confidence interval was not calculated.
        group Id
        OG001
        lower Limit
        NA
        upper Limit
        NA
        value
        0
    title
    12 Months Post-randomization
  2. categories
    1. measurements
      1. comment
        Because there were no events, the confidence interval was not calculated.
        group Id
        OG000
        lower Limit
        NA
        upper Limit
        NA
        value
        0
      2. group Id
        OG001
        lower Limit
        0.5
        upper Limit
        16.5
        value
        4.5
    title
    21 Months Post-randomization
denoms
  1. counts
    1. group Id
      OG000
      value
      21
    2. group Id
      OG001
      value
      22
    units
    Participants
description
Treatment-related mortality is defined as death occurring in a patient from causes other than disease relapse or progression. The cumulative incidence of treatment-related mortality (TRM) post-randomization is estimated for each treatment arm using the Aalen-Johansen estimator, with progression treated as a competing risk. Participants who were event-free at two years post-transplant are censored at that time. Confidence intervals for values of 0% were not calculated.
dispersion Type
90% Confidence Interval
groups
  1. description
    Allogeneic HSCT and Fludarabine/Melphalan/Bortezomib conditioning followed by Ixazomib maintenance Allogeneic HSCT: Eligible patients with a Human Leukocyte Antigen (HLA)-matched related or unrelated donor will undergo allogeneic hematopoietic stem cell transplant at Day 0. For GVHD prophylaxis, patients will be given Tacrolimus from Day -3 until at least 6 months following initiation, at an intravenous daily dose of 0.015 mg/kg. This will be combined with an intravenous administration of Methotrexate at 5 mg/m2 on Days +1, +3, +6, and +11. Fludarabine: Patients will receive conditioning treatment before and after HSCT. Fludarabine will be given at 30 mg/m2 intravenously on Day -6 through Day -3. Melphalan: Melphalan will be given at 70 mg/m2 intravenously on Days -4 and -3. Bortezomib: Bortezomib will be administered at 1.3 mg/m2 intravenously on Day -3. Ixazomib: Between 60 and 120 days following HSCT, patients randomized to the experimental arm will receive Ixazomib maintenance. Maintenance will begin at 3-mg oral doses on Days 1, 8, and 15 of a 28-day cycle. This will increase to 4-mg doses on the same cycle schedule, following successful completion of 3 cycles at the previous dosage, for a total of 12 cycles.
    id
    OG000
    title
    Ixazomib Maintenance
  2. description
    Allogeneic HSCT and Fludarabine/Melphalan/Bortezomib conditioning followed by placebo maintenance. Allogeneic HSCT: Eligible patients with a Human Leukocyte Antigen (HLA)-matched related or unrelated donor will undergo allogeneic hematopoietic stem cell transplant at Day 0. For GVHD prophylaxis, patients will be given Tacrolimus from Day -3 until at least 6 months following initiation, at an intravenous daily dose of 0.015 mg/kg. This will be combined with an intravenous administration of Methotrexate at 5 mg/m2 on Days +1, +3, +6, and +11. Fludarabine: Patients will receive conditioning treatment before and after HSCT. Fludarabine will be given at 30 mg/m2 intravenously on Day -6 through Day -3. Melphalan: Melphalan will be given at 70 mg/m2 intravenously on Days -4 and -3. Bortezomib: Bortezomib will be administered at 1.3 mg/m2 intravenously on Day -3. Placebo: Between 60 and 120 days following HSCT, patients randomized to the control group will be given 3 mg of placebo orally on Days 1, 8, and 15 of a 28-day cycle. This will increase to 4-mg doses on the same cycle schedule following successful completion of 3 cycles of placebo maintenance at the 3-mg dose. This will continue for a total of 12 cycles.
    id
    OG001
    title
    Placebo
param Type
NUMBER
population Description
All randomized participants were included in the analysis.
reporting Status
POSTED
time Frame
12 months and 21 months post-randomization
title
Percentage of Participants With Treatment-Related Mortality (TRM)
type
SECONDARY
unit Of Measure
percentage of participants

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

Preserved source evidence · Not medical advice