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NCT02604433 · OUTCOME

Mean Change From Baseline In Mean Daily Dose Of Iron Chelation Therapies (ICT) At Week 48

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia · Source last updated 2023-04-18

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

Measure
MEAN
Unit
mg
Interval / dispersion
Standard Deviation
Time frame
Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48

What was measured

Three different types of Iron Chelation Therapy (ICT) were analyzed: 1. Deferasirox 2. Deferiprone 3. Deferoxamine Mesilate/Deferoxamine The baseline mean daily dose was calculated using the ICT dosage during the 12 weeks prior to first study drug administration and the postbaseline mean daily dose was calculated during the last 12 weeks of the 48-week double-blind Treatment Period or the last 12 weeks of the study treatment for early discontinued participants.

Analysis population: All randomized participants with available measurements at the specified timepoints

Groups in this outcome

Luspatercept + BSC

Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care

Placebo + BSC

Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care

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

Analysis denominator · Participants

GroupSource count
Luspatercept + BSC129
Placebo + BSC66

Reported measurements

Deferasirox

Analysis denominator · Participants

GroupSource count
Luspatercept + BSC93
Placebo + BSC49
Values in mg · Interval/dispersion: Standard Deviation
GroupValueSpreadLowerUpperComment
Luspatercept + BSC-105.0378.67Not reportedNot reportedNot reported
Placebo + BSC-60.4297.11Not reportedNot reportedNot reported
Deferiprone

Analysis denominator · Participants

GroupSource count
Luspatercept + BSC30
Placebo + BSC12
Values in mg · Interval/dispersion: Standard Deviation
GroupValueSpreadLowerUpperComment
Luspatercept + BSC-229.1893.62Not reportedNot reportedNot reported
Placebo + BSC-73.4614.80Not reportedNot reportedNot reported
Deferoxamine Mesilate/Deferoxamine

Analysis denominator · Participants

GroupSource count
Luspatercept + BSC6
Placebo + BSC5
Values in mg · Interval/dispersion: Standard Deviation
GroupValueSpreadLowerUpperComment
Luspatercept + BSC84.9523.45Not reportedNot reportedNot reported
Placebo + BSC274.2613.05Not reportedNot reportedNot reported

Source statistical analyses

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

  1. ci Lower Limit
    -185.8
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    49.7
    estimate Comment
    luspatercept - placebo
    group Description
    Deferasirox: Change from baseline at Week 48 LS = least squares
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.2552
    p Value Comment
    Significance level of 0.050 for 2-sided tests
    param Type
    LS Mean of Difference
    param Value
    -68.0
    statistical Comment
    Estimates are based on an ANCOVA model with geographical regions defined at randomization and baseline ICT as covariates.
    statistical Method
    ANCOVA
  2. ci Lower Limit
    -612.9
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    460.1
    estimate Comment
    luspatercept - placebo
    group Description
    Deferiprone: Change from baseline at Week 48 LS = least squares
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.7746
    p Value Comment
    Significance level of 0.050 for 2-sided tests
    param Type
    LS Mean of Difference
    param Value
    -76.4
    statistical Comment
    Estimates are based on an ANCOVA model with geographical regions defined at randomization and baseline ICT as covariates.
    statistical Method
    ANCOVA
  3. ci Lower Limit
    -673.1
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    378.5
    estimate Comment
    luspatercept - placebo
    group Description
    Deferoxamine Mesilate / Deferoxamine: Change from baseline at Week 48 LS = least squares
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.5186
    p Value Comment
    Significance level of 0.050 for 2-sided tests
    param Type
    LS Mean of Difference
    param Value
    -147.3
    statistical Comment
    Estimates are based on an ANCOVA model with geographical regions defined at randomization and baseline ICT as covariates.
    statistical Method
    ANCOVA
Complete source fields
analyses
  1. ci Lower Limit
    -185.8
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    49.7
    estimate Comment
    luspatercept - placebo
    group Description
    Deferasirox: Change from baseline at Week 48 LS = least squares
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.2552
    p Value Comment
    Significance level of 0.050 for 2-sided tests
    param Type
    LS Mean of Difference
    param Value
    -68.0
    statistical Comment
    Estimates are based on an ANCOVA model with geographical regions defined at randomization and baseline ICT as covariates.
    statistical Method
    ANCOVA
  2. ci Lower Limit
    -612.9
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    460.1
    estimate Comment
    luspatercept - placebo
    group Description
    Deferiprone: Change from baseline at Week 48 LS = least squares
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.7746
    p Value Comment
    Significance level of 0.050 for 2-sided tests
    param Type
    LS Mean of Difference
    param Value
    -76.4
    statistical Comment
    Estimates are based on an ANCOVA model with geographical regions defined at randomization and baseline ICT as covariates.
    statistical Method
    ANCOVA
  3. ci Lower Limit
    -673.1
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    378.5
    estimate Comment
    luspatercept - placebo
    group Description
    Deferoxamine Mesilate / Deferoxamine: Change from baseline at Week 48 LS = least squares
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.5186
    p Value Comment
    Significance level of 0.050 for 2-sided tests
    param Type
    LS Mean of Difference
    param Value
    -147.3
    statistical Comment
    Estimates are based on an ANCOVA model with geographical regions defined at randomization and baseline ICT as covariates.
    statistical Method
    ANCOVA
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        spread
        378.67
        value
        -105.0
      2. group Id
        OG001
        spread
        297.11
        value
        -60.4
    denoms
    1. counts
      1. group Id
        OG000
        value
        93
      2. group Id
        OG001
        value
        49
      units
      Participants
    title
    Deferasirox
  2. categories
    1. measurements
      1. group Id
        OG000
        spread
        893.62
        value
        -229.1
      2. group Id
        OG001
        spread
        614.80
        value
        -73.4
    denoms
    1. counts
      1. group Id
        OG000
        value
        30
      2. group Id
        OG001
        value
        12
      units
      Participants
    title
    Deferiprone
  3. categories
    1. measurements
      1. group Id
        OG000
        spread
        523.45
        value
        84.9
      2. group Id
        OG001
        spread
        613.05
        value
        274.2
    denoms
    1. counts
      1. group Id
        OG000
        value
        6
      2. group Id
        OG001
        value
        5
      units
      Participants
    title
    Deferoxamine Mesilate/Deferoxamine
denoms
  1. counts
    1. group Id
      OG000
      value
      129
    2. group Id
      OG001
      value
      66
    units
    Participants
description
Three different types of Iron Chelation Therapy (ICT) were analyzed: 1. Deferasirox 2. Deferiprone 3. Deferoxamine Mesilate/Deferoxamine The baseline mean daily dose was calculated using the ICT dosage during the 12 weeks prior to first study drug administration and the postbaseline mean daily dose was calculated during the last 12 weeks of the 48-week double-blind Treatment Period or the last 12 weeks of the study treatment for early discontinued participants.
dispersion Type
Standard Deviation
groups
  1. description
    Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care
    id
    OG000
    title
    Luspatercept + BSC
  2. description
    Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care
    id
    OG001
    title
    Placebo + BSC
param Type
MEAN
population Description
All randomized participants with available measurements at the specified timepoints
reporting Status
POSTED
time Frame
Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48
title
Mean Change From Baseline In Mean Daily Dose Of Iron Chelation Therapies (ICT) At Week 48
type
SECONDARY
unit Of Measure
mg

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