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NCT02604433 · OUTCOME

Mean Change From Baseline in Transfusion Burden - Week 13 to Week 24

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
RBC units
Interval / dispersion
Standard Deviation
Time frame
Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24

What was measured

Baseline was defined as the total number of Red Blood Cells (RBC) units transfused during the 12-week interval on or prior to Dose 1 Day 1. This is compared to the total number of RBC units transfused during the 12-week interval from treatment weeks 13-24.

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 + BSC210
Placebo + BSC102

Reported measurements

Source class 1
Values in RBC units · Interval/dispersion: Standard Deviation
GroupValueSpreadLowerUpperComment
Luspatercept + BSC-0.671.792Not reportedNot reportedNot reported
Placebo + BSC0.661.774Not reportedNot reportedNot reported

Source statistical analyses

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

  1. ci Lower Limit
    -1.76
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    -0.93
    estimate Comment
    luspatercept - placebo
    group Description
    Change from baseline at Week 48 LSM = least squares mean
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    <0.0001
    p Value Comment
    Significance level of 0.050 for 2-sided tests.
    param Type
    LSM Difference
    param Value
    -1.34
    statistical Comment
    Estimates were based on an ANCOVA model with geographical regions defined at randomization and baseline transfusion burden as covariates.
    statistical Method
    ANCOVA
Complete source fields
analyses
  1. ci Lower Limit
    -1.76
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    -0.93
    estimate Comment
    luspatercept - placebo
    group Description
    Change from baseline at Week 48 LSM = least squares mean
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    <0.0001
    p Value Comment
    Significance level of 0.050 for 2-sided tests.
    param Type
    LSM Difference
    param Value
    -1.34
    statistical Comment
    Estimates were based on an ANCOVA model with geographical regions defined at randomization and baseline transfusion burden as covariates.
    statistical Method
    ANCOVA
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        spread
        1.792
        value
        -0.67
      2. group Id
        OG001
        spread
        1.774
        value
        0.66
denoms
  1. counts
    1. group Id
      OG000
      value
      210
    2. group Id
      OG001
      value
      102
    units
    Participants
description
Baseline was defined as the total number of Red Blood Cells (RBC) units transfused during the 12-week interval on or prior to Dose 1 Day 1. This is compared to the total number of RBC units transfused during the 12-week interval from treatment weeks 13-24.
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: Weeks 13 to Week 24
title
Mean Change From Baseline in Transfusion Burden - Week 13 to Week 24
type
SECONDARY
unit Of Measure
RBC units

Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0

Preserved source evidence · Not medical advice