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NCT02604433 · OUTCOME

Mean Change From Baseline In Myocardial Iron 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
ms
Interval / dispersion
Standard Deviation
Time frame
Baseline: Day 1; Treatment: Week 48

What was measured

Myocardial Iron levels were measured by Magnetic Resonance Imaging (MRI), using MRI parameter T2\* (Unit: ms). T2\* values correlates with heart failure (HF) risk (e.g. T2\*\<6ms: high HF risk).

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 + BSC201
Placebo + BSC102

Reported measurements

Source class 1
Values in ms · Interval/dispersion: Standard Deviation
GroupValueSpreadLowerUpperComment
Luspatercept + BSC-1.8315.084Not reportedNot reportedNot reported
Placebo + BSC-0.016.780Not reportedNot reportedNot reported

Source statistical analyses

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

  1. ci Lower Limit
    -4.48
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    0.04
    estimate Comment
    luspatercept - placebo
    group Description
    Change from baseline at Week 48 LS = least square
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.0543
    p Value Comment
    Significance level of 0.050 for 2-sided tests.
    param Type
    LS Mean of Difference
    param Value
    -2.22
    statistical Comment
    Estimates were based on an ANCOVA model with geographical regions defined at randomization and baseline myocardial T2\* as covariates.
    statistical Method
    ANCOVA
Complete source fields
analyses
  1. ci Lower Limit
    -4.48
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    0.04
    estimate Comment
    luspatercept - placebo
    group Description
    Change from baseline at Week 48 LS = least square
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.0543
    p Value Comment
    Significance level of 0.050 for 2-sided tests.
    param Type
    LS Mean of Difference
    param Value
    -2.22
    statistical Comment
    Estimates were based on an ANCOVA model with geographical regions defined at randomization and baseline myocardial T2\* as covariates.
    statistical Method
    ANCOVA
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        spread
        15.084
        value
        -1.83
      2. group Id
        OG001
        spread
        6.780
        value
        -0.01
denoms
  1. counts
    1. group Id
      OG000
      value
      201
    2. group Id
      OG001
      value
      102
    units
    Participants
description
Myocardial Iron levels were measured by Magnetic Resonance Imaging (MRI), using MRI parameter T2\* (Unit: ms). T2\* values correlates with heart failure (HF) risk (e.g. T2\*\<6ms: high HF risk).
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 1; Treatment: Week 48
title
Mean Change From Baseline In Myocardial Iron At Week 48
type
SECONDARY
unit Of Measure
ms

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

Preserved source evidence · Not medical advice