Skip to content
Triangle logo TriangleGet started
← Full study record

NCT02604433 · OUTCOME

Duration of Reduction in Transfusion Burden

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
Days
Interval / dispersion
Standard Deviation
Time frame
From first dose to end of study treatment (up to approximately 215 weeks)

What was measured

Responders were defined as subjects who achieved ≥ 33% reduction or ≥ 50% reduction in Red Blood Cells Transfusion (RBC-T) burden from baseline with a reduction of at least 2 RBC units during any rolling 12-week interval. The duration of reduction is calculated as Last Day of Response - First day of response +1

Analysis population: All randomized participants with ≥ 33% reduction or ≥ 50% reduction in RBC-T burden

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 + BSC173
Placebo + BSC39

Reported measurements

Number of participants analyzed for 33%

Analysis denominator · Participants

GroupSource count
Luspatercept + BSC173
Placebo + BSC39
Values in Days · Interval/dispersion: Standard Deviation
GroupValueSpreadLowerUpperComment
Luspatercept + BSC627.3390.54Not reportedNot reportedNot reported
Placebo + BSC224.0155.15Not reportedNot reportedNot reported
Number of participants analyzed for 50%

Analysis denominator · Participants

GroupSource count
Luspatercept + BSC112
Placebo + BSC9
Values in Days · Interval/dispersion: Standard Deviation
GroupValueSpreadLowerUpperComment
Luspatercept + BSC491.1386.37Not reportedNot reportedNot reported
Placebo + BSC193.0142.51Not reportedNot reportedNot reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        spread
        390.54
        value
        627.3
      2. group Id
        OG001
        spread
        155.15
        value
        224.0
    denoms
    1. counts
      1. group Id
        OG000
        value
        173
      2. group Id
        OG001
        value
        39
      units
      Participants
    title
    Number of participants analyzed for 33%
  2. categories
    1. measurements
      1. group Id
        OG000
        spread
        386.37
        value
        491.1
      2. group Id
        OG001
        spread
        142.51
        value
        193.0
    denoms
    1. counts
      1. group Id
        OG000
        value
        112
      2. group Id
        OG001
        value
        9
      units
      Participants
    title
    Number of participants analyzed for 50%
denoms
  1. counts
    1. group Id
      OG000
      value
      173
    2. group Id
      OG001
      value
      39
    units
    Participants
description
Responders were defined as subjects who achieved ≥ 33% reduction or ≥ 50% reduction in Red Blood Cells Transfusion (RBC-T) burden from baseline with a reduction of at least 2 RBC units during any rolling 12-week interval. The duration of reduction is calculated as Last Day of Response - First day of response +1
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 ≥ 33% reduction or ≥ 50% reduction in RBC-T burden
reporting Status
POSTED
time Frame
From first dose to end of study treatment (up to approximately 215 weeks)
title
Duration of Reduction in Transfusion Burden
type
SECONDARY
unit Of Measure
Days

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

Preserved source evidence · Not medical advice