Participant flow

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.

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

   FG000

   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

   FG001

   title

   Placebo + BSC

periods

1. drop Withdraws

   1. reasons

      1. group Id

         FG000

         num Subjects

         37

      2. group Id

         FG001

         num Subjects

         16

      type

      Withdrawal by Subject

   2. reasons

      1. group Id

         FG000

         num Subjects

         4

      2. group Id

         FG001

         num Subjects

         0

      type

      Adverse Event

   3. reasons

      1. group Id

         FG000

         num Subjects

         3

      2. group Id

         FG001

         num Subjects

         5

      type

      Other reasons

   4. reasons

      1. group Id

         FG000

         num Subjects

         4

      2. group Id

         FG001

         num Subjects

         1

      type

      Death

   5. reasons

      1. group Id

         FG000

         num Subjects

         169

      2. group Id

         FG001

         num Subjects

         82

      type

      Transition to Rollover Protocol

   6. reasons

      1. group Id

         FG000

         num Subjects

         1

      2. group Id

         FG001

         num Subjects

         1

      type

      Physician Decision

   7. reasons

      1. group Id

         FG000

         num Subjects

         0

      2. group Id

         FG001

         num Subjects

         1

      type

      Lack of Efficacy

   milestones

   1. achievements

      1. group Id

         FG000

         num Subjects

         224

      2. group Id

         FG001

         num Subjects

         112

      comment

      Participants randomized

      type

      STARTED

   2. achievements

      1. group Id

         FG000

         num Subjects

         223

      2. group Id

         FG001

         num Subjects

         109

      comment

      All participants who received at least 1 dose of study drug

      type

      Participants Treated

   3. achievements

      1. group Id

         FG000

         num Subjects

         211

      2. group Id

         FG001

         num Subjects

         102

      type

      Completed 24 Weeks of Treatment

   4. achievements

      1. group Id

         FG000

         num Subjects

         202

      2. group Id

         FG001

         num Subjects

         96

      type

      Completed 48 Weeks of Treatment

   5. achievements

      1. group Id

         FG000

         num Subjects

         155

      2. group Id

         FG001

         num Subjects

         3

      type

      Completed 96 Weeks of Treatment

   6. achievements

      1. group Id

         FG000

         num Subjects

         125

      2. group Id

         FG001

         num Subjects

         0

      type

      Completed 144 Weeks of Treatment

   7. achievements

      1. group Id

         FG000

         num Subjects

         6

      2. group Id

         FG001

         num Subjects

         0

      type

      Completed 192 Weeks of Treatment

   8. achievements

      1. group Id

         FG000

         num Subjects

         6

      2. group Id

         FG001

         num Subjects

         6

      comment

      Completed study per original protocol

      type

      COMPLETED

   9. achievements

      1. group Id

         FG000

         num Subjects

         218

      2. group Id

         FG001

         num Subjects

         106

      type

      NOT COMPLETED

   title

   Overall Study

pre Assignment Details

336 participants were randomized, 332 participants were treated.

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Preserved source evidence · Not medical advice