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NCT02604433 · ADVERSE EVENTS

Adverse events

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.

Adverse events occurred during the study; they are not necessarily caused by treatment. Serious events are not interchangeable with severe/grade 3–4 events. These safety populations may differ from efficacy populations.

Collection time frame
All-cause mortality was assessed from first dose to study completion (approximately 56 months) SAEs and NSAEs were assessed from first dose to 90 days following last dose (up to approximately 52 months)
Other-event reporting threshold
5%

Adverse events were collected in all participants who received at least 1 dose of study drug.

Participants affected / participants at risk, as reported
Safety groupSerious eventsOther eventsDeaths
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

53 / 223211 / 2234 / 223
Placebo + BSC

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

8 / 10995 / 1091 / 109

Group identifiers: EG000: Luspatercept + BSC · EG001: Placebo + BSC

Serious adverse events

Event terms and counts are source-reported; expand to read more. A reporting threshold may omit less frequent events.

  1. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      4
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Anaemia
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      2
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Extramedullary haemopoiesis
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Febrile neutropenia
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Neutropenia
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Pancytopenia
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Thrombocytopenia
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Cardiac arrest
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Cardiac failure congestive
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Cardiac iron overload
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Left ventricular dysfunction
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Myocardial ischaemia
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      2
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Abdominal pain

Other reported adverse events

Event terms and counts are source-reported; expand to read more. A reporting threshold may omit less frequent events.

  1. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      30
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      7
      num At Risk
      109
    term
    Abdominal pain
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      25
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      8
      num At Risk
      109
    term
    Abdominal pain upper
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      12
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      4
      num At Risk
      109
    term
    Constipation
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      41
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      14
      num At Risk
      109
    term
    Diarrhoea
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      19
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Dyspepsia
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      29
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      6
      num At Risk
      109
    term
    Nausea
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      15
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      5
      num At Risk
      109
    term
    Toothache
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      30
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      8
      num At Risk
      109
    term
    Vomiting
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    General disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      26
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      11
      num At Risk
      109
    term
    Asthenia
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    General disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      38
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      16
      num At Risk
      109
    term
    Fatigue
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    General disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      21
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      8
      num At Risk
      109
    term
    Influenza like illness
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    General disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      12
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      3
      num At Risk
      109
    term
    Injection site pain
Complete source fields
description
Adverse events were collected in all participants who received at least 1 dose of study drug.
event Groups
  1. deaths Num Affected
    4
    deaths Num At Risk
    223
    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
    EG000
    other Num Affected
    211
    other Num At Risk
    223
    serious Num Affected
    53
    serious Num At Risk
    223
    title
    Luspatercept + BSC
  2. deaths Num Affected
    1
    deaths Num At Risk
    109
    description
    Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care
    id
    EG001
    other Num Affected
    95
    other Num At Risk
    109
    serious Num Affected
    8
    serious Num At Risk
    109
    title
    Placebo + BSC
frequency Threshold
5
other Events
  1. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      30
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      7
      num At Risk
      109
    term
    Abdominal pain
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      25
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      8
      num At Risk
      109
    term
    Abdominal pain upper
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      12
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      4
      num At Risk
      109
    term
    Constipation
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      41
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      14
      num At Risk
      109
    term
    Diarrhoea
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      19
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Dyspepsia
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      29
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      6
      num At Risk
      109
    term
    Nausea
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      15
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      5
      num At Risk
      109
    term
    Toothache
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      30
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      8
      num At Risk
      109
    term
    Vomiting
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    General disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      26
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      11
      num At Risk
      109
    term
    Asthenia
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    General disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      38
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      16
      num At Risk
      109
    term
    Fatigue
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    General disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      21
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      8
      num At Risk
      109
    term
    Influenza like illness
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    General disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      12
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      3
      num At Risk
      109
    term
    Injection site pain
serious Events
  1. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      4
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Anaemia
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      2
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Extramedullary haemopoiesis
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Febrile neutropenia
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Neutropenia
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Pancytopenia
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Blood and lymphatic system disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Thrombocytopenia
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Cardiac arrest
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Cardiac failure congestive
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      109
    term
    Cardiac iron overload
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Left ventricular dysfunction
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Cardiac disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Myocardial ischaemia
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 23.0
    stats
    1. group Id
      EG000
      num Affected
      2
      num At Risk
      223
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      109
    term
    Abdominal pain
time Frame
All-cause mortality was assessed from first dose to study completion (approximately 56 months) SAEs and NSAEs were assessed from first dose to 90 days following last dose (up to approximately 52 months)

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