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NCT05309668 · ADVERSE EVENTS

Adverse events

Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN · Source last updated 2026-08-13

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 (death due to any cause): from first dose of study medication up to and including the data cut-off 1 analysis (08Apr2024), up to 26 months. Treatment-emergent adverse events: from first dose of study medication up to and including 30 days after the last dose of selumetinib, an average of 12.6 months.
Other-event reporting threshold
5%

All-cause mortality was reported for all subjects. Adverse events were reported for subjects who received at least one dose of study drug, and includes AEs that start prior to and worsen after treatment or with an onset or worsening date on or after the date of first selumetinib dose up to and including 30 days after the date of last selumetinib dose.

Participants affected / participants at risk, as reported
Safety groupSerious eventsOther eventsDeaths
Global Cohort 1

Global Cohort 1 (\>=4 to \<7 years)

1 / 1515 / 150 / 15
Global Cohort 2

Global Cohort 2 (\>=1 to \<4 years)

1 / 1717 / 170 / 17
Total in Global

Global Cohorts (\>=1 to \<7 years)

2 / 3232 / 320 / 32
Japan Cohort

Japan Cohort (\>=1 to \<7 years)

0 / 44 / 40 / 4
Total in Study

Total in study (\>=1 to \<7 years)

2 / 3636 / 360 / 36

Group identifiers: EG000: Global Cohort 1 · EG001: Global Cohort 2 · EG002: Total in Global · EG003: Japan Cohort · EG004: Total in Study

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
    General disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      1
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      17
      num Events
      0
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Pyrexia
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Infections and infestations
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Gastroenteritis
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Infections and infestations
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Upper respiratory tract infection

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 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      1
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      2
      num At Risk
      32
      num Events
      2
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      2
      num At Risk
      36
      num Events
      2
    term
    Abdominal pain upper
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Psychiatric disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      1
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      2
      num At Risk
      32
      num Events
      2
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      2
      num At Risk
      36
      num Events
      2
    term
    Irritability
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Psychiatric disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Staring
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Renal and urinary disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Haemoglobinuria
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Renal and urinary disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Proteinuria
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Respiratory, thoracic and mediastinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Catarrh
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Respiratory, thoracic and mediastinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      3
    2. group Id
      EG001
      num Affected
      2
      num At Risk
      17
      num Events
      3
    3. group Id
      EG002
      num Affected
      3
      num At Risk
      32
      num Events
      6
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      3
      num At Risk
      36
      num Events
      6
    term
    Cough
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Respiratory, thoracic and mediastinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      1
    2. group Id
      EG001
      num Affected
      2
      num At Risk
      17
      num Events
      2
    3. group Id
      EG002
      num Affected
      3
      num At Risk
      32
      num Events
      3
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      3
      num At Risk
      36
      num Events
      3
    term
    Epistaxis
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Respiratory, thoracic and mediastinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      2
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      2
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      2
    term
    Productive cough
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Respiratory, thoracic and mediastinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      1
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      17
      num Events
      0
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Respiratory disorder
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Respiratory, thoracic and mediastinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      1
    2. group Id
      EG001
      num Affected
      3
      num At Risk
      17
      num Events
      4
    3. group Id
      EG002
      num Affected
      4
      num At Risk
      32
      num Events
      5
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      4
      num At Risk
      36
      num Events
      5
    term
    Rhinorrhoea
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Anal fissure
Complete source fields
description
All-cause mortality was reported for all subjects. Adverse events were reported for subjects who received at least one dose of study drug, and includes AEs that start prior to and worsen after treatment or with an onset or worsening date on or after the date of first selumetinib dose up to and including 30 days after the date of last selumetinib dose.
event Groups
  1. deaths Num Affected
    0
    deaths Num At Risk
    15
    description
    Global Cohort 1 (\>=4 to \<7 years)
    id
    EG000
    other Num Affected
    15
    other Num At Risk
    15
    serious Num Affected
    1
    serious Num At Risk
    15
    title
    Global Cohort 1
  2. deaths Num Affected
    0
    deaths Num At Risk
    17
    description
    Global Cohort 2 (\>=1 to \<4 years)
    id
    EG001
    other Num Affected
    17
    other Num At Risk
    17
    serious Num Affected
    1
    serious Num At Risk
    17
    title
    Global Cohort 2
  3. deaths Num Affected
    0
    deaths Num At Risk
    32
    description
    Global Cohorts (\>=1 to \<7 years)
    id
    EG002
    other Num Affected
    32
    other Num At Risk
    32
    serious Num Affected
    2
    serious Num At Risk
    32
    title
    Total in Global
  4. deaths Num Affected
    0
    deaths Num At Risk
    4
    description
    Japan Cohort (\>=1 to \<7 years)
    id
    EG003
    other Num Affected
    4
    other Num At Risk
    4
    serious Num Affected
    0
    serious Num At Risk
    4
    title
    Japan Cohort
  5. deaths Num Affected
    0
    deaths Num At Risk
    36
    description
    Total in study (\>=1 to \<7 years)
    id
    EG004
    other Num Affected
    36
    other Num At Risk
    36
    serious Num Affected
    2
    serious Num At Risk
    36
    title
    Total in Study
frequency Threshold
5
other Events
  1. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      1
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      2
      num At Risk
      32
      num Events
      2
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      2
      num At Risk
      36
      num Events
      2
    term
    Abdominal pain upper
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Psychiatric disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      1
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      2
      num At Risk
      32
      num Events
      2
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      2
      num At Risk
      36
      num Events
      2
    term
    Irritability
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Psychiatric disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Staring
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Renal and urinary disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Haemoglobinuria
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Renal and urinary disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Proteinuria
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Respiratory, thoracic and mediastinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Catarrh
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Respiratory, thoracic and mediastinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      3
    2. group Id
      EG001
      num Affected
      2
      num At Risk
      17
      num Events
      3
    3. group Id
      EG002
      num Affected
      3
      num At Risk
      32
      num Events
      6
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      3
      num At Risk
      36
      num Events
      6
    term
    Cough
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Respiratory, thoracic and mediastinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      1
    2. group Id
      EG001
      num Affected
      2
      num At Risk
      17
      num Events
      2
    3. group Id
      EG002
      num Affected
      3
      num At Risk
      32
      num Events
      3
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      3
      num At Risk
      36
      num Events
      3
    term
    Epistaxis
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Respiratory, thoracic and mediastinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      2
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      2
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      2
    term
    Productive cough
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Respiratory, thoracic and mediastinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      1
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      17
      num Events
      0
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Respiratory disorder
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Respiratory, thoracic and mediastinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      1
    2. group Id
      EG001
      num Affected
      3
      num At Risk
      17
      num Events
      4
    3. group Id
      EG002
      num Affected
      4
      num At Risk
      32
      num Events
      5
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      4
      num At Risk
      36
      num Events
      5
    term
    Rhinorrhoea
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Anal fissure
serious Events
  1. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    General disorders
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      15
      num Events
      1
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      17
      num Events
      0
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Pyrexia
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Infections and infestations
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Gastroenteritis
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Infections and infestations
    source Vocabulary
    MedDRA 26.1
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      15
      num Events
      0
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      17
      num Events
      1
    3. group Id
      EG002
      num Affected
      1
      num At Risk
      32
      num Events
      1
    4. group Id
      EG003
      num Affected
      0
      num At Risk
      4
      num Events
      0
    5. group Id
      EG004
      num Affected
      1
      num At Risk
      36
      num Events
      1
    term
    Upper respiratory tract infection
time Frame
All-cause mortality (death due to any cause): from first dose of study medication up to and including the data cut-off 1 analysis (08Apr2024), up to 26 months. Treatment-emergent adverse events: from first dose of study medication up to and including 30 days after the last dose of selumetinib, an average of 12.6 months.

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Preserved source evidence · Not medical advice