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NCT03962543 · ADVERSE EVENTS

Adverse events

MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas · Source last updated 2026-07-10

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 SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment, an average of 1 year and 10 months and up to 3 years and 10 months.
Other-event reporting threshold
5%

All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment. Medical occurrences that begin before the start of study treatment but after obtaining informed consent will be recorded as AEs.

Participants affected / participants at risk, as reported
Safety groupSerious eventsOther eventsDeaths
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901)

Patients aged 2 to 17 years old at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily

8 / 5656 / 560 / 56
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901)

Patients aged 18 and above at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily

10 / 5857 / 581 / 58

Group identifiers: EG000: Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) · EG001: Treatment Phase - Adult Cohort Mirdametinib (PD-0325901)

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
    Infections and infestations
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      2
      num At Risk
      58
    term
    COVID-19
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Infections and infestations
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      58
    term
    Gastrointestinal viral infection
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Infections and infestations
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      58
    term
    Rotavirus infection
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Infections and infestations
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      58
    term
    Urinary tract infection
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Injury, poisoning and procedural complications
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      58
    term
    Fall
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Injury, poisoning and procedural complications
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      58
    term
    Femoral neck fracture
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Injury, poisoning and procedural complications
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      58
    term
    Near drowning
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Renal and urinary disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      2
      num At Risk
      58
    term
    Nephrolithiasis
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Renal and urinary disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      58
    term
    Acute kidney injury
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Eye disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      58
    term
    Diplopia
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Eye disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      58
    term
    Retinal vein occlusion
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      58
    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
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      24
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      45
      num At Risk
      58
    term
    Dermatitis acneiform
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      10
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      9
      num At Risk
      58
    term
    Dry skin
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      8
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      7
      num At Risk
      58
    term
    Alopecia
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      11
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      4
      num At Risk
      58
    term
    Rash
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      7
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      7
      num At Risk
      58
    term
    Pruritus
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      8
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      2
      num At Risk
      58
    term
    Eczema
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      7
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      58
    term
    Hair colour changes
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      3
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      4
      num At Risk
      58
    term
    Urticaria
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      31
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      34
      num At Risk
      58
    term
    Diarrhoea
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      15
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      30
      num At Risk
      58
    term
    Nausea
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      22
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      22
      num At Risk
      58
    term
    Vomiting
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      15
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      10
      num At Risk
      58
    term
    Abdominal pain
Complete source fields
description
All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment. Medical occurrences that begin before the start of study treatment but after obtaining informed consent will be recorded as AEs.
event Groups
  1. deaths Num Affected
    0
    deaths Num At Risk
    56
    description
    Patients aged 2 to 17 years old at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
    id
    EG000
    other Num Affected
    56
    other Num At Risk
    56
    serious Num Affected
    8
    serious Num At Risk
    56
    title
    Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901)
  2. deaths Num Affected
    1
    deaths Num At Risk
    58
    description
    Patients aged 18 and above at the time of informed consent. Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
    id
    EG001
    other Num Affected
    57
    other Num At Risk
    58
    serious Num Affected
    10
    serious Num At Risk
    58
    title
    Treatment Phase - Adult Cohort Mirdametinib (PD-0325901)
frequency Threshold
5
other Events
  1. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      24
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      45
      num At Risk
      58
    term
    Dermatitis acneiform
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      10
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      9
      num At Risk
      58
    term
    Dry skin
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      8
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      7
      num At Risk
      58
    term
    Alopecia
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      11
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      4
      num At Risk
      58
    term
    Rash
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      7
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      7
      num At Risk
      58
    term
    Pruritus
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      8
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      2
      num At Risk
      58
    term
    Eczema
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      7
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      58
    term
    Hair colour changes
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Skin and subcutaneous tissue disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      3
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      4
      num At Risk
      58
    term
    Urticaria
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      31
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      34
      num At Risk
      58
    term
    Diarrhoea
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      15
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      30
      num At Risk
      58
    term
    Nausea
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      22
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      22
      num At Risk
      58
    term
    Vomiting
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      15
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      10
      num At Risk
      58
    term
    Abdominal pain
serious Events
  1. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Infections and infestations
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      2
      num At Risk
      58
    term
    COVID-19
  2. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Infections and infestations
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      58
    term
    Gastrointestinal viral infection
  3. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Infections and infestations
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      58
    term
    Rotavirus infection
  4. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Infections and infestations
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      58
    term
    Urinary tract infection
  5. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Injury, poisoning and procedural complications
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      58
    term
    Fall
  6. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Injury, poisoning and procedural complications
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      58
    term
    Femoral neck fracture
  7. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Injury, poisoning and procedural complications
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      58
    term
    Near drowning
  8. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Renal and urinary disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      2
      num At Risk
      58
    term
    Nephrolithiasis
  9. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Renal and urinary disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      58
    term
    Acute kidney injury
  10. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Eye disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      1
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      0
      num At Risk
      58
    term
    Diplopia
  11. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Eye disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      58
    term
    Retinal vein occlusion
  12. assessment Type
    SYSTEMATIC_ASSESSMENT
    organ System
    Gastrointestinal disorders
    source Vocabulary
    MedDRA 24.0
    stats
    1. group Id
      EG000
      num Affected
      0
      num At Risk
      56
    2. group Id
      EG001
      num Affected
      1
      num At Risk
      58
    term
    Abdominal pain
time Frame
All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment, an average of 1 year and 10 months and up to 3 years and 10 months.

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