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NCT03962543 · OUTCOME

Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version.

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.

Measure
LEAST_SQUARES_MEAN
Unit
units on a scale
Interval / dispersion
95% Confidence Interval
Time frame
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months

What was measured

The PedsQL consists of a 23-item core measure of global QOL that can be completed in approximately 5 minutes. There is a total score and four subscales: physical functioning, emotional functioning, social functioning and school/work functioning. Participants ≥ 5 years of age complete an age-appropriate self-report; and parents/guardians of children ages 2-17 complete a parent proxy report of the age-specific QOL. The recall period is 7 days. PedsQL items are answered on a Likert scale with responses ranging from 0 to 4 (where 0 means it is never a problem and 4 means it is almost always a problem). These items are then reverse scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. On this scale, higher scores indicate better outcomes. Overall scale scores are calculated as the mean of the scores for the questions in said scale (or of all questions for the total score). The change from baseline is modelled using a mixed-model for repeated measures.

Analysis population: The Full Analysis Set patients who are eligible for the Peds-QL assessments at baseline.

Groups in this outcome

Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report

Patients self-reported results aged 5 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

Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy

Patients parent proxy results 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

Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report

Patients self-reported results aged 18 years 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

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report50
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy56
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report58

Reported measurements

Total Score
Values in units on a scale · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report4.01Not reported-0.748.76Not reported
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy5.64Not reported1.759.53Not reported
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report3.94Not reported0.707.17Not reported
School/Work Functioning
Values in units on a scale · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report-0.02Not reported-7.167.13Not reported
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy1.71Not reported-4.027.45Not reported
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report4.67Not reported0.528.82Not reported
Social Functioning
Values in units on a scale · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report1.85Not reported-4.318.01Not reported
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy7.64Not reported2.9112.37Not reported
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report2.28Not reported-1.746.29Not reported
Emotional Functioning
Values in units on a scale · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report5.60Not reported-0.6611.86Not reported
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy8.15Not reported3.7112.58Not reported
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report1.51Not reported-2.785.80Not reported
Physical Functioning
Values in units on a scale · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report6.71Not reported0.5612.87Not reported
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy5.75Not reported0.3511.14Not reported
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report5.90Not reported1.3310.47Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        -0.74
        upper Limit
        8.76
        value
        4.01
      2. group Id
        OG001
        lower Limit
        1.75
        upper Limit
        9.53
        value
        5.64
      3. group Id
        OG002
        lower Limit
        0.70
        upper Limit
        7.17
        value
        3.94
    title
    Total Score
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        -7.16
        upper Limit
        7.13
        value
        -0.02
      2. group Id
        OG001
        lower Limit
        -4.02
        upper Limit
        7.45
        value
        1.71
      3. group Id
        OG002
        lower Limit
        0.52
        upper Limit
        8.82
        value
        4.67
    title
    School/Work Functioning
  3. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        -4.31
        upper Limit
        8.01
        value
        1.85
      2. group Id
        OG001
        lower Limit
        2.91
        upper Limit
        12.37
        value
        7.64
      3. group Id
        OG002
        lower Limit
        -1.74
        upper Limit
        6.29
        value
        2.28
    title
    Social Functioning
  4. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        -0.66
        upper Limit
        11.86
        value
        5.60
      2. group Id
        OG001
        lower Limit
        3.71
        upper Limit
        12.58
        value
        8.15
      3. group Id
        OG002
        lower Limit
        -2.78
        upper Limit
        5.80
        value
        1.51
    title
    Emotional Functioning
  5. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        0.56
        upper Limit
        12.87
        value
        6.71
      2. group Id
        OG001
        lower Limit
        0.35
        upper Limit
        11.14
        value
        5.75
      3. group Id
        OG002
        lower Limit
        1.33
        upper Limit
        10.47
        value
        5.90
    title
    Physical Functioning
denoms
  1. counts
    1. group Id
      OG000
      value
      50
    2. group Id
      OG001
      value
      56
    3. group Id
      OG002
      value
      58
    units
    Participants
description
The PedsQL consists of a 23-item core measure of global QOL that can be completed in approximately 5 minutes. There is a total score and four subscales: physical functioning, emotional functioning, social functioning and school/work functioning. Participants ≥ 5 years of age complete an age-appropriate self-report; and parents/guardians of children ages 2-17 complete a parent proxy report of the age-specific QOL. The recall period is 7 days. PedsQL items are answered on a Likert scale with responses ranging from 0 to 4 (where 0 means it is never a problem and 4 means it is almost always a problem). These items are then reverse scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. On this scale, higher scores indicate better outcomes. Overall scale scores are calculated as the mean of the scores for the questions in said scale (or of all questions for the total score). The change from baseline is modelled using a mixed-model for repeated measures.
dispersion Type
95% Confidence Interval
groups
  1. description
    Patients self-reported results aged 5 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
    OG000
    title
    Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Self-Report
  2. description
    Patients parent proxy results 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
    OG001
    title
    Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901) - Parent Proxy
  3. description
    Patients self-reported results aged 18 years 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
    OG002
    title
    Treatment Phase - Adult Cohort Mirdametinib (PD-0325901) - Self-Report
param Type
LEAST_SQUARES_MEAN
population Description
The Full Analysis Set patients who are eligible for the Peds-QL assessments at baseline.
reporting Status
POSTED
time Frame
Baseline and Cycle 13 (1 cycle = 28 days), up to 12 months
title
Change From Baseline on Quality of Life (QOL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) at Cycle 13, Acute Version.
type
SECONDARY
unit Of Measure
units on a scale

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