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NCT04924608 · OUTCOME

(First Key Secondary) The Difference of the Means in the Change From Baseline in PAINS-pNF Chronic Target PN Pain Intensity Score at Cycle 12 Between Selumetinib and Placebo, Supplemental Analysis

Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas · Source last updated 2026-03-23

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
LEAST_SQUARES_MEAN
Unit
Scores on a scale
Interval / dispersion
Standard Error
Time frame
Baseline and end of cycle 12 of study intervention

What was measured

The PAINS-pNF (Pain Intensity Scale for Plexiform Neurofibroma) chronic target PN pain intensity measures the participants' experience of chronic PN-related pain intensity with score from 0 (no chronic tumor pain) to 10 (worst chronic tumor pain). Baseline PAINS-pNF chronic target PN pain score is defined as the average of the available daily PAINS-pNF chronic target PN pain scores in the screening perio, while for Cycle 12, it is defined as the average of the available daily scores for the 28-days cycle up to and including the last day assessment of cycle 12. The difference of the means in the change from baseline at Cycle 12 between selumetinib and placebo participants is presented.

Analysis population: Full analysis set - subjects randomised to study intervention

Groups in this outcome

Selumetinib 25 mg/m2

Actual Treatment Group

Placebo

Actual Treatment Group

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

Analysis denominator · Participants

GroupSource count
Selumetinib 25 mg/m257
Placebo62

Reported measurements

Source class 1
Values in Scores on a scale · Interval/dispersion: Standard Error
GroupValueSpreadLowerUpperComment
Selumetinib 25 mg/m2-1.60.22Not reportedNot reportedNot reported
Placebo-0.90.21Not reportedNot reportedNot reported

Source statistical analyses

Group order, estimate direction, methods and comments are retained. No treatment ranking is inferred.

  1. ci Lower Limit
    -1.3
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    -0.1
    dispersion Type
    STANDARD_ERROR_OF_MEAN
    dispersion Value
    0.30
    group Description
    supplementary analysis is not controlled for multiplicity.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.024
    p Value Comment
    nominal
    param Type
    LS mean difference
    param Value
    -0.7
    statistical Comment
    Analysis is based on a MMRM model for repeated measures, Kenward-Roger method is used to estimate the degrees of freedom.
    statistical Method
    Mixed Models Analysis
Complete source fields
analyses
  1. ci Lower Limit
    -1.3
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    -0.1
    dispersion Type
    STANDARD_ERROR_OF_MEAN
    dispersion Value
    0.30
    group Description
    supplementary analysis is not controlled for multiplicity.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.024
    p Value Comment
    nominal
    param Type
    LS mean difference
    param Value
    -0.7
    statistical Comment
    Analysis is based on a MMRM model for repeated measures, Kenward-Roger method is used to estimate the degrees of freedom.
    statistical Method
    Mixed Models Analysis
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        spread
        0.22
        value
        -1.6
      2. group Id
        OG001
        spread
        0.21
        value
        -0.9
denoms
  1. counts
    1. group Id
      OG000
      value
      57
    2. group Id
      OG001
      value
      62
    units
    Participants
description
The PAINS-pNF (Pain Intensity Scale for Plexiform Neurofibroma) chronic target PN pain intensity measures the participants' experience of chronic PN-related pain intensity with score from 0 (no chronic tumor pain) to 10 (worst chronic tumor pain). Baseline PAINS-pNF chronic target PN pain score is defined as the average of the available daily PAINS-pNF chronic target PN pain scores in the screening perio, while for Cycle 12, it is defined as the average of the available daily scores for the 28-days cycle up to and including the last day assessment of cycle 12. The difference of the means in the change from baseline at Cycle 12 between selumetinib and placebo participants is presented.
dispersion Type
Standard Error
groups
  1. description
    Actual Treatment Group
    id
    OG000
    title
    Selumetinib 25 mg/m2
  2. description
    Actual Treatment Group
    id
    OG001
    title
    Placebo
param Type
LEAST_SQUARES_MEAN
population Description
Full analysis set - subjects randomised to study intervention
reporting Status
POSTED
time Frame
Baseline and end of cycle 12 of study intervention
title
(First Key Secondary) The Difference of the Means in the Change From Baseline in PAINS-pNF Chronic Target PN Pain Intensity Score at Cycle 12 Between Selumetinib and Placebo, Supplemental Analysis
type
SECONDARY
unit Of Measure
Scores on a scale

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Preserved source evidence · Not medical advice