NCT04924608 · OUTCOME

(Second Key Secondary Endpoint) The Difference of the Means in the Change From Baseline in PlexiQoL Total Score at Cycle 12

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

PlexiQoL (Plexiform Neurofibroma Quality of Life scale) is a patient-derived QoL measure specific to adults with NF1-associated PNs. It assesses the impact of PNs on patients' ability to fulfil their human needs. The measure consists of 18 dichotomous items with 0 =Not True and 1 = True. PlexiQoL total scores were calculated by the sum of all items to a maximum of 18, with lower scores indicating better quality of life. The change from baseline to the end of each cycle in PlexiQoL total score was derived as the PlexiQoL total score at the cycle 12 minus baseline PlexiQoL total score and 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

Group	Source count
Selumetinib 25 mg/m2	57
Placebo	59

Reported measurements

Source class 1

Values in Scores on a scale · Interval/dispersion: Standard Error
Group	Value	Spread	Lower	Upper	Comment
Selumetinib 25 mg/m2	-0.4	0.45	Not reported	Not reported	Not reported
Placebo	-0.3	0.44	Not reported	Not reported	Not reported

Source statistical analyses

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

ci Lower Limit
-1.2
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
1.1
dispersion Type
STANDARD_ERROR_OF_MEAN
dispersion Value
0.59
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
0.918
param Type
LS mean difference
param Value
-0.1
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

ci Lower Limit
-1.2
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
1.1
dispersion Type
STANDARD_ERROR_OF_MEAN
dispersion Value
0.59
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
0.918
param Type
LS mean difference
param Value
-0.1
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

categories
measurements
group Id
OG000
spread
0.45
value
-0.4
group Id
OG001
spread
0.44
value
-0.3

denoms

counts
group Id
OG000
value
57
group Id
OG001
value
59
units
Participants

description

dispersion Type

Standard Error

groups

description
Actual Treatment Group
id
OG000
title
Selumetinib 25 mg/m2
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

(Second Key Secondary Endpoint) The Difference of the Means in the Change From Baseline in PlexiQoL Total Score at Cycle 12

type

SECONDARY

unit Of Measure

Scores on a scale

Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0

Preserved source evidence · Not medical advice