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NCT04924608 · OUTCOME

Confirmed Partial and Complete Response Rate (ORR) by End of Cycle 16 Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) in Participants With NF1 Who Have Symptomatic, Inoperable PN.

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
NUMBER
Unit
Percentage
Interval / dispersion
95% Confidence Interval
Time frame
From first dose up until progression (if it occurs prior to the end of Cycle 16), or the last evaluable assessment up to and including the end of Cycle 16, excluding MRI during prolonged study intervention interruption (defined as interruption >= 28 days)

What was measured

Objective response rate is defined as the proportion of participants who have a confirmed CR (defined as disappearance of the target PN, confirmed by a consecutive scan within 3 to 6 months after the first response) or confirmed PR (defined as a target PN volume decrease ≥ 20%, compared to baseline, confirmed by a consecutive scan within 3 to 6 months after the first response) by end of Cycle 16 as determined by ICR per REiNS criteria. Increase in the volume of the target PN by 20% or more compared to baseline or the time of best response after documenting a PR is considered as PD.

Analysis population: Full analysis set - All patients who are randomized 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/m271
Placebo74

Reported measurements

Source class 1
Values in Percentage · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Selumetinib 25 mg/m219.7Not reported11.230.9Not reported
Placebo5.4Not reported1.513.3Not reported

Source statistical analyses

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

  1. group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.0112
    statistical Comment
    The ORR will be compared at the end of Cycle 16 between selumetinib vs placebo with the 2-sided significance level 0.047.
    statistical Method
    Fisher Exact
Complete source fields
analyses
  1. group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.0112
    statistical Comment
    The ORR will be compared at the end of Cycle 16 between selumetinib vs placebo with the 2-sided significance level 0.047.
    statistical Method
    Fisher Exact
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        11.2
        upper Limit
        30.9
        value
        19.7
      2. group Id
        OG001
        lower Limit
        1.5
        upper Limit
        13.3
        value
        5.4
denoms
  1. counts
    1. group Id
      OG000
      value
      71
    2. group Id
      OG001
      value
      74
    units
    Participants
description
Objective response rate is defined as the proportion of participants who have a confirmed CR (defined as disappearance of the target PN, confirmed by a consecutive scan within 3 to 6 months after the first response) or confirmed PR (defined as a target PN volume decrease ≥ 20%, compared to baseline, confirmed by a consecutive scan within 3 to 6 months after the first response) by end of Cycle 16 as determined by ICR per REiNS criteria. Increase in the volume of the target PN by 20% or more compared to baseline or the time of best response after documenting a PR is considered as PD.
dispersion Type
95% Confidence Interval
groups
  1. description
    Actual Treatment Group
    id
    OG000
    title
    Selumetinib 25 mg/m2
  2. description
    Actual Treatment Group
    id
    OG001
    title
    Placebo
param Type
NUMBER
population Description
Full analysis set - All patients who are randomized to study intervention
reporting Status
POSTED
time Frame
From first dose up until progression (if it occurs prior to the end of Cycle 16), or the last evaluable assessment up to and including the end of Cycle 16, excluding MRI during prolonged study intervention interruption (defined as interruption >= 28 days)
title
Confirmed Partial and Complete Response Rate (ORR) by End of Cycle 16 Using Volumetric MRI Analysis as Determined by ICR (Per REiNS Criteria) in Participants With NF1 Who Have Symptomatic, Inoperable PN.
type
PRIMARY
unit Of Measure
Percentage

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

Preserved source evidence · Not medical advice