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NCT03962543 · OUTCOME

Time to Progression, From the First Dose to the First Date of a ≥ 20% Increase in Tumor Volume From Baseline.

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
MEDIAN
Unit
months
Interval / dispersion
95% Confidence Interval
Time frame
On the 15th day of every 4 cycles (each cycle is 28 days) until disease progression is observed, assessed up to approximately 3.5 years

What was measured

Evidence of progression or tumor growth will be determined by a blinded centralized review of volumetric MRI. Time to progression is defined as the time in months between the first treatment date until the date of radiographic disease progression. For participants who enter the LTFU Phase, all MRI assessments in both the Treatment Phase and LTFU Phase will be used to determine time to progression. Participants without radiographic disease progression will have their results censored to their most recent adequate (i.e. evaluable) tumor assessment date.

Analysis population: The Full Analysis Set defined as all patients who received at least one dose of Mirdametinib.

Groups in this outcome

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

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

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)56
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901)58

Reported measurements

Source class 1
Values in months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901)NANot reportedNANANA Explanation: Not reached. Only less than 50% of the patients had events in the Pediatric cohort.
Treatment Phase - Adult Cohort Mirdametinib (PD-0325901)NANot reportedNANANA Explanation: Not reached. Only less than 50% of the patients had events in the Adult cohort.
Complete source fields
classes
  1. categories
    1. measurements
      1. comment
        NA Explanation: Not reached. Only less than 50% of the patients had events in the Pediatric cohort.
        group Id
        OG000
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      2. comment
        NA Explanation: Not reached. Only less than 50% of the patients had events in the Adult cohort.
        group Id
        OG001
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
denoms
  1. counts
    1. group Id
      OG000
      value
      56
    2. group Id
      OG001
      value
      58
    units
    Participants
description
Evidence of progression or tumor growth will be determined by a blinded centralized review of volumetric MRI. Time to progression is defined as the time in months between the first treatment date until the date of radiographic disease progression. For participants who enter the LTFU Phase, all MRI assessments in both the Treatment Phase and LTFU Phase will be used to determine time to progression. Participants without radiographic disease progression will have their results censored to their most recent adequate (i.e. evaluable) tumor assessment date.
dispersion Type
95% Confidence Interval
groups
  1. 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
    OG000
    title
    Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901)
  2. 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
    OG001
    title
    Treatment Phase - Adult Cohort Mirdametinib (PD-0325901)
param Type
MEDIAN
population Description
The Full Analysis Set defined as all patients who received at least one dose of Mirdametinib.
reporting Status
POSTED
time Frame
On the 15th day of every 4 cycles (each cycle is 28 days) until disease progression is observed, assessed up to approximately 3.5 years
title
Time to Progression, From the First Dose to the First Date of a ≥ 20% Increase in Tumor Volume From Baseline.
type
OTHER_PRE_SPECIFIED
unit Of Measure
months

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

Preserved source evidence · Not medical advice