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NCT00789828 · OUTCOME

Time to SEGA Worsening

Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1) · Source last updated 2016-02-03

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
Baseline up to week 48 (end of core period), and end of extension period (up to 4 years)

What was measured

Time to SEGA worsening was defined as the time from the start of everolimus to date of the first SEGA worsening. SEGA worsening was defined as either; increase from nadir of ≥ 25% in SEGA volume or unequivocal worsening of non-target SEGA lesions, or appearance of new SEGA lesion ≥ 1.0 cm in longest diameter, or new or worsening hydrocephalus. The median value was not reached in either treatment arm of core period as SEGA worsening was observed in less participants (everolimus - 7 and placebo - 8).

Analysis population: The analysis was performed in the FAS population. Here, "Number of participants analysed" signifies the participants assessed for time to SEGA worsening during the study for each arm, respectively.

Groups in this outcome

Everolimus (Core Period)

Participants received oral dose of everolimus 4.5 mg/m\^2 daily as an initial starting dose to attain the whole blood trough concentrations in range of 5-15 ng/mL. Dose adjustments were permitted based on safety and whole blood trough concentrations.

Placebo (Core Period)

Participants received oral dose of placebo matching to everolimus daily.

Everolimus (Extension Period)

Participants received oral dose of everolimus 4.5 mg/m\^2 daily as an initial starting dose to attain blood trough concentrations in range of 5-15 ng/mL.

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

Analysis denominator · Participants

GroupSource count
Everolimus (Core Period)78
Placebo (Core Period)39
Everolimus (Extension Period)111

Reported measurements

Source class 1
Values in months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus (Core Period)NANot reportedNANAMedian was not achieved as only 7 events of SEGA progression were observed.
Placebo (Core Period)NANot reportedNANAMedian was not achieved as only 8 events of SEGA progression were observed.
Everolimus (Extension Period)55.72Not reported55.72NAMedian upper value was not available as only single of SEGA progression were observed.
Complete source fields
classes
  1. categories
    1. measurements
      1. comment
        Median was not achieved as only 7 events of SEGA progression were observed.
        group Id
        OG000
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      2. comment
        Median was not achieved as only 8 events of SEGA progression were observed.
        group Id
        OG001
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      3. comment
        Median upper value was not available as only single of SEGA progression were observed.
        group Id
        OG002
        lower Limit
        55.72
        upper Limit
        NA
        value
        55.72
denoms
  1. counts
    1. group Id
      OG000
      value
      78
    2. group Id
      OG001
      value
      39
    3. group Id
      OG002
      value
      111
    units
    Participants
description
Time to SEGA worsening was defined as the time from the start of everolimus to date of the first SEGA worsening. SEGA worsening was defined as either; increase from nadir of ≥ 25% in SEGA volume or unequivocal worsening of non-target SEGA lesions, or appearance of new SEGA lesion ≥ 1.0 cm in longest diameter, or new or worsening hydrocephalus. The median value was not reached in either treatment arm of core period as SEGA worsening was observed in less participants (everolimus - 7 and placebo - 8).
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants received oral dose of everolimus 4.5 mg/m\^2 daily as an initial starting dose to attain the whole blood trough concentrations in range of 5-15 ng/mL. Dose adjustments were permitted based on safety and whole blood trough concentrations.
    id
    OG000
    title
    Everolimus (Core Period)
  2. description
    Participants received oral dose of placebo matching to everolimus daily.
    id
    OG001
    title
    Placebo (Core Period)
  3. description
    Participants received oral dose of everolimus 4.5 mg/m\^2 daily as an initial starting dose to attain blood trough concentrations in range of 5-15 ng/mL.
    id
    OG002
    title
    Everolimus (Extension Period)
param Type
MEDIAN
population Description
The analysis was performed in the FAS population. Here, "Number of participants analysed" signifies the participants assessed for time to SEGA worsening during the study for each arm, respectively.
reporting Status
POSTED
time Frame
Baseline up to week 48 (end of core period), and end of extension period (up to 4 years)
title
Time to SEGA Worsening
type
SECONDARY
unit Of Measure
months

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

Preserved source evidence · Not medical advice