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NCT00789828 · OUTCOME

Percentage of Participants With Skin Lesions Assessed Using Physician's Global Assessement Overall Score

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
NUMBER
Unit
Percentage of participants
Interval / dispersion
95% Confidence Interval
Time frame
End of core period (Week 48), and end of extension period (up to 4 years)

What was measured

Skin lesions included hypomelanotic macules, the shagreen patch, periungual or subungual fibromas, facial angiofibromas and/or forehead plaques. Response was evaluated using the Physician's Global Assessment of Clinical Condition (PGA) on a 7-point scale: Grade 0 = complete clinical response, indicated absence of disease, Grade 1, 2, and 3 = partial response, indicated improvements of ≥ 50% but \< 100%, Grade 4, 5 = stable disease, indicated some or no improvements of 25% - \< 50% and 6 = progressive disease, indicated worse than at baseline evaluation by \> 25%. Response rate was determined for participants with ≥ 1 skin lesion at baseline, defined as the percentage of participants with overall status as complete clinical response or partial response.

Analysis population: The analysis was performed in the FAS population. Here, "Number of participants analysed" signifies the participants assessed for skin lesion response 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)72
Placebo (Core Period)38
Everolimus (Extension Period)105

Reported measurements

Source class 1
Values in Percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus (Core Period)41.7Not reported30.253.9Not reported
Placebo (Core Period)10.5Not reported2.924.8Not reported
Everolimus (Extension Period)58.1Not reported48.167.7Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        30.2
        upper Limit
        53.9
        value
        41.7
      2. group Id
        OG001
        lower Limit
        2.9
        upper Limit
        24.8
        value
        10.5
      3. group Id
        OG002
        lower Limit
        48.1
        upper Limit
        67.7
        value
        58.1
denoms
  1. counts
    1. group Id
      OG000
      value
      72
    2. group Id
      OG001
      value
      38
    3. group Id
      OG002
      value
      105
    units
    Participants
description
Skin lesions included hypomelanotic macules, the shagreen patch, periungual or subungual fibromas, facial angiofibromas and/or forehead plaques. Response was evaluated using the Physician's Global Assessment of Clinical Condition (PGA) on a 7-point scale: Grade 0 = complete clinical response, indicated absence of disease, Grade 1, 2, and 3 = partial response, indicated improvements of ≥ 50% but \< 100%, Grade 4, 5 = stable disease, indicated some or no improvements of 25% - \< 50% and 6 = progressive disease, indicated worse than at baseline evaluation by \> 25%. Response rate was determined for participants with ≥ 1 skin lesion at baseline, defined as the percentage of participants with overall status as complete clinical response or partial response.
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
NUMBER
population Description
The analysis was performed in the FAS population. Here, "Number of participants analysed" signifies the participants assessed for skin lesion response during the study for each arm, respectively.
reporting Status
POSTED
time Frame
End of core period (Week 48), and end of extension period (up to 4 years)
title
Percentage of Participants With Skin Lesions Assessed Using Physician's Global Assessement Overall Score
type
SECONDARY
unit Of Measure
Percentage of participants

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

Preserved source evidence · Not medical advice