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NCT00790400 · OUTCOME

Angiomyolipoma Response Rate as Per Central Radiology Review

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) · Source last updated 2017-02-17

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
From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to 5.7 years

What was measured

Angiomyolipoma response defined as the combination of the following criteria: reduction in angiomyolipoma volume of ≥ 50% relative to baseline, where angiomyolipoma volume was sum of volumes of all target lesions identified at baseline, and with a confirmatory scan performed approximately 12 weeks later (no sooner than 8 weeks later); no new angiomyolipoma lesions ≥ 1.0 cm in longest diameter were identified; there were no kidney increases in volume \> 20% from nadir. The patient did not have any angiomyolipoma-related bleeding of ≥ grade 2. For the everolimus (core/extension periods) treatment group, the baseline means the latest value on or before starting everolimus.

Analysis population: The Full Analysis Set (FAS) is defined according to the Intention-to-Treat principle and consists of all randomized patients.

Groups in this outcome

Everolimus Randomized (Core Period)

Study drug was given by continuous oral daily dosing of two 5 mg tablets.

Placebo Randomized (Core Period)

Placebo was given by continuous oral daily dosing of two 5 mg tablets.

Everolimus (Core and/or Extension Period)

Patients initially randomized in everolimus and patients initially randomized in placebo but who crossed-over to everolimus during extension.

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 Randomized (Core Period)79
Placebo Randomized (Core Period)39
Everolimus (Core and/or Extension Period)112

Reported measurements

Source class 1
Values in Percentage of Participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus Randomized (Core Period)41.8Not reported30.853.4Not reported
Placebo Randomized (Core Period)0Not reported0.09.0Not reported
Everolimus (Core and/or Extension Period)58.0Not reported48.367.3Not 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_OR_OTHER
    p Value
    <0.0001
    statistical Method
    Clopper-Pearson
    tested Non Inferiority
    false
Complete source fields
analyses
  1. group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY_OR_OTHER
    p Value
    <0.0001
    statistical Method
    Clopper-Pearson
    tested Non Inferiority
    false
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        30.8
        upper Limit
        53.4
        value
        41.8
      2. group Id
        OG001
        lower Limit
        0.0
        upper Limit
        9.0
        value
        0
      3. group Id
        OG002
        lower Limit
        48.3
        upper Limit
        67.3
        value
        58.0
denoms
  1. counts
    1. group Id
      OG000
      value
      79
    2. group Id
      OG001
      value
      39
    3. group Id
      OG002
      value
      112
    units
    Participants
description
Angiomyolipoma response defined as the combination of the following criteria: reduction in angiomyolipoma volume of ≥ 50% relative to baseline, where angiomyolipoma volume was sum of volumes of all target lesions identified at baseline, and with a confirmatory scan performed approximately 12 weeks later (no sooner than 8 weeks later); no new angiomyolipoma lesions ≥ 1.0 cm in longest diameter were identified; there were no kidney increases in volume \> 20% from nadir. The patient did not have any angiomyolipoma-related bleeding of ≥ grade 2. For the everolimus (core/extension periods) treatment group, the baseline means the latest value on or before starting everolimus.
dispersion Type
95% Confidence Interval
groups
  1. description
    Study drug was given by continuous oral daily dosing of two 5 mg tablets.
    id
    OG000
    title
    Everolimus Randomized (Core Period)
  2. description
    Placebo was given by continuous oral daily dosing of two 5 mg tablets.
    id
    OG001
    title
    Placebo Randomized (Core Period)
  3. description
    Patients initially randomized in everolimus and patients initially randomized in placebo but who crossed-over to everolimus during extension.
    id
    OG002
    title
    Everolimus (Core and/or Extension Period)
param Type
NUMBER
population Description
The Full Analysis Set (FAS) is defined according to the Intention-to-Treat principle and consists of all randomized patients.
reporting Status
POSTED
time Frame
From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or up to 5.7 years
title
Angiomyolipoma Response Rate as Per Central Radiology Review
type
PRIMARY
unit Of Measure
Percentage of Participants

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

Preserved source evidence · Not medical advice