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NCT01713946 · OUTCOME

Core Phase: Change From Baseline in the QOLIE-AD-48 Overall Quality-of-life Score for Patients >=11 to 18 Years

A Placebo-controlled Study of Efficacy & Safety of 2 Trough-ranges of Everolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Partial-onset Seizures · Source last updated 2018-11-07

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
MEDIAN
Unit
scores on a scale
Interval / dispersion
Full Range
Time frame
Baseline, Week 18

What was measured

Comparison of quality of life (from 3 age specific questionnaires) in the everolimus low-trough treatment arm (3-7 ng/mL), hightrough treatment arm (9-15 ng/mL) and placebo arm at the end of the core phase. The Quality of Life in Epilepsy Inventory for Adolescents-48 (QOLIE-AD-48) is a survey of health-related quality of life for adolescents 11 to 18 years of age with epilepsy. The QOLIE-AD-48 is completed by the patient. It contains 48 items which assess 8 subscales. Scores range from 0-100, with higher scores corresponding to improved QoL. The overall quality of life score is obtained by summing a linear combination of the 8 subscale scores, where each subscale is multiplied by a relative weight that is provided in the original publication.

Analysis population: The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization

Groups in this outcome

Everolimus LT Target of 3 to 7 ng/mL

Participants were randomized to receive everolimus dispersible tablets for oral suspension with titration to a low trough (LT) range of 3 to 7 ng/mL

Everolimus HT Target of 9 to 15 ng/mL

Participants were randomized to receive everolimus dispersible tablets for oral suspension with titration to a high trough (HT) range of 9 to 15 ng/mL

Placebo

Participants were randomized to receive placebo dispersible tablets for oral suspension.

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 LT Target of 3 to 7 ng/mL31
Everolimus HT Target of 9 to 15 ng/mL38
Placebo33

Reported measurements

Baseline

Analysis denominator · Participants

GroupSource count
Everolimus LT Target of 3 to 7 ng/mL13
Everolimus HT Target of 9 to 15 ng/mL15
Placebo14
Values in scores on a scale · Interval/dispersion: Full Range
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL56.1Not reported4386Not reported
Everolimus HT Target of 9 to 15 ng/mL58.8Not reported3581Not reported
Placebo59.6Not reported1388Not reported
End of Core Phase

Analysis denominator · Participants

GroupSource count
Everolimus LT Target of 3 to 7 ng/mL13
Everolimus HT Target of 9 to 15 ng/mL13
Placebo12
Values in scores on a scale · Interval/dispersion: Full Range
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL58.5Not reported4789Not reported
Everolimus HT Target of 9 to 15 ng/mL60.7Not reported4674Not reported
Placebo65.4Not reported2987Not reported
Change from Baseline

Analysis denominator · Participants

GroupSource count
Everolimus LT Target of 3 to 7 ng/mL12
Everolimus HT Target of 9 to 15 ng/mL13
Placebo12
Values in scores on a scale · Interval/dispersion: Full Range
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL4.7Not reported-1111Not reported
Everolimus HT Target of 9 to 15 ng/mL5.8Not reported-1338Not reported
Placebo7.2Not reported-1825Not reported

Source statistical analyses

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

  1. ci Lower Limit
    -10.5
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    6.2
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Difference in least square means
    param Value
    -2.1
  2. ci Lower Limit
    -7.8
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    8.6
    group Ids
    1. OG001
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Difference in least square means
    param Value
    0.4
Complete source fields
analyses
  1. ci Lower Limit
    -10.5
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    6.2
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Difference in least square means
    param Value
    -2.1
  2. ci Lower Limit
    -7.8
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    8.6
    group Ids
    1. OG001
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Difference in least square means
    param Value
    0.4
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        43
        upper Limit
        86
        value
        56.1
      2. group Id
        OG001
        lower Limit
        35
        upper Limit
        81
        value
        58.8
      3. group Id
        OG002
        lower Limit
        13
        upper Limit
        88
        value
        59.6
    denoms
    1. counts
      1. group Id
        OG000
        value
        13
      2. group Id
        OG001
        value
        15
      3. group Id
        OG002
        value
        14
      units
      Participants
    title
    Baseline
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        47
        upper Limit
        89
        value
        58.5
      2. group Id
        OG001
        lower Limit
        46
        upper Limit
        74
        value
        60.7
      3. group Id
        OG002
        lower Limit
        29
        upper Limit
        87
        value
        65.4
    denoms
    1. counts
      1. group Id
        OG000
        value
        13
      2. group Id
        OG001
        value
        13
      3. group Id
        OG002
        value
        12
      units
      Participants
    title
    End of Core Phase
  3. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        -11
        upper Limit
        11
        value
        4.7
      2. group Id
        OG001
        lower Limit
        -13
        upper Limit
        38
        value
        5.8
      3. group Id
        OG002
        lower Limit
        -18
        upper Limit
        25
        value
        7.2
    denoms
    1. counts
      1. group Id
        OG000
        value
        12
      2. group Id
        OG001
        value
        13
      3. group Id
        OG002
        value
        12
      units
      Participants
    title
    Change from Baseline
denoms
  1. counts
    1. group Id
      OG000
      value
      31
    2. group Id
      OG001
      value
      38
    3. group Id
      OG002
      value
      33
    units
    Participants
description
Comparison of quality of life (from 3 age specific questionnaires) in the everolimus low-trough treatment arm (3-7 ng/mL), hightrough treatment arm (9-15 ng/mL) and placebo arm at the end of the core phase. The Quality of Life in Epilepsy Inventory for Adolescents-48 (QOLIE-AD-48) is a survey of health-related quality of life for adolescents 11 to 18 years of age with epilepsy. The QOLIE-AD-48 is completed by the patient. It contains 48 items which assess 8 subscales. Scores range from 0-100, with higher scores corresponding to improved QoL. The overall quality of life score is obtained by summing a linear combination of the 8 subscale scores, where each subscale is multiplied by a relative weight that is provided in the original publication.
dispersion Type
Full Range
groups
  1. description
    Participants were randomized to receive everolimus dispersible tablets for oral suspension with titration to a low trough (LT) range of 3 to 7 ng/mL
    id
    OG000
    title
    Everolimus LT Target of 3 to 7 ng/mL
  2. description
    Participants were randomized to receive everolimus dispersible tablets for oral suspension with titration to a high trough (HT) range of 9 to 15 ng/mL
    id
    OG001
    title
    Everolimus HT Target of 9 to 15 ng/mL
  3. description
    Participants were randomized to receive placebo dispersible tablets for oral suspension.
    id
    OG002
    title
    Placebo
param Type
MEDIAN
population Description
The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization
reporting Status
POSTED
time Frame
Baseline, Week 18
title
Core Phase: Change From Baseline in the QOLIE-AD-48 Overall Quality-of-life Score for Patients >=11 to 18 Years
type
SECONDARY
unit Of Measure
scores on a scale

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Preserved source evidence · Not medical advice