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NCT01713946 · OUTCOME

Core Phase: Change From Baseline in the QOLIE-31-P Overall Quality-of-life Score for Patients Aged >=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-31-Problems (QOLIE-31-P) is a survey of health-related quality of life for adults with epilepsy. The QOLIE-31-P is completed by the patient. It contains 39 items, of which a total of 30 are used to make up 7 different subscales. Scores range from 0-100, with higher scores indicating a greater level of functioning and QoL. The overall quality of life score is obtained by summing a linear combination of the 7 subscale scores, where each subscale is multiplied by a relative weight that is obtained from the patient's answer to 7 items of this questionnaire.

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/mL21
Everolimus HT Target of 9 to 15 ng/mL23
Placebo23

Reported measurements

Baseline

Analysis denominator · Participants

GroupSource count
Everolimus LT Target of 3 to 7 ng/mL11
Everolimus HT Target of 9 to 15 ng/mL11
Placebo15
Values in scores on a scale · Interval/dispersion: Full Range
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL39.5Not reported1364Not reported
Everolimus HT Target of 9 to 15 ng/mL43.2Not reported589Not reported
Placebo43.6Not reported1183Not reported
End of Core Phase

Analysis denominator · Participants

GroupSource count
Everolimus LT Target of 3 to 7 ng/mL10
Everolimus HT Target of 9 to 15 ng/mL11
Placebo12
Values in scores on a scale · Interval/dispersion: Full Range
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL48.6Not reported1154Not reported
Everolimus HT Target of 9 to 15 ng/mL37.1Not reported697Not reported
Placebo54.8Not reported2690Not reported
Change from Baseline

Analysis denominator · Participants

GroupSource count
Everolimus LT Target of 3 to 7 ng/mL10
Everolimus HT Target of 9 to 15 ng/mL11
Placebo12
Values in scores on a scale · Interval/dispersion: Full Range
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL-0.5Not reported-1734Not reported
Everolimus HT Target of 9 to 15 ng/mL0.4Not reported-4720Not reported
Placebo5.3Not reported-1636Not reported

Source statistical analyses

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

  1. ci Lower Limit
    -17.9
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    12.3
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Difference in least square means
    param Value
    -2.8
  2. ci Lower Limit
    -22.0
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    6.6
    group Ids
    1. OG001
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Difference in least square means
    param Value
    -7.7
Complete source fields
analyses
  1. ci Lower Limit
    -17.9
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    12.3
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Difference in least square means
    param Value
    -2.8
  2. ci Lower Limit
    -22.0
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    6.6
    group Ids
    1. OG001
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Difference in least square means
    param Value
    -7.7
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        13
        upper Limit
        64
        value
        39.5
      2. group Id
        OG001
        lower Limit
        5
        upper Limit
        89
        value
        43.2
      3. group Id
        OG002
        lower Limit
        11
        upper Limit
        83
        value
        43.6
    denoms
    1. counts
      1. group Id
        OG000
        value
        11
      2. group Id
        OG001
        value
        11
      3. group Id
        OG002
        value
        15
      units
      Participants
    title
    Baseline
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        11
        upper Limit
        54
        value
        48.6
      2. group Id
        OG001
        lower Limit
        6
        upper Limit
        97
        value
        37.1
      3. group Id
        OG002
        lower Limit
        26
        upper Limit
        90
        value
        54.8
    denoms
    1. counts
      1. group Id
        OG000
        value
        10
      2. group Id
        OG001
        value
        11
      3. group Id
        OG002
        value
        12
      units
      Participants
    title
    End of Core Phase
  3. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        -17
        upper Limit
        34
        value
        -0.5
      2. group Id
        OG001
        lower Limit
        -47
        upper Limit
        20
        value
        0.4
      3. group Id
        OG002
        lower Limit
        -16
        upper Limit
        36
        value
        5.3
    denoms
    1. counts
      1. group Id
        OG000
        value
        10
      2. group Id
        OG001
        value
        11
      3. group Id
        OG002
        value
        12
      units
      Participants
    title
    Change from Baseline
denoms
  1. counts
    1. group Id
      OG000
      value
      21
    2. group Id
      OG001
      value
      23
    3. group Id
      OG002
      value
      23
    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-31-Problems (QOLIE-31-P) is a survey of health-related quality of life for adults with epilepsy. The QOLIE-31-P is completed by the patient. It contains 39 items, of which a total of 30 are used to make up 7 different subscales. Scores range from 0-100, with higher scores indicating a greater level of functioning and QoL. The overall quality of life score is obtained by summing a linear combination of the 7 subscale scores, where each subscale is multiplied by a relative weight that is obtained from the patient's answer to 7 items of this questionnaire.
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-31-P Overall Quality-of-life Score for Patients Aged >=18 Years
type
SECONDARY
unit Of Measure
scores on a scale

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Preserved source evidence · Not medical advice