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NCT01713946 · OUTCOME

Core Phase: Change From Baseline in the QOLCE Overall Quality-of-life Score for Patients <11 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 in the everolimus (from 3 age specific questionnaires) low-trough treatment arm (3-7 ng/mL), high-trough treatment arm (9-15 ng/mL) and placebo arm at the end of the core phase. The Quality of Life Childhood Epilepsy (QOLCE) questionnaire, used for patients \< 11 years at baseline, was completed by the patient's parent or caregiver. It consists of 16 subscales (13 multi-item scales and 3 single item scales) and one overall quality-of-life score. Scores range from 0-100, with higher scores corresponding to improved QoL. The Overall Quality of Life Score is computed by adding each subscale score for each individual and then dividing by 16.

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/mL65
Everolimus HT Target of 9 to 15 ng/mL69
Placebo63

Reported measurements

Baseline

Analysis denominator · Participants

GroupSource count
Everolimus LT Target of 3 to 7 ng/mL63
Everolimus HT Target of 9 to 15 ng/mL68
Placebo55
Values in scores on a scale · Interval/dispersion: Full Range
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL52.3Not reported2675Not reported
Everolimus HT Target of 9 to 15 ng/mL56.5Not reported2676Not reported
Placebo55.3Not reported3374Not reported
End of Core Phase

Analysis denominator · Participants

GroupSource count
Everolimus LT Target of 3 to 7 ng/mL56
Everolimus HT Target of 9 to 15 ng/mL56
Placebo50
Values in scores on a scale · Interval/dispersion: Full Range
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL53.6Not reported2479Not reported
Everolimus HT Target of 9 to 15 ng/mL59.5Not reported3678Not reported
Placebo57.2Not reported2077Not reported
Change from Baseline

Analysis denominator · Participants

GroupSource count
Everolimus LT Target of 3 to 7 ng/mL55
Everolimus HT Target of 9 to 15 ng/mL56
Placebo47
Values in scores on a scale · Interval/dispersion: Full Range
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL0.2Not reported-2524Not reported
Everolimus HT Target of 9 to 15 ng/mL0.0Not reported-1918Not reported
Placebo1.0Not reported-1828Not reported

Source statistical analyses

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

  1. ci Lower Limit
    -4.4
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    2.1
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Difference in least square means
    param Value
    -1.1
  2. ci Lower Limit
    -2.2
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    4.3
    group Ids
    1. OG001
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Difference in least square means
    param Value
    1.0
Complete source fields
analyses
  1. ci Lower Limit
    -4.4
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    2.1
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Difference in least square means
    param Value
    -1.1
  2. ci Lower Limit
    -2.2
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    4.3
    group Ids
    1. OG001
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Difference in least square means
    param Value
    1.0
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        26
        upper Limit
        75
        value
        52.3
      2. group Id
        OG001
        lower Limit
        26
        upper Limit
        76
        value
        56.5
      3. group Id
        OG002
        lower Limit
        33
        upper Limit
        74
        value
        55.3
    denoms
    1. counts
      1. group Id
        OG000
        value
        63
      2. group Id
        OG001
        value
        68
      3. group Id
        OG002
        value
        55
      units
      Participants
    title
    Baseline
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        24
        upper Limit
        79
        value
        53.6
      2. group Id
        OG001
        lower Limit
        36
        upper Limit
        78
        value
        59.5
      3. group Id
        OG002
        lower Limit
        20
        upper Limit
        77
        value
        57.2
    denoms
    1. counts
      1. group Id
        OG000
        value
        56
      2. group Id
        OG001
        value
        56
      3. group Id
        OG002
        value
        50
      units
      Participants
    title
    End of Core Phase
  3. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        -25
        upper Limit
        24
        value
        0.2
      2. group Id
        OG001
        lower Limit
        -19
        upper Limit
        18
        value
        0.0
      3. group Id
        OG002
        lower Limit
        -18
        upper Limit
        28
        value
        1.0
    denoms
    1. counts
      1. group Id
        OG000
        value
        55
      2. group Id
        OG001
        value
        56
      3. group Id
        OG002
        value
        47
      units
      Participants
    title
    Change from Baseline
denoms
  1. counts
    1. group Id
      OG000
      value
      65
    2. group Id
      OG001
      value
      69
    3. group Id
      OG002
      value
      63
    units
    Participants
description
Comparison of quality of life in the everolimus (from 3 age specific questionnaires) low-trough treatment arm (3-7 ng/mL), high-trough treatment arm (9-15 ng/mL) and placebo arm at the end of the core phase. The Quality of Life Childhood Epilepsy (QOLCE) questionnaire, used for patients \< 11 years at baseline, was completed by the patient's parent or caregiver. It consists of 16 subscales (13 multi-item scales and 3 single item scales) and one overall quality-of-life score. Scores range from 0-100, with higher scores corresponding to improved QoL. The Overall Quality of Life Score is computed by adding each subscale score for each individual and then dividing by 16.
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 QOLCE Overall Quality-of-life Score for Patients <11 Years
type
SECONDARY
unit Of Measure
scores on a scale

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

Preserved source evidence · Not medical advice