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NCT01713946 · OUTCOME

Core Phase: Change From Baseline in Wechsler Nonverbal Composite Score

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

The brief version of the WNV consists of a 2-subtest battery: only Matrices and Recognition subtests for patients under 8, and Matrices and Spatial Span subtests for patients aged 8 to 21. Based on the raw scores obtained from the subtests, standardized z-scores were calculated for each subtest using the following formula: Zscore = (X - b)/Sb where X is the raw score of the subtest, b and Sb represent the mean and standard deviation respectively of the subtest score recorded at baseline for the study population. The composite WNV score was computed by summing up the Z-scores of the 3 subtests of the WNV (i.e. matrices, recognition, and coding for patients aged \<8 years and matrices, spatial span, and coding for patients aged 8 to 21 years). The composite WNV score has no range.

Analysis population: The safety Set comprised all patients who received at least one dose of study treatment and had at least one post-baseline safety assessment in the Core phase (where the statement that a patient had no AE constitutes a safety assessment.

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.

Everolimus Long Term Evaluation (LTE)

Participants who were treated with everolimus either in the Core or Extension phases of the study and were evaluated for longer term safety and efficacy.

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/mL89
Everolimus HT Target of 9 to 15 ng/mL94
Placebo85
Everolimus Long Term Evaluation (LTE)0

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/mL63
Placebo52
Everolimus Long Term Evaluation (LTE)0
Values in scores on a scale · Interval/dispersion: Full Range
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL-0.48Not reported-2.38.1Not reported
Everolimus HT Target of 9 to 15 ng/mL-0.69Not reported-2.39.6Not reported
Placebo-1.11Not reported-2.38.3Not reported
End of Core Phase

Analysis denominator · Participants

GroupSource count
Everolimus LT Target of 3 to 7 ng/mL60
Everolimus HT Target of 9 to 15 ng/mL63
Placebo48
Everolimus Long Term Evaluation (LTE)0
Values in scores on a scale · Interval/dispersion: Full Range
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL-0.04Not reported-2.38.1Not reported
Everolimus HT Target of 9 to 15 ng/mL-0.89Not reported-2.37.5Not reported
Placebo-0.51Not reported-2.310.1Not reported
Change from Baseline

Analysis denominator · Participants

GroupSource count
Everolimus LT Target of 3 to 7 ng/mL58
Everolimus HT Target of 9 to 15 ng/mL59
Placebo46
Everolimus Long Term Evaluation (LTE)0
Values in scores on a scale · Interval/dispersion: Full Range
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL0.00Not reported-2.51.6Not reported
Everolimus HT Target of 9 to 15 ng/mL0.00Not reported-10.02.1Not reported
Placebo0.00Not reported-3.31.7Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        -2.3
        upper Limit
        8.1
        value
        -0.48
      2. group Id
        OG001
        lower Limit
        -2.3
        upper Limit
        9.6
        value
        -0.69
      3. group Id
        OG002
        lower Limit
        -2.3
        upper Limit
        8.3
        value
        -1.11
    denoms
    1. counts
      1. group Id
        OG000
        value
        63
      2. group Id
        OG001
        value
        63
      3. group Id
        OG002
        value
        52
      4. group Id
        OG003
        value
        0
      units
      Participants
    title
    Baseline
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        -2.3
        upper Limit
        8.1
        value
        -0.04
      2. group Id
        OG001
        lower Limit
        -2.3
        upper Limit
        7.5
        value
        -0.89
      3. group Id
        OG002
        lower Limit
        -2.3
        upper Limit
        10.1
        value
        -0.51
    denoms
    1. counts
      1. group Id
        OG000
        value
        60
      2. group Id
        OG001
        value
        63
      3. group Id
        OG002
        value
        48
      4. group Id
        OG003
        value
        0
      units
      Participants
    title
    End of Core Phase
  3. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        -2.5
        upper Limit
        1.6
        value
        0.00
      2. group Id
        OG001
        lower Limit
        -10.0
        upper Limit
        2.1
        value
        0.00
      3. group Id
        OG002
        lower Limit
        -3.3
        upper Limit
        1.7
        value
        0.00
    denoms
    1. counts
      1. group Id
        OG000
        value
        58
      2. group Id
        OG001
        value
        59
      3. group Id
        OG002
        value
        46
      4. group Id
        OG003
        value
        0
      units
      Participants
    title
    Change from Baseline
denoms
  1. counts
    1. group Id
      OG000
      value
      89
    2. group Id
      OG001
      value
      94
    3. group Id
      OG002
      value
      85
    4. group Id
      OG003
      value
      0
    units
    Participants
description
The brief version of the WNV consists of a 2-subtest battery: only Matrices and Recognition subtests for patients under 8, and Matrices and Spatial Span subtests for patients aged 8 to 21. Based on the raw scores obtained from the subtests, standardized z-scores were calculated for each subtest using the following formula: Zscore = (X - b)/Sb where X is the raw score of the subtest, b and Sb represent the mean and standard deviation respectively of the subtest score recorded at baseline for the study population. The composite WNV score was computed by summing up the Z-scores of the 3 subtests of the WNV (i.e. matrices, recognition, and coding for patients aged \<8 years and matrices, spatial span, and coding for patients aged 8 to 21 years). The composite WNV score has no range.
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
  4. description
    Participants who were treated with everolimus either in the Core or Extension phases of the study and were evaluated for longer term safety and efficacy.
    id
    OG003
    title
    Everolimus Long Term Evaluation (LTE)
param Type
MEDIAN
population Description
The safety Set comprised all patients who received at least one dose of study treatment and had at least one post-baseline safety assessment in the Core phase (where the statement that a patient had no AE constitutes a safety assessment.
reporting Status
POSTED
time Frame
Baseline, Week 18
title
Core Phase: Change From Baseline in Wechsler Nonverbal Composite Score
type
SECONDARY
unit Of Measure
scores on a scale

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

Preserved source evidence · Not medical advice