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NCT01713946 · OUTCOME

Core Phase: Probability That a Patient Remains On-treatment up to a Specified Time Point

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
NUMBER
Unit
Percentage event-free prob. estimates
Interval / dispersion
95% Confidence Interval
Time frame
Week 6, Week 12, Week 18

What was measured

Comparison of time to treatment discontinuation in the everolimus low-trough treatment arm (3-7 ng/mL), high-trough treatment arm (9-15 ng/mL) and placebo arm during the core phase. Treatment duration is defined as the time from randomization until the date of permanent study treatment discontinuation (for any reason) at any time during the Core phase. The percentage event-free probability estimate is the estimated probability that a patient will remain on-treatment up to a specified time point (Week 6, 12, 18)

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/mL117
Everolimus HT Target of 9 to 15 ng/mL130
Placebo119

Reported measurements

Week 6
Values in Percentage event-free prob. estimates · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL97.4Not reported92.399.2Not reported
Everolimus HT Target of 9 to 15 ng/mL96.2Not reported91.098.4Not reported
Placebo99.2Not reported94.299.9Not reported
Week 12
Values in Percentage event-free prob. estimates · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL95.7Not reported90.098.2Not reported
Everolimus HT Target of 9 to 15 ng/mL95.4Not reported90.097.9Not reported
Placebo97.5Not reported92.499.2Not reported
Week 18
Values in Percentage event-free prob. estimates · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus LT Target of 3 to 7 ng/mL70.1Not reported60.977.5Not reported
Everolimus HT Target of 9 to 15 ng/mL71.5Not reported62.978.5Not reported
Placebo75.6Not reported66.982.4Not reported

Source statistical analyses

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

  1. ci Lower Limit
    0.77
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    2.07
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Hazard Ratio (HR)
    param Value
    1.27
  2. ci Lower Limit
    0.74
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    1.96
    group Ids
    1. OG001
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Hazard Ratio (HR)
    param Value
    1.20
Complete source fields
analyses
  1. ci Lower Limit
    0.77
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    2.07
    group Ids
    1. OG000
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Hazard Ratio (HR)
    param Value
    1.27
  2. ci Lower Limit
    0.74
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    1.96
    group Ids
    1. OG001
    2. OG002
    non Inferiority Type
    SUPERIORITY
    param Type
    Hazard Ratio (HR)
    param Value
    1.20
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        92.3
        upper Limit
        99.2
        value
        97.4
      2. group Id
        OG001
        lower Limit
        91.0
        upper Limit
        98.4
        value
        96.2
      3. group Id
        OG002
        lower Limit
        94.2
        upper Limit
        99.9
        value
        99.2
    title
    Week 6
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        90.0
        upper Limit
        98.2
        value
        95.7
      2. group Id
        OG001
        lower Limit
        90.0
        upper Limit
        97.9
        value
        95.4
      3. group Id
        OG002
        lower Limit
        92.4
        upper Limit
        99.2
        value
        97.5
    title
    Week 12
  3. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        60.9
        upper Limit
        77.5
        value
        70.1
      2. group Id
        OG001
        lower Limit
        62.9
        upper Limit
        78.5
        value
        71.5
      3. group Id
        OG002
        lower Limit
        66.9
        upper Limit
        82.4
        value
        75.6
    title
    Week 18
denoms
  1. counts
    1. group Id
      OG000
      value
      117
    2. group Id
      OG001
      value
      130
    3. group Id
      OG002
      value
      119
    units
    Participants
description
Comparison of time to treatment discontinuation in the everolimus low-trough treatment arm (3-7 ng/mL), high-trough treatment arm (9-15 ng/mL) and placebo arm during the core phase. Treatment duration is defined as the time from randomization until the date of permanent study treatment discontinuation (for any reason) at any time during the Core phase. The percentage event-free probability estimate is the estimated probability that a patient will remain on-treatment up to a specified time point (Week 6, 12, 18)
dispersion Type
95% Confidence Interval
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
NUMBER
population Description
The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization
reporting Status
POSTED
time Frame
Week 6, Week 12, Week 18
title
Core Phase: Probability That a Patient Remains On-treatment up to a Specified Time Point
type
SECONDARY
unit Of Measure
Percentage event-free prob. estimates

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Preserved source evidence · Not medical advice