NCT01713946 · OUTCOME

Core Phase: European Medicine Agency (EMA): Seizure Frequency Response Rate

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 of responders
Interval / dispersion: 95% Confidence Interval
Time frame: Baseline (8-week period before randomization), Week 7 to 18 (12-week maintenance period of the core phase)

What was measured

Comparison of response rates in the everolimus low-trough treatment arm (3-7 ng/mL), high-trough treatment arm (9-15 ng/mL) and placebo arm. Response means at least a 50% reduction from baseline in partial-onset seizure frequency during the maintenance period of the core phase.

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

Group	Source count
Everolimus LT Target of 3 to 7 ng/mL	117
Everolimus HT Target of 9 to 15 ng/mL	130
Placebo	119

Reported measurements

Source class 1

Values in Percentage of responders · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Everolimus LT Target of 3 to 7 ng/mL	28.2	Not reported	20.3	37.3	Not reported
Everolimus HT Target of 9 to 15 ng/mL	40.0	Not reported	31.5	49.0	Not reported
Placebo	15.1	Not reported	9.2	22.8	Not reported

Source statistical analyses

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

ci Lower Limit
1.16
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
4.20
group Ids
OG000
OG002
non Inferiority Type
SUPERIORITY
p Value
0.008
param Type
Odds Ratio (OR)
param Value
2.21
statistical Method
Bonferroni-Holm
ci Lower Limit
2.10
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
7.32
group Ids
OG001
OG002
non Inferiority Type
SUPERIORITY
p Value
<0.001
param Type
Odds Ratio (OR)
param Value
3.93
statistical Method
Bonferroni-Holm

Complete source fields

analyses

ci Lower Limit
1.16
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
4.20
group Ids
OG000
OG002
non Inferiority Type
SUPERIORITY
p Value
0.008
param Type
Odds Ratio (OR)
param Value
2.21
statistical Method
Bonferroni-Holm
ci Lower Limit
2.10
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
7.32
group Ids
OG001
OG002
non Inferiority Type
SUPERIORITY
p Value
<0.001
param Type
Odds Ratio (OR)
param Value
3.93
statistical Method
Bonferroni-Holm

classes

categories
measurements
group Id
OG000
lower Limit
20.3
upper Limit
37.3
value
28.2
group Id
OG001
lower Limit
31.5
upper Limit
49.0
value
40.0
group Id
OG002
lower Limit
9.2
upper Limit
22.8
value
15.1

denoms

counts
group Id
OG000
value
117
group Id
OG001
value
130
group Id
OG002
value
119
units
Participants

description

dispersion Type

95% Confidence Interval

groups

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
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
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

Baseline (8-week period before randomization), Week 7 to 18 (12-week maintenance period of the core phase)

title

Core Phase: European Medicine Agency (EMA): Seizure Frequency Response Rate

type

PRIMARY

unit Of Measure

Percentage of responders

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Preserved source evidence · Not medical advice