NCT01713946 · OUTCOME

Percentage of Seizure-free Patients During the Maintenance Period of the Core Phase

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 seizure-free participants
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 seizure freedom (100% reduction in seizure frequency) in the everolimus low-trough treatment arm (3-7 ng/mL), high-trough treatment arm (9-15 ng/mL) and placebo arm during maintenance period of the core phase. Seizure free means a 100% reduction from baseline in partial-onset seizure frequency during 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 seizure-free participants · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Everolimus LT Target of 3 to 7 ng/mL	5.1	Not reported	1.9	10.8	Not reported
Everolimus HT Target of 9 to 15 ng/mL	3.8	Not reported	1.3	8.7	Not reported
Placebo	0.8	Not reported	0.0	4.6	Not reported

Source statistical analyses

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

ci Lower Limit
0.77
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
55.73
group Ids
OG000
OG002
non Inferiority Type
SUPERIORITY
param Type
Odds Ratio (OR)
param Value
6.55
ci Lower Limit
0.57
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
44.03
group Ids
OG001
OG002
non Inferiority Type
SUPERIORITY
param Type
Odds Ratio (OR)
param Value
4.99

Complete source fields

analyses

ci Lower Limit
0.77
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
55.73
group Ids
OG000
OG002
non Inferiority Type
SUPERIORITY
param Type
Odds Ratio (OR)
param Value
6.55
ci Lower Limit
0.57
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
44.03
group Ids
OG001
OG002
non Inferiority Type
SUPERIORITY
param Type
Odds Ratio (OR)
param Value
4.99

classes

categories
measurements
group Id
OG000
lower Limit
1.9
upper Limit
10.8
value
5.1
group Id
OG001
lower Limit
1.3
upper Limit
8.7
value
3.8
group Id
OG002
lower Limit
0.0
upper Limit
4.6
value
0.8

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

Percentage of Seizure-free Patients During the Maintenance Period of the Core Phase

type

SECONDARY

unit Of Measure

Percentage of seizure-free participants

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

Preserved source evidence · Not medical advice