NCT01713946 · OUTCOME
Long Term Evaluation: Effect of Everolimus Over Time in the Overall 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, Weeks 18, 42, 66 and 90
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 Long Term Evaluation (LTE) efficacy set consists of all patients who received at least one dose of everolimus and had at least one efficacy assessment while on everolimus.
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
| Group | Source count |
|---|
| Everolimus LT Target of 3 to 7 ng/mL | 0 |
|---|
| Everolimus HT Target of 9 to 15 ng/mL | 0 |
|---|
| Placebo | 0 |
|---|
| Everolimus Long Term Evaluation (LTE) | 265 |
|---|
Reported measurements
Baseline
Analysis denominator · Participants
| Group | Source count |
|---|
| Everolimus LT Target of 3 to 7 ng/mL | 0 |
|---|
| Everolimus HT Target of 9 to 15 ng/mL | 0 |
|---|
| Placebo | 0 |
|---|
| Everolimus Long Term Evaluation (LTE) | 151 |
|---|
Values in scores on a scale · Interval/dispersion: Full Range| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Everolimus Long Term Evaluation (LTE) | -0.53 | Not reported | -2.3 | 10.1 | Not reported |
|---|
Week 18
Analysis denominator · Participants
| Group | Source count |
|---|
| Everolimus LT Target of 3 to 7 ng/mL | 0 |
|---|
| Everolimus HT Target of 9 to 15 ng/mL | 0 |
|---|
| Placebo | 0 |
|---|
| Everolimus Long Term Evaluation (LTE) | 160 |
|---|
Values in scores on a scale · Interval/dispersion: Full Range| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Everolimus Long Term Evaluation (LTE) | -0.44 | Not reported | -2.3 | 9.5 | Not reported |
|---|
Week 42
Analysis denominator · Participants
| Group | Source count |
|---|
| Everolimus LT Target of 3 to 7 ng/mL | 0 |
|---|
| Everolimus HT Target of 9 to 15 ng/mL | 0 |
|---|
| Placebo | 0 |
|---|
| Everolimus Long Term Evaluation (LTE) | 139 |
|---|
Values in scores on a scale · Interval/dispersion: Full Range| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Everolimus Long Term Evaluation (LTE) | -0.36 | Not reported | -2.3 | 10.8 | Not reported |
|---|
Week 66
Analysis denominator · Participants
| Group | Source count |
|---|
| Everolimus LT Target of 3 to 7 ng/mL | 0 |
|---|
| Everolimus HT Target of 9 to 15 ng/mL | 0 |
|---|
| Placebo | 0 |
|---|
| Everolimus Long Term Evaluation (LTE) | 104 |
|---|
Values in scores on a scale · Interval/dispersion: Full Range| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Everolimus Long Term Evaluation (LTE) | 0.13 | Not reported | -2.3 | 9.1 | Not reported |
|---|
Week 90
Analysis denominator · Participants
| Group | Source count |
|---|
| Everolimus LT Target of 3 to 7 ng/mL | 0 |
|---|
| Everolimus HT Target of 9 to 15 ng/mL | 0 |
|---|
| Placebo | 0 |
|---|
| Everolimus Long Term Evaluation (LTE) | 82 |
|---|
Values in scores on a scale · Interval/dispersion: Full Range| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Everolimus Long Term Evaluation (LTE) | -0.06 | Not reported | -2.3 | 9.0 | Not reported |
|---|
Complete source fields
- classes
- categories
- measurements
- group Id
- OG003
- lower Limit
- -2.3
- upper Limit
- 10.1
- value
- -0.53
- denoms
- counts
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- group Id
- OG002
- value
- 0
- group Id
- OG003
- value
- 151
- units
- Participants
- title
- Baseline
- categories
- measurements
- group Id
- OG003
- lower Limit
- -2.3
- upper Limit
- 9.5
- value
- -0.44
- denoms
- counts
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- group Id
- OG002
- value
- 0
- group Id
- OG003
- value
- 160
- units
- Participants
- title
- Week 18
- categories
- measurements
- group Id
- OG003
- lower Limit
- -2.3
- upper Limit
- 10.8
- value
- -0.36
- denoms
- counts
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- group Id
- OG002
- value
- 0
- group Id
- OG003
- value
- 139
- units
- Participants
- title
- Week 42
- categories
- measurements
- group Id
- OG003
- lower Limit
- -2.3
- upper Limit
- 9.1
- value
- 0.13
- denoms
- counts
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- group Id
- OG002
- value
- 0
- group Id
- OG003
- value
- 104
- units
- Participants
- title
- Week 66
- categories
- measurements
- group Id
- OG003
- lower Limit
- -2.3
- upper Limit
- 9.0
- value
- -0.06
- denoms
- counts
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- group Id
- OG002
- value
- 0
- group Id
- OG003
- value
- 82
- units
- Participants
- title
- Week 90
- denoms
- counts
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- group Id
- OG002
- value
- 0
- group Id
- OG003
- value
- 265
- 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
- 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
- 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 Long Term Evaluation (LTE) efficacy set consists of all patients who received at least one dose of everolimus and had at least one efficacy assessment while on everolimus.
- reporting Status
- POSTED
- time Frame
- Baseline, Weeks 18, 42, 66 and 90
- title
- Long Term Evaluation: Effect of Everolimus Over Time in the Overall Wechsler Nonverbal Composite Score
- type
- SECONDARY
- unit Of Measure
- scores on a scale
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