NCT01713946 · OUTCOME
Long Term Evaluation: Effect of Everolimus Over Time in the Overall Vineland-II Adaptive Behavior Composite (ABC) 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
Comparison of adaptive functioning using the VABS-II composite score in the everolimus low-trough treatment arm (3-7 ng/mL), high-trough treatment arm (9-15 ng/mL) and placebo arm. The Vineland II assesses an individual's development of personal independence \& social responsibility. The questionnaire contains 433 items which assess 15 subdomains organized into the five domains of Communication, Daily Living Skills, Socialization, Motor Skills and Maladaptive Behavior. The overall Adaptive Behavior Composite (ABC) score is obtained by summing the standard scores of the first four domain scores for patients aged less than 7 years, or the first 3 domain scores for patients aged 7 or older (the Maladaptive Behavior domain is optional). The ABC standard score ranges from 20 to 160 with a mean of 100 and a standard deviation of 15. Higher scores correspond to improved adaptive level. Note that 2 questionnaires with ABC scores\<20 (data issues) were included in this analysis.
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) | 239 |
|---|
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) | 88 |
|---|
Values in scores on a scale · Interval/dispersion: Full Range| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Everolimus Long Term Evaluation (LTE) | 56.00 | Not reported | 20.0 | 112.0 | 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) | 94 |
|---|
Values in scores on a scale · Interval/dispersion: Full Range| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Everolimus Long Term Evaluation (LTE) | 53.50 | Not reported | 4.0 | 121.0 | 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) | 94 |
|---|
Values in scores on a scale · Interval/dispersion: Full Range| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Everolimus Long Term Evaluation (LTE) | 55.50 | Not reported | 20.0 | 111.0 | 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) | 88 |
|---|
Values in scores on a scale · Interval/dispersion: Full Range| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Everolimus Long Term Evaluation (LTE) | 57.00 | Not reported | 20.0 | 112.0 | 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) | 66 |
|---|
Values in scores on a scale · Interval/dispersion: Full Range| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Everolimus Long Term Evaluation (LTE) | 49.50 | Not reported | 20.0 | 150.0 | Not reported |
|---|
Complete source fields
- classes
- categories
- measurements
- group Id
- OG003
- lower Limit
- 20.0
- upper Limit
- 112.0
- value
- 56.00
- denoms
- counts
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- group Id
- OG002
- value
- 0
- group Id
- OG003
- value
- 88
- units
- Participants
- title
- Baseline
- categories
- measurements
- group Id
- OG003
- lower Limit
- 4.0
- upper Limit
- 121.0
- value
- 53.50
- denoms
- counts
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- group Id
- OG002
- value
- 0
- group Id
- OG003
- value
- 94
- units
- Participants
- title
- Week 18
- categories
- measurements
- group Id
- OG003
- lower Limit
- 20.0
- upper Limit
- 111.0
- value
- 55.50
- denoms
- counts
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- group Id
- OG002
- value
- 0
- group Id
- OG003
- value
- 94
- units
- Participants
- title
- Week 42
- categories
- measurements
- group Id
- OG003
- lower Limit
- 20.0
- upper Limit
- 112.0
- value
- 57.00
- denoms
- counts
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- group Id
- OG002
- value
- 0
- group Id
- OG003
- value
- 88
- units
- Participants
- title
- Week 66
- categories
- measurements
- group Id
- OG003
- lower Limit
- 20.0
- upper Limit
- 150.0
- value
- 49.50
- denoms
- counts
- group Id
- OG000
- value
- 0
- group Id
- OG001
- value
- 0
- group Id
- OG002
- value
- 0
- group Id
- OG003
- value
- 66
- 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
- 239
- units
- Participants
- description
- Comparison of adaptive functioning using the VABS-II composite score in the everolimus low-trough treatment arm (3-7 ng/mL), high-trough treatment arm (9-15 ng/mL) and placebo arm. The Vineland II assesses an individual's development of personal independence \& social responsibility. The questionnaire contains 433 items which assess 15 subdomains organized into the five domains of Communication, Daily Living Skills, Socialization, Motor Skills and Maladaptive Behavior. The overall Adaptive Behavior Composite (ABC) score is obtained by summing the standard scores of the first four domain scores for patients aged less than 7 years, or the first 3 domain scores for patients aged 7 or older (the Maladaptive Behavior domain is optional). The ABC standard score ranges from 20 to 160 with a mean of 100 and a standard deviation of 15. Higher scores correspond to improved adaptive level. Note that 2 questionnaires with ABC scores\<20 (data issues) were included in this analysis.
- 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 Vineland-II Adaptive Behavior Composite (ABC) Score
- type
- SECONDARY
- unit Of Measure
- scores on a scale
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