Trial · NCT01289912
Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)
NCT01289912, Phase 2, Completed, Mustafa Sahin; results first posted 2018-01-25
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC). Trials, record NCT01289912. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01289912
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)
- NCT number
- NCT01289912
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 52 participants, actual
- Lead sponsor
- Mustafa Sahin
- Conditions
- Tuberous Sclerosis Complex
- Interventions
- RAD001, Placebo
- First posted
- 2011-02-04
- Last updated
- 2018-01-25
- Results first posted
- 2018-01-25
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Evaluation of the Safety of RAD001 on Neurocognition in Patients With TSC Compared With Placebo in Patients With TSC. | 6 months | Evaluation of the safety of RAD001 compared with placebo in patients with TSC focusing on NCI CTCAE Grade 3 and 4 adverse events, serious adverse events, and Grade 3 and 4 laboratory toxicities. |
| Primary | Evaluation of the Efficacy of RAD001 on Neurocognition in Patients With TSC Compared With Placebo. | 6 months | Baseline and 6 month Timepoint scores are reported for the following primary outcome measures: 1. Peabody Picture Vocabulary Test 4 (PPVT-4; Receptive Language Measure). Scores reported as (mean, SD). Range=40-160, higher scores are better. 2. Expressive Vocabulary Test 2 (EVT-2; Expressive Language Measure). Scores reported as (mean, SD). Range=40-160, higher scores are better. 3. Wide Range Assessment of Memory and Learning 2 (WRAML2; Measure of Verbal Memory and Attention ). Scores reported as (mean, SD). Range=1-19, higher scores are better. 4. Vineland Adaptive Behavior Scales-II (VABS-II; Measure of Adaptive Behavior). Scores reported as (mean, SD). Range = 40-160, higher scores are better. 5. Purdue Pegboard Test (Measure of Fine Motor Speed and Coordination). Scores reported as (mean, SD). Range = 40-160, higher scores are better. |
| Primary | Evaluation of the Efficacy of RAD001 on Neurocognition (Cambridge Neuropsychological Test Automated Battery) in Patients With TSC Compared With Placebo. | 6 months | Scores are reported for baseline and 6 month timepoints on the Cambridge Neuropsychological Test Automated Battery (CANTAB) subscales below. For all subscales, scores are reported as the mean difference between the study subjects and a normative population matched for age, gender and IQ (e.g., subject subscale score - mean of matched normative group = reported score). Higher scores represent a better outcome. 1. Spatial Span (SSP) (spatial memory span) Range: -3 to 3 2. Spatial Working Memory (working memory) Range: -3 to 3 3. Pattern Recognition Memory (PRM) (visual pattern recognition memory) Range: -3 to 3 4. Spatial Recognition Memory (SRM) (spatial recognition memory) Range: -4 to 4 5. Rapid Visual Information Processing (RVIP) (sustained attention) Range: -4 to 4 6. Stockings of Cambridge (SOC) (spatial planning) Range: -4 to 4 7. Intra-Extra Dimensional Set Shift (IDED) (cognitive flexibility) Range: -5 to 5 8. Reaction Time (processing speed) Range: -5 to 5 |
| Secondary | Comparison of Absolute Change From Baseline in Frequency of Epileptiform Events Between Patients Taking RAD001 vs. Placebo | 6 months | Comparison of absolute change from baseline in frequency of epileptiform events as recorded on seizure diaries between patients taking RAD001 vs. placebo |
| Secondary | Comparison of Sleep Disturbances Between Patients Taking RAD001 vs. Placebo | 6 months | Comparison of sleep disturbances between patients taking RAD001 vs. placebo, measured by the Pediatric Sleep Questionnaire (PSQ) and sleep logs |
| Secondary | Comparison of Autism Spectrum Disorders Features Between Patients Taking RAD001 vs. Placebo | 6 months | Scores for the Baseline and 6 Month Timepoints are reported. The secondary outcome measure was the Social Responsiveness Scale (SRS). Standard scores are reported with a mean of 100 and standard deviation of 15. The range is 40-160 with higher scores indicating a better outcome. |
| Secondary | Comparison of Academic Skills Between Patients Taking RAD001 vs. Placebo | 6 months | Scores are reported for Baseline and 6 Month Timepoints. The secondary outcome measure was the Wide Range Achievement Test 4 (WRAT4), which was used to assess academic skills. The Reading and Math subtests were used. Standard scores are reported which have a mean of 100 and a standard deviation of 15 (range=40-160 where higher is better). |
| Secondary | Comparison of Behavioral Problems Between Patients Taking RAD001 vs Placebo | 6 months | Scores for Baseline and 6 Month Timepoints are reported for the following secondary outcome measures: 1. Behavior Rating Inventory of Executive Function (BRIEF) (Measure of executive functions) T-scores are reported, with a mean of 50 and a standard deviation of 10. The range is 30-100 with higher scores indicating a worse outcome. 2. Behavioral Assessment System for Children (BASC) (Measure of emotional and behavioral problems) T-scores are reported (mean of 50, SD of 10). The range is 30-100 with higher scores indicating a worse outcome. Conversely, on the Adaptive Skills subscale of the BASC, lower scores indicate a poorer outcome. 3. Strengths and Difficulties Questionnaire (SDQ). Includes questions related to emotional symptoms, conduct problems, inattention/hyperactivity, peer relationship problems and prosocial behavior. Responses to these items are summed to comprise a Total Difficulties Score, which ranges from 0-40, with lower scores indicating a better outcome |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Evaluation of the Safety of RAD001 on Neurocognition in Patients With TSC Compared With Placebo in Patients With TSC. | 6 months | Adverse Events | NUMBER | |
| PRIMARY | Evaluation of the Efficacy of RAD001 on Neurocognition in Patients With TSC Compared With Placebo. | 6 months | units on a scale | MEAN | |
| PRIMARY | Evaluation of the Efficacy of RAD001 on Neurocognition (Cambridge Neuropsychological Test Automated Battery) in Patients With TSC Compared With Placebo. | 6 months | units on a scale | MEAN | |
| SECONDARY | Comparison of Absolute Change From Baseline in Frequency of Epileptiform Events Between Patients Taking RAD001 vs. Placebo | 6 months | Data was not collected reliably and therefore was not analyzed. | ||
| SECONDARY | Comparison of Sleep Disturbances Between Patients Taking RAD001 vs. Placebo | 6 months | Data was not collected reliably and therefore was not analyzed. | ||
| SECONDARY | Comparison of Autism Spectrum Disorders Features Between Patients Taking RAD001 vs. Placebo | 6 months | units on a scale (SRS) | MEAN | |
| SECONDARY | Comparison of Academic Skills Between Patients Taking RAD001 vs. Placebo | 6 months | units on a scale | MEAN | |
| SECONDARY | Comparison of Behavioral Problems Between Patients Taking RAD001 vs Placebo | 6 months | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group142
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Evaluation of the Safety of RAD001 on Neurocognition in Patients With TSC Compared With Placebo in Patients With TSC. | RAD001 | 325 | Adverse Events | ||
| Evaluation of the Safety of RAD001 on Neurocognition in Patients With TSC Compared With Placebo in Patients With TSC. | Placebo | 147 | Adverse Events | ||
| Evaluation of the Efficacy of RAD001 on Neurocognition in Patients With TSC Compared With Placebo. | Placebo - Baseline | PPVT4 (Receptive Language) | 86.00 | Standard Deviation 17.78 | units on a scale |
| Evaluation of the Efficacy of RAD001 on Neurocognition in Patients With TSC Compared With Placebo. | RAD001 - Baseline | PPVT4 (Receptive Language) | 81.06 | Standard Deviation 23.96 | units on a scale |
| Evaluation of the Efficacy of RAD001 on Neurocognition in Patients With TSC Compared With Placebo. | Placebo - 6 Months | PPVT4 (Receptive Language) | 88.54 | Standard Deviation 17.21 | units on a scale |
| Evaluation of the Efficacy of RAD001 on Neurocognition in Patients With TSC Compared With Placebo. | RAD001 - 6 Months | PPVT4 (Receptive Language) | 82.5 | Standard Deviation 22.32 | units on a scale |
| Evaluation of the Efficacy of RAD001 on Neurocognition in Patients With TSC Compared With Placebo. | Placebo - Baseline | EVT2 (Expressive Language) | 85.47 | Standard Deviation 12.22 | units on a scale |
| Evaluation of the Efficacy of RAD001 on Neurocognition in Patients With TSC Compared With Placebo. | RAD001 - Baseline | EVT2 (Expressive Language) | 90.00 | Standard Deviation 21.15 | units on a scale |
| Evaluation of the Efficacy of RAD001 on Neurocognition in Patients With TSC Compared With Placebo. | Placebo - 6 Months | EVT2 (Expressive Language) | 89.15 | Standard Deviation 16.08 | units on a scale |
| Evaluation of the Efficacy of RAD001 on Neurocognition in Patients With TSC Compared With Placebo. | RAD001 - 6 Months | EVT2 (Expressive Language) | 82.55 | Standard Deviation 21.22 | units on a scale |
1 to 10 of 142Source ClinicalTrials.gov
See all 142 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | RAD001 | 32 | |
| Overall Study | STARTED | Placebo | 15 | |
| Overall Study | COMPLETED | RAD001 | 29 | |
| Overall Study | COMPLETED | Placebo | 13 | |
| Overall Study | NOT COMPLETED | RAD001 | 3 | |
| Overall Study | NOT COMPLETED | Placebo | 2 |
Source ClinicalTrials.gov
Adverse events126
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Conjunctivitis | Eye disorders | RAD001 | 2 | 32 | 2 |
| other | Conjunctivitis | Eye disorders | Placebo | 0 | 15 | 0 |
| other | Constipation | Gastrointestinal disorders | RAD001 | 2 | 32 | 2 |
| other | Constipation | Gastrointestinal disorders | Placebo | 0 | 15 | 0 |
| other | Diarrhea | Gastrointestinal disorders | RAD001 | 11 | 32 | 18 |
| other | Diarrhea | Gastrointestinal disorders | Placebo | 5 | 15 | 8 |
| other | Nausea | Gastrointestinal disorders | RAD001 | 4 | 32 | 5 |
| other | Nausea | Gastrointestinal disorders | Placebo | 1 | 15 | 2 |
| other | Stomach ache/pain/upset/irritation | Gastrointestinal disorders | RAD001 | 8 | 32 | 11 |
| other | Stomach ache/pain/upset/irritation | Gastrointestinal disorders | Placebo | 6 | 15 | 8 |
1 to 10 of 126Source ClinicalTrials.gov
See all 126 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | RAD001 | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | RAD001 | 30 | 32 |
| Total, other adverse events | Placebo | 10 | 15 |
| Total, serious adverse events | RAD001 | 3 | 32 |
| Total, serious adverse events | Placebo | 0 | 15 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| RAD001 vs Placebo: serious adverse events |
Source Trial comparisons
Tests6
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as | Source |
|---|---|---|
| Intra/Extra Dimensional Set Shift | Primary outcome | ConductCognition |
| Pattern Recognition Memory | Primary outcome | ConductCognition |
| Rapid Visual Information Processing | Primary outcome | ConductCognition |
| Spatial Recognition Memory | Primary outcome | ConductCognition |
| Tower Planning | Primary outcome | ConductCognition |
| Vocabulary Test | Primary outcome | ConductCognition |
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01289912
GET https://conductscience.org/api/trials/v1/NCT01289912/results?page=2