Trial · NCT01929642
Rapalogues for Autism Phenotype in TSC: A Feasibility Study
NCT01929642, Phase 2, Completed, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; results first posted 2021-03-29
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Rapalogues for Autism Phenotype in TSC: A Feasibility Study. Trials, record NCT01929642. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01929642
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Rapalogues for Autism Phenotype in TSC: A Feasibility Study
- NCT number
- NCT01929642
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 3 participants, actual
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Conditions
- Tuberous Sclerosis Complex, Self-injury, Autism
- Interventions
- Sirolimus, Everolimus
- First posted
- 2013-08-28
- Last updated
- 2021-03-29
- Results first posted
- 2021-03-29
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Compliance to the Treatment Protocol. | Change from baseline to EOT visit 12 week 53 | One outcome measurement of feasibility will include family/patient compliance with the treatment protocol, which will be assessed and documented at every study visit and telephone follow-up call, by the physician and/or study team member. This was calculated by calculating dividing the total number of study visits and study assessments completed by the total number of study visits and study assessments indicated by the treatment protocol. |
| Primary | Caregiver Burden | Change from baseline to EOT visit 12 week 53 | The Caregiver Burden Scale is a standard set of questions which will be used to measure the non-medical impact of TSC on caregivers and how it affects the feasibility of study completion. The Caregiver's Burden Scale (CBS) is a 22-item scale that assess subjectively experienced burden by caregiver's to chronically disabled persons. maximum scores: 88 \& Minumum scores: 22 High values represent a worse outcome |
| Primary | Feasibility Measurements of Parental Stress | Change from baseline to EOT visit 12 week 53 | Measurements of stress will be administered. Specifically, we will use the Parental Stress Index. Quantifying stress, as well as compliance with the study protocol, will allow investigators to objectively assess the feasibility of a larger clinical trial of sirolimus in patients with TSC. Parental stress index maximum score: 180 Parental stress index minimum score: 36 higher raw scores indicate higher levels of stress. |
| Secondary | Total Number of Aggressions or Self-injuries | 1 year | This is the total number of aggressions or self-injuries for all participants. |
| Secondary | Cognitive Function as Assessed by the Capute Scale | 1 year | Score range maximum: 100 Score range minimum: 0 High values represent a high cognitive function Below 70 is abnormal. 70-100 is the normal range. |
| Secondary | Repetitive Behavior | 1 year | Repetitive behavior will be assessed using the Repetitive Behavior Scale - revised, a questionnaire to characterize several domains of repetitive behavior including ritualistic behavior, stereotypic behavior, self-injurious behavior, compulsive behavior, and restricted interests. There are 36 items on the scale. Behaviors are rated on a 4-point scale: 0-Behavior does not occur, 1-Behavior occurs and is a mild problem, 2-Behavior occurs and is a moderate problem, 3-Behavior occurs and is a severe problem. Maximum score: 108 \& minimum score: 0 A high score represents the worse outcome |
| Secondary | Self-Injury Trauma Scale--SIT Scale | 1 year | The SIT Scale is a 3-part clinician-completed scale used to quantify visible injuries caused by self-injurious behavior(SIB). Part 1 includes sections to indicate SIB topographies and any evidence of healed injury. In Part 2 evaluators document the location and severity of injury (on a 3-point scale). In Part 3, respective scores from Parts 1 and 2 are summed to obtain a Number Index, a Severity Index, and Estimate of Current Risk. This Scale has been used in research with adults with SIB with inter-rater reliability averaging 85%. Maximum score: 100 Minimum score: 0 High score represent worse outcome. |
| Secondary | Frequency of Seizures Assessed by Total Number of Seizures | at baseline | Parents will be asked to document the frequency of their child's seizures using a manual or electronic (seizuretracker.com) seizure diary. The total number of seizures at baseline for all participants. |
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 | Number of Participants With Compliance to the Treatment Protocol. | Change from baseline to EOT visit 12 week 53 | percentage of completed visits/measures | NUMBER | |
| PRIMARY | Caregiver Burden | Change from baseline to EOT visit 12 week 53 | units on a scale | MEAN | |
| PRIMARY | Feasibility Measurements of Parental Stress | Change from baseline to EOT visit 12 week 53 | units on a scale | MEAN | |
| SECONDARY | Total Number of Aggressions or Self-injuries | 1 year | Number of aggressions or self-injuries | NUMBER | |
| SECONDARY | Cognitive Function as Assessed by the Capute Scale | 1 year | score on a scale | MEAN | |
| SECONDARY | Repetitive Behavior | 1 year | score on a scale | MEAN | |
| SECONDARY | Self-Injury Trauma Scale--SIT Scale | 1 year | Self-injury was not severe enough to use the scale. Therefore analysis was not done. | ||
| SECONDARY | Frequency of Seizures Assessed by Total Number of Seizures | at baseline | seizures | NUMBER |
Source ClinicalTrials.gov
Results by group9
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Compliance to the Treatment Protocol. | Everolimus or Sirolimus | Participant 002 | 100 | percentage of completed visits/measures | |
| Number of Participants With Compliance to the Treatment Protocol. | Everolimus or Sirolimus | Participant 003 | 58 | percentage of completed visits/measures | |
| Number of Participants With Compliance to the Treatment Protocol. | Everolimus or Sirolimus | Participant 001 | 58 | percentage of completed visits/measures | |
| Caregiver Burden | Everolimus or Sirolimus | 61 | Full Range 45 to 70 | units on a scale | |
| Feasibility Measurements of Parental Stress | Everolimus or Sirolimus | 111 | Full Range 86 to 132 | units on a scale | |
| Total Number of Aggressions or Self-injuries | Everolimus or Sirolimus | 82 | Number of aggressions or self-injuries | ||
| Cognitive Function as Assessed by the Capute Scale | Everolimus or Sirolimus | 15 | Full Range 14 to 21 | score on a scale | |
| Repetitive Behavior | Everolimus or Sirolimus | 69 | Full Range 58 to 80 | score on a scale | |
| Frequency of Seizures Assessed by Total Number of Seizures | Everolimus or Sirolimus | 0 | seizures |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sirolimus or Everolimus | 3 | |
| Overall Study | COMPLETED | Sirolimus or Everolimus | 1 | |
| Overall Study | NOT COMPLETED | Sirolimus or Everolimus | 2 |
Source ClinicalTrials.gov
Adverse events1
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 | aphthous ulcer | Skin and subcutaneous tissue disorders | Everolimus | 1 | 3 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Everolimus | ||
| Total, other adverse events | Everolimus | 1 | 3 |
| Total, serious adverse events | Everolimus | 0 | 3 |
Source ClinicalTrials.gov
Papers38
The papers the trial lists on ClinicalTrials.gov.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
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/NCT01929642
GET https://conductscience.org/api/trials/v1/NCT01929642/papers?page=2