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A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD

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ClinicalTrials.gov Identifier: NCT02451696
Recruitment Status : Completed
First Posted : May 22, 2015
Results First Posted : September 1, 2021
Last Update Posted : September 1, 2021
Sponsor:
Information provided by (Responsible Party):
NYU Langone Health

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Epilepsy
Tuberous Sclerosis Complex
Focal Cortical Dysplasia
Intervention Drug: Everolimus
Enrollment 15
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Treated Subjects Reference Subjects
Hide Arm/Group Description This group will be treated with everolimus 7-28 days prior to surgery Everolimus: This study will measure if the drug called Everolimus effects mTOR signaling (an electrical activity signal in the brain) in patients with Tuberous Sclerosis Complex (TSC) and Focal Cortical Dysplasia (FCD) with treatment resistant epilepsy (TRE) who will be undergoing brain surgery. One group of patients will be treated with Everolimus, for 7-28 days prior to epilepsy surgery and another will not. We will determine if there is a difference in mTOR signaling between the patients who were treated with Everolimus and those who were not This group will be enrolled as reference subjects, and will be undergoing routine surgery as part of standard of care treatment. No intervention will be provided to these subjects as part of the study
Period Title: Overall Study
Started 5 10
Completed 4 10
Not Completed 1 0
Reason Not Completed
Surgery postponed             1             0
Arm/Group Title Treated Subjects Reference Subjects Total
Hide Arm/Group Description

This group will be treated with everolimus 7-28 days prior to surgery

Everolimus: This study will measure if the drug called Everolimus effects mTOR signaling (an electrical activity signal in the brain) in patients with Tuberous Sclerosis Complex (TSC) and Focal Cortical Dysplasia (FCD) with treatment resistant epilepsy (TRE) who will be undergoing brain surgery. One group of patients will be treated with Everolimus, for 7-28 days prior to epilepsy surgery and another will not. We will determine if there is a difference in mTOR signaling between the patients who were treated with Everolimus and those who were not

This group will be enrolled as reference subjects, and will be undergoing routine surgery as part of standard of care treatment. No intervention will be provided to these subjects as part of the study. Total of all reporting groups
Overall Number of Baseline Participants 4 10 14
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants 10 participants 14 participants
18.25  (10.1) 13.1  (12.3) 14.6  (11.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 10 participants 14 participants
Female
3
  75.0%
6
  60.0%
9
  64.3%
Male
1
  25.0%
4
  40.0%
5
  35.7%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 10 participants 14 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
0
   0.0%
0
   0.0%
0
   0.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
4
 100.0%
10
 100.0%
14
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 4 participants 10 participants 14 participants
4 10 14
1.Primary Outcome
Title Number of Patients With Adverse Events
Hide Description .Adverse event monitoring should be continued for at least 30 days (or 5 half-lives, whichever is longer) following the last dose of study treatment
Time Frame 6 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treated Subjects Reference Subjects
Hide Arm/Group Description:

This group will be treated with everolimus 7-28 days prior to surgery

Everolimus: This study will measure if the drug called Everolimus effects mTOR signaling (an electrical activity signal in the brain) in patients with Tuberous Sclerosis Complex (TSC) and Focal Cortical Dysplasia (FCD) with treatment resistant epilepsy (TRE) who will be undergoing brain surgery. One group of patients will be treated with Everolimus, for 7-28 days prior to epilepsy surgery and another will not. We will determine if there is a difference in mTOR signaling between the patients who were treated with Everolimus and those who were not

This group will be enrolled as reference subjects, and will be undergoing routine surgery as part of standard of care treatment. No intervention will be provided to these subjects as part of the study.
Overall Number of Participants Analyzed 4 10
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
2.Secondary Outcome
Title Blood Everolimus Levels
Hide Description mTOR signaling in blood
Time Frame 28 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treated Subjects Reference Subjects
Hide Arm/Group Description:

This group will be treated with everolimus 7-28 days prior to surgery

Everolimus: This study will measure if the drug called Everolimus effects mTOR signaling (an electrical activity signal in the brain) in patients with Tuberous Sclerosis Complex (TSC) and Focal Cortical Dysplasia (FCD) with treatment resistant epilepsy (TRE) who will be undergoing brain surgery. One group of patients will be treated with Everolimus, for 7-28 days prior to epilepsy surgery and another will not. We will determine if there is a difference in mTOR signaling between the patients who were treated with Everolimus and those who were not

This group will be enrolled as reference subjects, and will be undergoing routine surgery as part of standard of care treatment. No intervention will be provided to these subjects as part of the study.
Overall Number of Participants Analyzed 4 10
Mean (Standard Deviation)
Unit of Measure: ng/ml
12.35  (5.36) 2  (0)
3.Secondary Outcome
Title Blood Total VEGF Levels (Not Only VEGF-D)
Hide Description [Not Specified]
Time Frame 28 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treated Subjects Reference Subjects
Hide Arm/Group Description:
This group will be treated with everolimus 7-28 days prior to surgery Everolimus: This study will measure if the drug called Everolimus effects mTOR signaling (an electrical activity signal in the brain) in patients with Tuberous Sclerosis Complex (TSC) and Focal Cortical Dysplasia (FCD) with treatment resistant epilepsy (TRE) who will be undergoing brain surgery. One group of patients will be treated with Everolimus, for 7-28 days prior to epilepsy surgery and another will not. We will determine if there is a difference in mTOR signaling between the patients who were treated with Everolimus and those who were not
This group will be enrolled as reference subjects, and will be undergoing routine surgery as part of standard of care treatment. No intervention will be provided to these subjects as part of the study
Overall Number of Participants Analyzed 4 10
Mean (Standard Deviation)
Unit of Measure: pg/ml
56.5  (46.9) 50.85  (54.1)
4.Secondary Outcome
Title mTOR Brain Tissue-S6 Phosphate by Western Blot
Hide Description [Not Specified]
Time Frame 28 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treated Subjects Reference Subjects
Hide Arm/Group Description:
This group will be treated with everolimus 7-28 days prior to surgery Everolimus: This study will measure if the drug called Everolimus effects mTOR signaling (an electrical activity signal in the brain) in patients with Tuberous Sclerosis Complex (TSC) and Focal Cortical Dysplasia (FCD) with treatment resistant epilepsy (TRE) who will be undergoing brain surgery. One group of patients will be treated with Everolimus, for 7-28 days prior to epilepsy surgery and another will not. We will determine if there is a difference in mTOR signaling between the patients who were treated with Everolimus and those who were not
This group will be enrolled as reference subjects, and will be undergoing routine surgery as part of standard of care treatment. No intervention will be provided to these subjects as part of the study
Overall Number of Participants Analyzed 4 10
Mean (Standard Deviation)
Unit of Measure: normalized val-protein relative to actin
0.49  (0.54) 0.81  (0.22)
5.Secondary Outcome
Title HMGB1 Expression in Brain Tissue
Hide Description HMGB1 expression is measured through label-free quantification (LFQ). LFQ is a method in mass spectroscopy that determines the relative amount of proteins in biological samples. The unit of measure is LFQ intensity; a higher LFQ intensity indicates greater HMGB1 expression.
Time Frame 28 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treated Subjects Reference Subjects
Hide Arm/Group Description:
This group will be treated with everolimus 7-28 days prior to surgery Everolimus: This study will measure if the drug called Everolimus effects mTOR signaling (an electrical activity signal in the brain) in patients with Tuberous Sclerosis Complex (TSC) and Focal Cortical Dysplasia (FCD) with treatment resistant epilepsy (TRE) who will be undergoing brain surgery. One group of patients will be treated with Everolimus, for 7-28 days prior to epilepsy surgery and another will not. We will determine if there is a difference in mTOR signaling between the patients who were treated with Everolimus and those who were not
This group will be enrolled as reference subjects, and will be undergoing routine surgery as part of standard of care treatment. No intervention will be provided to these subjects as part of the study
Overall Number of Participants Analyzed 4 10
Mean (Standard Deviation)
Unit of Measure: LFQ intensity
14.85  (0.6) 15.06  (0.35)
Time Frame 30 days
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Treated Subject Reference Subject
Hide Arm/Group Description

This group will be treated with everolimus 7-28 days prior to surgery

Everolimus: This study will measure if the drug called Everolimus effects mTOR signaling (an electrical activity signal in the brain) in patients with Tuberous Sclerosis Complex (TSC) and Focal Cortical Dysplasia (FCD) with treatment resistant epilepsy (TRE) who will be undergoing brain surgery. One group of patients will be treated with Everolimus, for 7-28 days prior to epilepsy surgery and another will not. We will determine if there is a difference in mTOR signaling between the patients who were treated with Everolimus and those who were not

This group will be enrolled as reference subjects, and will be undergoing routine surgery as part of standard of care treatment. No intervention will be provided to these subjects as part of the study.
All-Cause Mortality
Treated Subject Reference Subject
Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/10 (0.00%) 
Hide Serious Adverse Events
Treated Subject Reference Subject
Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/10 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Treated Subject Reference Subject
Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/10 (0.00%) 
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Orrin Devinsky
Organization: NYU Langone
Phone: 646-558-0800
EMail: od4@nyu.edu
Layout table for additonal information
Responsible Party: NYU Langone Health
ClinicalTrials.gov Identifier: NCT02451696    
Other Study ID Numbers: 14-00245
First Submitted: October 8, 2014
First Posted: May 22, 2015
Results First Submitted: November 17, 2020
Results First Posted: September 1, 2021
Last Update Posted: September 1, 2021