Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
|Eligible Ages||2 Years - 40 Years|
Inclusion Criteria 1. Patients: 1 year to 40 years. 2. Diagnosis: treatment resistant epilepsy due to Tuberous Sclerosis Complex or Focal Cortical Dysplasia Inclusion Criteria (Concurrent Comparison Group) 1. Patients: 1 year to 40 years. Matched for age (+/- 7 years) and sex of subjects in the treatment group. 2. Diagnosis: treatment resistant due to TSC or FCD. Matched for diagnosis of TSC and FCD. 3. Brain surgery for seizure control in which tissue is banked for research utilizing an existing IRB-approved study. Exclusion Criteria 1. Treatment with an mTOR inhibitor (everolimus, sirolimus) during the past four weeks. 2. Known hypersensitivity to an mTOR inhibitor (everolimus, sirolimus) 3. Failure to establish diagnosis of treatment resistant epilepsy (i.e., adequate trials of two appropriately-chosen, tolerated and adequate trials of antiepileptic drugs) . 4. Exposure to any investigational agent in the month prior to study entry. 5. History of malignancy patients who are receiving anti-cancer treatments, such as radiation therapy and/or chemotherapy. 6. Patients with severe and/or uncontrolled medical conditions, 7. Patients on chronic corticosteroid therapy 8. A history of HIV seropositivity 9. Patients who have received live attenuated vaccines within 1 week of start of everolimus and during the study; 10. Known impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral everolimus; 11. Uncontrolled diabetes mellitus 12. Patients who have any severe and/or uncontrolled medical conditions 13. Known impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral everolimus;
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
|NYU Langone Health|
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Orrin Devinsky, MD|
|Principal Investigator Affiliation||NYU Langone Health|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
The disease, disorder, syndrome, illness, or injury that is being studied.
|Epilepsy, Tuberous Sclerosis Complex, Focal Cortical Dysplasia|
This is a single center open-label pilot clinical trial of patients with TRE, ages 1 to 40 years old, with TSC or FCD who are scheduled for epilepsy surgery. Patients will be treated with everolimus for 7 to 28 days prior to epilepsy surgery with extension of time from 7 to 28 days in successive cohorts of patients. The initial cohort of at least three patients will be treated for 7 days and after the safety of therapy is assured for this group, there will be an extension of the treatment to 14 days for at least three patients. This will be extended at one week intervals/three patient groups to a maximum treatment duration of 28 days. Resected brain tissue will be analyzed for activation of mTORC1 and mTORC2 signaling pathways, glutamatergic and GABA-ergic neurotransmission using histochemistry, genetic analysis, as well as extracellular field recordings in acute ex-vivo brain slices from surgery. A blood sample, collected at the time of surgery, will be analyzed for everolimus levels and VEGF-D. All patients will undergo standardized intra-operative ECoG recordings over the primary epileptogenic region and reviewed blindly. Subjects will be in the study for 7-28 days. The investigators will study variables listed in specific aims 1 and 2 in TSC and FCD patients treated with 7 to 28 days of everolimus and compare these to untreated control patients with TRE and TSC or FCD. A concurrent comparison group of 12 subjects will also be enrolled. They will all be undergoing routine surgery for the diagnosis of TRE with TSC or FCD. All study procedures will be performed at the Comprehensive Epilepsy Center (CEC) with the exception of the surgery, which will be performed at Tisch Hospital.
Experimental: Treated Subjects
This group will be treated with everolimus 7-28 days prior to surgery
No Intervention: Reference Subjects
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.
Drug: - 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
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.