Looking Beyond Seizure Control
A Clinical Study for Children and Adults with Tuberous Sclerosis Complex (TSC)
who are living with (TAND)
(TSC Associated Neuropsychiatric Disorders)
Epicom is a virtual study meaning participants can complete all study visits at home (using remote technology).
If you or your child are diagnosed with tuberous sclerosis complex (TSC) and experience seizures, you may be eligible to take part in the Epicom study. This study is evaluating the changes in behavioral and neuropsychiatric symptoms when an investigational drug is taken as an add-on therapy for patients with TSC who experience seizures.
To find out if you or your child may be eligible for the Epicom study, please consult your treating doctor to check if you can participate in clinical trials before contacting a study site.
About the Epicom study
The Epicom study will evaluate the changes in behavioral and neuropsychiatric symptoms of TSC when an investigational drug is taken as an add-on therapy for patients with TSC who experience seizures. The study will also assess changes in seizure frequency and how well patients tolerate the investigational drug.
Important points about the Epicom study:
The investigational drug will be taken as add-on therapy to the participant’s other current treatments for TSC
This is an open-label study, which means that all investigators and participants know which treatment is being given to participants.
This study has a virtual option, which means that participants will choose whether they want to complete all study visits either from their home (using telemedicine) or at the study site
What is the investigational drug?
The investigational drug used in this study is not approved to treat co-occurring conditions associated with TSC, such as cognitive impairment and specific behavioral disorders, and is therefore called “investigational.”
What is a decentralized (virtual) study?
Decentralized clinical trials use virtual technology (such as telemedicine) to interact with study participants and collect data. The use of remote visits enables a greater level of convenience for participants by reducing or eliminating the need to travel to specific study sites. Since patient assessments are not restricted to visits to the study site, more frequent data collection is possible as well.
Details of the study
The Epicom study is a global study being conducted in the United States, United Kingdom, Canada, and Poland. The study will enroll up to 75 children and adults who experience seizures associated with TSC.
Using an electronic diary (eDiary), participants or caregivers will complete a variety of questionnaires that assess behaviors, mood, seizures, sleep, and other measures at various timepoints throughout the study. Daily health checks regarding any changes in the participant’s health and medications will also be recorded in the eDiary.
Caregivers will complete eDiary assessments; however, there are a few optional questionnaires that adult participants may complete. Appropriate training and instructions on how to enter data will be provided.
Participation in this study will last up to 62 weeks and includes 4 periods:
Participants will be screened to see if the study is right for them and to decide if they want to take part. Participants and/or caregiver(s) will be trained on how to record information in the eDiary.
Participants will complete additional screening and baseline assessments.
Participants will take the investigational drug twice a day. The dose will be adjusted until the optimal dose is achieved.
Participants will take the investigational drug at their optimal dose twice a day.
Assessment using an eDiary will be completed throughout this period by the participant and/or caregiver.
Participants will have the option to continue taking the investigational drug at the same dose or stop the investigational drug and continue their other TSC treatments (standard of care).
Assessments using an eDiary will be completed throughout this period by the participant and/or caregiver.
Participants continuing treatment will undergo a final assessment.
During these periods, virtual (telemedicine) or in-person study site visits will be required.
Participants who choose to stop taking the investigational drug at the end of Treatment Period 1 or 2 will have their treatment dose tapered down over 10 days, followed by a 2-week safety follow-up period.
Participants who are eligible and participate in the Epicom study will receive the investigational drug, as well as any study-related visits, tests and assessments, at no cost. Participants or their caregivers will also be reimbursed for some study-related expenses, such as costs associated with travel and hotels.
Is the Epicom study right for me or my child?
You or your child may be eligible to participate in the Epicom study if all eligibility criteria are met, including:
In the US: 1 to 65 years of age (inclusive)
Outside the US: 2 to 65 years of age (inclusive)
Diagnosis of TSC with a history of seizures
Has problematic behaviors that are considered moderate to severe, for example:
– Aggression, impulsivity, temper tantrum, self-injury, hyperactivity, extreme shyness, mood swings, poor eye contact, repetitive behaviors, restlessness, difficulty getting along with peers, rigid/inflexible to procedure and/or change.
Current treatment with 1 or more anti-seizure medications at a stable dose for at least 4 weeks
For women of childbearing potential: not pregnant or breastfeeding and using a highly effective method of contraception
Male participants must use an acceptable form of contraception and must refrain from donating semen
Willing and able to provide informed consent or has a caregiver or legal representative willing and able to do so on their behalf
Parent(s)/ or legal representative is willing to allow the responsible authorities, primary care practitioner and/or consultant to be notified of the participant’s involvement in the study
Participant (including adults lacking capacity) has a dedicated caregiver willing to complete assessments in the study
Has never been treated with cannabidiol (CBD) oral solution or stopped taking it at least 3 months prior to study screening
Willing to undergo a 1-month washout period before being rescreened if using or has in the past used recreational or medicinal cannabis or synthetic cannabinoid-based medications within the 3 months prior to screening
Has NOT started felbamate within the last 12 months prior to screening
Has NOT received an investigational medicinal product within the 3 months prior to Screening
Has NOT previously been assigned study intervention for this study NOR is currently enrolled in any other interventional study (including behavioral intervention studies)
Does NOT have a known NOR suspected history of alcohol or substance abuse
To find out if you or your child may be eligible
You or your child must meet all study criteria to take part in the Epicom study.
Participating in a clinical study can help advance medical science and support the TSC community.
About Tuberous Sclerosis Complex (TSC)
births each year1,2
affected worldwide1,2
affected in the U.S.2
Tuberous sclerosis complex (TSC) is a rare genetic disease that causes non-cancerous (benign) tumors to grow in the brain and several areas of the body, including the spinal cord, eyes, lung, heart, kidneys, and skin. Approximately 85% of patients with TSC experience seizures, with onset usually during the first 2 years of life. An estimated one in 6,000 children are born each year with TSC, with approximately 1 million people affected worldwide.1,2
In addition to seizures, approximately 90% of TSC patients experience behavioral, cognitive, and neuropsychiatric symptoms at some point during their lifetimes.3 TSC-Associated Neuropsychiatric Disorders (TAND) is an umbrella term that includes the range of symptoms of brain dysfunction in TSC, including aggressive behaviors, autism spectrum disorder (ASD), intellectual disabilities, psychiatric disorders, as well as school and occupational difficulties.2,3 These co-occurring symptoms of TSC place a heavy burden on patients, as well as their caregivers and families.
How is TSC treated?
Anti-seizure drugs may be prescribed to control seizures. Other drugs may help manage behavior problems or other symptoms. Intervention services, such as physical, occupational, speech, and behavioral therapy, may be helpful. Early intervention and special needs services can help children with developmental delays and behavior issues adapt to the classroom.2
The Epicom study is evaluating the changes in behavioral and neuropsychiatric symptoms (TAND) when an investigational drug is taken as an add-on therapy for patients with TSC who experience seizures.
References
- Whitney R, Zak M, Haile D, Nabavi Nouri M. The state of pediatric tuberous sclerosis complex epilepsy care: Results from a national survey. Epilepsia Open. 2022;7(4):718-728. doi: 10.1002/epi4.12652
- What is TSC? Tuberous Sclerosis Complex Alliance. https://www.tscalliance.org/about-tsc/what-is-tsc/. Accessed February 21, 2023.
- de Vries PJ, Wilde L, de Vries MC, et al. A clinical update on tuberous sclerosis complex-associated neuropsychiatric disorders (TAND). Am J Med Genet C Semin Med Genet. 2018;178(3):309-320. doi: 10.1002/ajmg.c.31637
To find out if you or your child may be eligible
for the Epicom study, please consult your treating doctor to check if you can participate in clinical trials before contacting a study site.
The Epicom Study Is Taking Place Now
Please consult your treating doctor to check if you can participate in clinical trials before contacting the site closest to you.
There are Epicom study sites located in the United States, United Kingdom, Canada, and Poland. View the study sites by location to determine which is closest to you. For patients in the US, if you do not see a study site close to you and are interested in participating virtually or you are unable to reach one of the below sites, please email clinicaltrialdisclosure@jazzpharma.com.
United States
Canada
United Kingdom
Poland
Colorado
Children’s Hospital Colorado
Aurora, CO
Contact: Ryan Osselborn
Email: ryan.osselborn@childrenscolorado.org
Phone: 720-777-0493
Principal Investigator: Dr. Susan Koh
Connecticut
Yale University School of Medicine
New Haven, CT
Contact: Kelly Benson-Atwood
Email: kelly.benson-atwood@yale.edu
Phone: 203-785-2445
Principal Investigator: Dr. Aline Herlopian
Florida
Nicklaus Children’s Hospital Research Institute
Miami, FL
Contact: Damaris Rodriguez
Email: Damaris.rodriguez@nicklaushealth.org
Phone: 305-903-4416
Principal Investigator: Dr. Paula Schleifer
University of Florida
Gainesville, FL
Contact: Whitney Miller
Email: Whitney.Miller@neurology.ufl.edu
Phone: 352-733-1821
Principal Investigator: Dr. Hans Shuhaiber
Massachusetts
Massachusetts General Hospital
Boston, MA
Contact: Lydia Tsega
Email: ltsega@mgh.harvard.edu
Phone: 617-726-6524
Principal Investigator: Dr. Elizabeth Thiele
Minnesota
Minnesota Epilepsy Group
Roseville, MN
Contact: Sarah Ellis
Email: sellis@mnepilepsy.net
Phone: 651-377-8319
Principal Investigator: Dr. Douglas Smith
North Carolina
Wake Forest University School of Medicine
Winston-Salem, NC
Contact: Timothy Gilliam
Email: tpgillia@wakehealth.edu
Phone: 336-716-7422
Principal Investigator: Dr. Roy Ervin Strowd, III
Ohio
Cincinnati Children’s Hospital Medical Center
Cincinnati, OH
Contact: Adrienne Victory
Email: adrienne.victory@cchmc.org
Phone: 513-636-8016
Principal Investigator: Dr. Darcy Krueger
Pennsylvania
University of Pennsylvania
Philadelphia, PA 19104
Contact: Ilana Cohen
Email: Ilana.cohen@pennmedicine.upenn.edu
Phone: 215-662-7227
Principal Investigator: Dr. Michael Gelfand
Tennessee
LeBonheur Children’s Hospital
Memphis, TN 38103
Contact: Kara Lessel
Email: Kara.lessel@mlh.org
Phone: 901-287-4594
Principal Investigator: Dr. Sarah Weatherspoon
Texas
UT Health
Houston, TX
Contact: Luana Pimentel
Email: luana.h.pimentel@uth.tmc.edu
Phone: 713-500-7147
Principal Investigator: Dr. Sarah Wilson
San Antonio, TX
Contact: Anthony Torres
Email: torresa23@uthscsa.edu
Phone: 210-450-8455
Principal Investigator: Dr. Linda Leary
Virginia
University of Virginia
Charlottesville, VA
Contact: Stacy Thompson
Email: SRC2H@uvahealth.org
Phone: (434) 982-4315
Principal Investigator: Dr. Katheryn Frazier
Vancouver, British Columbia
Calgary, Alberta
Coming Soon!
Toronto, Ontario
The Hospital for Sick Children
Toronto, Ontario
Contact Name: Laura MacDougall
Email: laura.macdougall@sickkids.ca
Phone: 416-813-7996
Principal Investigator: Dr. Lauren Sham
Poland
Coming Soon!
United Kingdom
University Hospitals Bristol NHS Foundation Trust
Bristol, UK
Contact: Stephanie Evans/Clare Harrison/Helen Clark (Study co-ordinators/nurses)
Email: Stephanie.evans@uhbw.nhs.uk / Clare.Harrison@uhbw.nhs.uk / Helen.Clark@uhbw.nhs.uk
Phone: +44 01173 420196
Principal Investigator: Dr Sam Amin
Sheffield Children’s NHS Foundation Trust
Sheffield, UK
Contact: Janet Shackleton (Study Co-ordinator)
Email: janet.shackleton@nhs.net
Phone: 01143 053236 / 01142 717417
Principal Investigator: Dr Archana Desurkar
Frequently Asked Questions
What is a study?
A study (also called a clinical trial) is a process that helps to answer important questions about certain diseases, drugs, or therapies and how they may impact participants. For example, in the case of an investigational drug, a study may tell us how well the drug works for a certain condition including how safe and tolerable it is.
Why are studies important?
A study can help answer important medical questions to assist doctors in making the right decisions for the patients they care for. Such information once published, can also help the general public live happier and healthier lives. By participating in a clinical study, you can help advance medical science and support the TSC community.
What is the purpose of the Epicom study?
The Epicom study will evaluate the changes in behavioral and neuropsychiatric symptoms when an investigational drug is taken as an add-on therapy for children and adults (1 to 65 years of age in the USA, 2 to 65 years of age outside of USA) with TSC who experience seizures. The study will also assess changes in seizure frequency and how well patients tolerate the investigational drug.
What is the investigational drug?
The investigational drug will be taken as add-on therapy to the participant’s other current treatments for TSC. It is not approved to treat co-occurring conditions associated with TSC, such as cognitive impairment and specific behavioral disorders, and is therefore called “investigational.”
What is a decentralized (virtual) study?
Decentralized clinical trials use virtual technology (such as telemedicine) to interact with study participants and collect data. The use of remote visits enables a greater level of convenience for participants by reducing or eliminating the need to travel to specific study sites. Since patient assessments are not restricted to visits to the research site, more frequent data collection is possible as well.
What will the Epicom study involve?
The duration of participation in the study will be up to 62 weeks, which includes a 2-3-week Screening Period, 4-week Baseline Period, and two 26-week Treatment Periods.
During the 26-week Treatment Periods, participants will take their investigational drug twice a day at home as instructed by the study doctor (the first dose will be taken by the patient under medical supervision). The investigational drug will be taken in addition to the participant’s other current treatments. During Treatment Period 2, participants will have the option to continue taking the investigational drug or stop the investigational drug and continue taking their other current treatments (standard of care).
This is an open-label study, which means that all investigators and participants know which treatment is being given.
Using an electronic diary (eDiary), participants and/or caregivers will complete a variety of questionnaires that assess behaviors, mood, seizures, sleep, and other measures at different timepoints throughout the study. Daily health checks regarding any changes in the participant’s health and medications will also be recorded in the eDiary.
Will compensation for time and travel be provided?
Participants or caregivers in the study may receive reimbursement for travel and expenses. Please discuss this with the study team.
Is there a cost to participate?
There is no cost to participate in the Epicom study. If you or your child are eligible and decide to take part:
- You or your child will receive study-related care from a team of experienced doctors and nurses throughout the study
- All study-related visits, tests, assessments, and investigational drug will be provided at no cost to you
What else do I need to consider?
The research team will be able to explain more about what the Epicom study will involve, and it is up to you to decide if you want to take part. Participation in this study is voluntary. Your decision to participate or not participate will have no effect on the medical care you or your child receives now or in the future. If you or your child is eligible and you choose to participate in the Epicom study, you or your child may leave the study at any time without providing any reason.
Where are the study sites located?
The global Epicom study is being conducted across several study sites in the United States, United Kingdom, Canada, and Poland. Participants will have the option to complete all study visits either virtually (using telemedicine) or at the study site.
Find a study site location near you.
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