In 2022, the TSC Alliance hosted 5 webinars focused on research and featuring known experts in the TSC community as part of our Research Education Webinar Series. These free 1-hour presentations included an open question-and-answer period and offer valuable information on TSC research, clinical trials, and more.
November 9, 2022 – The TrustTSC Phase 3 Clinical Trial for Seizures in Children and Adults with TSC
Presenter: Mary Kay Koenig, MD, Co-Director, UT TSC Center of Excellence, University of Texas McGovern Medical School
The TrustTSC (NCT05323734) trial is a global Phase 3 randomized, double-blind, placebo-controlled study of ganaxolone treatment in children and adults with tuberous sclerosis complex (TSC). The study is exploring whether treatment with investigational ganaxolone can reduce the frequency of seizures associated with TSC in children and adults.
Dr. Koenig provides a general overview of clinical trials and why it is important to participate, discuss the results from the phase 2 clinical trial and what changed between phase 2 and 3, and share how people can learn more about the trial and talk to their doctors about participating.
September 14, 2022 – What is Facial Angiofibroma Associated with TSC and How Can it Be Managed?
Presenter: Joyce M. C. Teng, MD, PhD, Professor of Dermatology & Pediatrics, Stanford University
Moderator: Eric Beresford, PharmD, BCMAS, Vice President and Head of Medical Affairs, Nobelpharma America
Facial angiofibroma is one of the most visible signs of tuberous sclerosis complex (TSC) and occurs in approximately 75%-80% of TSC patients. If left untreated, facial angiofibroma can cause disfigurement. Patients have reported that facial angiofibroma can have negative effects on appearance and self-image, and the tumors may be a psychological and social burden for people living with this condition.
In this webinar session, Dr. Joyce Teng, Professor of Dermatology and Pediatrics, Stanford University and Eric Beresford, Vice President and Head of Medical Affairs, Nobelpharma America, guide attendees in exploring:
- What Is Facial Angiofibroma Associated with TSC?
- The Social and Psychological Impact of Facial Angiofibroma
- Taking Care of Your Skin
- What to Know About Treatments
June 23, 2022 – mTOR Inhibitors: Past, Present and Future
Presenter: Darcy Krueger, MD, PhD, TSC Clinic Director at Cincinnati Children’s Hospital Medical Center, Professor of Clinical Pediatrics and Neurology at the University of Cincinnati College of Medicine, Member of the TSC Alliance Board of Directors
Dr. Krueger provides a brief history of mTOR, how mTOR inhibitors are currently being used to treat different manifestations of TSC, the current limitations and side effects of mTOR treatments, and an overview of research to optimize the use of mTOR treatments. This webinar provides a foundational understanding of mTOR to prepare attendees for the research sessions at the 2022 World TSC Conference being held July 28-31 in Dallas, Texas.
May 25, 2022 – Basimglurant: Potential New Mode of Action for Seizures in TSC
Presenters: Mustafa Sahin, MD, PhD, Professor of Neurology at Harvard Medical School and Director the Rosamund Stone Zander Translational Neuroscience Center at Boston Children’s Hospital; Dr. George Garibaldi, Co-Founder and President, Head of Research and Development of Noema Pharma AG and Dr. Renata Lazarova, Vice President Development, Pediatric Programs at Noema Pharma and lead for TSC development program
Drs. Sahin, Garibaldi and Lazarova provide an overview of a clinical trial testing basimglurant, a potential new mode of action to provide seizure control to people with seizures associated with TSC. The study is currently enrolling in the US, Europe, Israel, Asia and Australia. The study is open to children 5 years old and older, adolescents and adults up to 30 years old whose seizures associated with tuberous sclerosis complex are not sufficiently controlled by current treatment. For more information including the list of participating sites, please contact Ashley Pounders, MSN, FNP-C, at firstname.lastname@example.org or 301-562-9890.
April 21, 2022 – STARS Clinical Trial Town Hall
Presenter: Dr. Robert Roebling, Senior Medical Director in Seizure Freedom Mission, UCB, Inc.
Dr. Roebling provides an introduction and overview to the STARS clinical research study. The global Phase 3 trial is designed for people who experience prolonged epileptic seizures, lasting more than 3 minutes. It is testing an inhaler containing an investigational drug to see whether it could help people who experience prolonged epileptic seizures.