Check out the latest updates from the TS Clinic

1. The TSC-STEPS clinical trial, led by Dr. Krueger and which has major funding support from the FDA Office of Orphan Products Development Division, is in its fourth and final year of enrollment and is expected to be completed in late 2026 or early 2027.  TSC-STEPS is a randomized clinical trial evaluating the safety and efficacy of using sirolimus, an mTOR inhibitor, to prevent seizures from starting or becoming resistant to traditional seizure treatment in patients with TSC when started within the first 6 months of life.  
2. The TSC natural history and biomarker development study within the Developmental Synaptopathies Consortium and funded by the NIH through its Rare Diseases Clinical Research Network program (RDCRN-DSC), is led by Dr. Krueger and in its 11th year and just received notice that the NIH will continue funding for another 5 years.  This study follows TSC patients as young as 3 years of age throughout adulthood, to understand how TSC changes over time in individuals, what treatments are being used (treatment landscape) for autism symptoms and intellectual disability, and seeking to identify MRI and EEG-based markers that help predict future risk, progression of symptoms, and/or treatment opportunities.  In the next 5 years, we have added study procedures to gain better understanding of sleep, sensory issues, and mental health concerns that to date we appreciate can be significant issues in patients with TSC but relative to other aspects of the condition, haven’t been rigorously studied.
3. TSC-FETAL is a new study started in 2025 and led by Dr. Ritter that is funded through the Pediatric Epilepsy Research Foundation.  This study is collecting and analyzing aspects of TSC when diagnosed prenatally and maternal factors leading up to birth and following the babies through the first year of life to learn what aspects impact later TSC symptoms and manifestations, with to goal of identifying new opportunities to intervene and change long term outcomes for the better.
4. Regulating Together in TSC is a clinical trial led by Dr. Capal that is adapting a behavior therapy for children with TSC who struggle to manage emotions and are prone to tantrums and outbursts.  Developed at Cincinnati Children’s for children with autism, we are in our third year of the study, with results expected in 2027.  It is funded through the Congressionally Directed Medical Research Program for Tuberous Sclerosis Complex.   
5. In 2025, we also launched the Cincinnati TSC Center for Adult Research Excellence (TSC-CARE).  We felt that there were too many clinical issues that were going unaddressed for our adult patients with TSC that required time and resources to investigate and implement change.  These questions range from how best to integrate specialist medical care not only between the TSC clinic and the patients’ primary care providers, but also among different specialists at different institutions that often may be in separate hospitals and cities and at times, different states.  We are also looking into what are the long term effects, if any, from being on chronic medications frequently used in TSC.  We are seeking to train more adult specialty providers in TSC (there are too few, as most TSC specialists are pediatric trained).  
6. We continue to collaborate on multiple studies being led by fellow investigators at CCHMC that are trying to improve when we can offer and in whom we can achieve better outcomes from epilepsy surgery in our patients with TSC when our medications to treat epilepsy simply aren’t doing enough.  This includes state of the art brain MRI imaging and MEG (magnetoencephalography) technologies for detecting where the seizures are coming from and how to best operate to safely remove them.  We also have a study that has been testing better ways to use EEGs in surgical planning to achieve better outcomes after surgery.

David Franz, MD, Founding Director
Darcy Krueger, MD, PhD, Co-Director
David Ritter, MD, PhD, Co-Director