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In this episode of TSC Now, host Dan Klein dives deep on a new clinical trial in tuberous sclerosis complex (TSC) called TSC-STEPS. TSC-STEPS is a study to learn more about a drug known as Sirolimus and determine if it can prevent seizures and epilepsy in children diagnosed with TSC. The study is currently enrolling infants diagnosed with TSC who are at risk of developing epilepsy. \
Dan interviews Darcy Krueger, MD, PhD, Director of the TSC Center of Excellence at Cincinnati Children’s Hospital and member of the TSC Alliance Board of Directors (0:59). Dr. Krueger provides an overview of the trial, the eligibility requirements, risks and benefits to participation, and what participation entails for families in terms of site visits and tests. He also discusses how the trial builds off findings of earlier intervention trials and may be part of a greater shift in the paradigm of care for those with TSC. Finally, he provides information about other upcoming and ongoing trials looking for older participants, and encourages everyone listening to help raise awareness of these important trials to help recruit participants and move research forward.
Resources and Links
- Learn more about the TSC-STEPS trial: https://tscsteps.org/
- Learn more about the MILED trial: https://www.thelamfoundation.org/MILED
- Learn more about the Developmental Synaptopathies Consortium: https://www1.rarediseasesnetwork.org/cms/dsc
- Learn more about the Bcureful Travel Fund at the TSC Alliance: https://www.tscalliance.org/individuals-families/bcureful-travel-lodging-assistance/
- Learn more about clinical trial participation: https://www.tscalliance.org/individuals-families/tsc-clinical-trials/
- Check out TSC clinical trials that are currently recruiting: https://trials.tscalliance.org/