An Open-Label Phase 1b/2a Study to Evaluate the Safety and Tolerability of Oral SVG103 (Paxalisib) in Adults With Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME)
This is a multinational, open-label, single-arm trial of adjunctive SVG103 (paxalisib) treatment in adults with FCD-II, TSC, and HME.
⁃ Participants diagnosed with:
∙ FCD-II: diagnosis of FCD Type II based on clinical symptoms and confirmed by a positive magnetic resonance imaging (MRI) or histological/pathological analysis of brain tissue, or
‣ TSC: diagnosis of TSC by either clinical or genetic diagnostic criteria as documented in the participant's medical record, or
‣ HME: Definitive HME confirmed with MRI.
⁃ Male or female between the ages of 18 and 65 years of age (inclusive).
⁃ History of failure to control seizures despite at least 2 ASMs at appropriate dosages and duration of treatment.
⁃ Participants must have experienced at least 8 countable seizures per month for 2 of the 3 months as documented in historical seizure diaries before the baseline period.
• 6\. If participants are on a ketogenic or modified atkins diet, that the regimen can remain unchanged throughout the study, in the opinion of the investigator.
• 7\. Participants with Neurostimulation devices (i.e. Vagus Nerve Stimulation (VNS), Responsive Neuro Stimulation (RNS), Deep Brain Stimulation (DBS) who meet all of the following conditions:
• The device has been implanted for ≥1 year prior to the screening visit.
• The settings must have remained constant for 3 months prior to the screening visit and can remain constant throughout the study, in the opinion of the investigator.
• The battery is expected to last throughout the study. 8. A participant/caregiver or LAR willing to give written informed consent, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures.