Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME)

Korábbi kezelés

• Immunocompromised participants, defined as acquired immune deficiency syndrome (AIDS), cancer, malnutrition, and certain genetic disorders or undergoing treatment with anticancer drugs, radiation therapy, and stem cell or organ transplant.
• Participants being considered for brain surgery during the study or has undergone brain surgery within 6 months prior to screening visit for epilepsy or any other reason.

Más betegség

• Clinically significant hepatic, renal, pulmonary, gastrointestinal, neurological (other than epilepsy; HME; TSC; FCD-II), autoimmune, immunological, infections, hematological, malignant conditions that may interfere with or impact the participation in the study or study conduct, as determined but the investigator or sponsor.
• Participants who have an active central nervous system (CNS) infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain imaging (magnetic resonance imaging [MRI]).
• Participants with active pneumonitis that are clinically symptomatic.
• Participants with a history of myocardial infarction or coronary artery disease or clinically significant ECG abnormality.

Laborértékek

• Participants with HbA1c levels ≥9.0%, or with hyperglycemia or diabetes requiring insulin therapy, or significant uncontrolled hyperglycaemia or diabetes mellitus that would compromise patient safety, as determined by the investigator.
• Participants who have clinically significant hepatic, renal and blood laboratory values at baseline period.
• Participants with known sensitivity or allergy to any component in the investigational product(s) (microcrystalline cellulose, lactose monohydrate, croscarmellose sodium, and colloidal silicon dioxide).

• 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.
• 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.
• 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.