This is a multinational, open-label, single-arm trial of adjunctive SVG103 (paxalisib) treatment in adults with FCD-II, TSC, and HME.
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).
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.
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 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 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.
Single group
SVG103
DrugThe study treatment is administered for 3 months during the core phase and for an additional 6 months during the extension phase. Oral, daily, dosage per protocol.
Incidence of Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs (SAEs), Adverse Drug Reactions (ADRs), TEAEs Leading to Discontinuation and Severity of TEAEs
An adverse event (AE) was any untoward medical occurrence in a clinical study patient, temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event was any untoward medical occurrence that, at any dose, results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or any other event that requires scientific judgment.
Change from baseline in seizure frequency
Assessed by seizure diaries
