This is a multinational, open-label, single-arm trial of adjunctive SVG103 (paxalisib) treatment in adults with FCD-II, TSC, and HME.
This is an open-label, phase 1b/2a study to evaluate safety, tolerability, and pharmacokinetics SVG103 (paxalisib) in 15 patients with Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME). The name of the study drug involved in this study is SVG103 (paxalisib). The trial consists of two parts: the core phase consists of a 4 week prospective baseline period (D-28\~D1) to collect seizure data, followed by a 12-week treatment phase. The extension phase consists of a 24-week treatment period. For participants not continuing in the extension phase, there is a 4-week washout (follow-up) phase. It is expected that about 15 participants will take part in this research study for up to 9 months as long as there is no serious side effects and disease progression. "Investigational" means that the drug is being studied. The U.S. Food and Drug Administration (FDA) has not approved SVG103 (paxalisib) for this specific disease.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
The 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.
Austin Hospital
Heidelberg, Victoria, Australia
The Alfred
Melbourne, Victoria, Australia
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.
Time frame: Up to 36 Weeks
Change from baseline in seizure frequency
Assessed by seizure diaries
Time frame: Baseline to Core Phase: week 1 to 12 and Baseline to Extension Phase: week 13 to 36
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