NCT07287202

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

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), Followed by Long-Term Treatment

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Sovargen · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This is a multinational, open-label, single-arm trial of adjunctive SVG103 (paxalisib) treatment in adults with FCD-II, TSC, and HME.

Detailed description

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.

Conditions

• Focal Cortical Dysplasia
• Tuberous Sclerosis Complex (TSC)
• Hemimegalencephaly

Interventions

Type	Name	Description
DRUG	SVG103	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.

Timeline

• Start date: 2026-03-01
• Primary completion: 2026-12-01
• Completion: 2027-06-01
• First posted: 2025-12-17
• Last updated: 2025-12-23

Locations

2 sites across 1 country: Australia

Regulatory

• FDA-regulated drug study