NCT04153175

Study to Assess Intracerebroventricular (ICV) Delivery of CT-010 in Subjects With Focal Seizures, With Temporal Lobe Onset With or Without Secondary Generalization

A Phase 2, Double-Blind, Randomized, Placebo-Controlled Study to Assess Intracerebroventricular (ICV) Delivery of CT-010 Via an Implantable Pump and a Cranial Port and Double Lumen Catheter (ICVRX) in Subjects With Focal Seizures, With Temporal Lobe Onset With or Without Secondary Generalization

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: Cerebral Therapeutics LLC · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2 double-blind, randomized, placebo-controlled study to assess the safety and efficacy of ICV delivery of CT-010 via an implantable pump and a cranial port and dual lumen catheter (CIC) in subjects with focal seizures, with temporal lobe onset with or without secondary generalization. Up to 70 subjects will be enrolled. Eligible subjects will be randomized in a 1:1 ratio to either CT-010 or placebo treatment. Up to 20 clinical centers will be enrolled.

Detailed description

Epilepsy patients that are refractory to oral anti-epileptic drug (AED) treatment have significantly higher mortality, higher morbidity, higher economic costs and diminished quality of life compared to those who suffer from epilepsy that can be adequately controlled with medical management. Current options for refractory patients include neurosurgical brain resection, responsive neurostimulation, and vagal nerve stimulation. None of these options is satisfactory due to the low applicability of surgery for patients with poorly localized or multifocal seizures and the limited success of currently available alternative treatment options. In this study, patients with medically refractory focal epilepsy will be treated with intracerebroventricular (ICV) administration of CT-010, a reformulation of valproate, using an implantable drug pump system. This is a randomized, double-blind Phase 2 study evaluating the efficacy and safety of this therapy. Clinical assessments, adverse events (AEs), seizure diaries, concomitant medications, blood samples and cerebrospinal fluid (CSF) will be collected and reviewed at designated time points. Magnetic resonance imaging (MRI) and electroencephalography (EEG) will also be performed. Subjects will have their surgery, dose changes and pharmacokinetics performed in an inpatient setting. Subjects will be enrolled based on Inclusion/Exclusion Criteria and undergo a 6-week baseline period confirming and establishing monthly seizure rate. Following the baseline period subjects will undergo system implant and a 1-month implant recovery period. Following successful implant and recovery subjects will be randomized to either active therapy or placebo for a 3-month blinded evaluation period. At the conclusion of the blinded evaluation period, subjects will roll into an Open Label Extension Period, CLN100P.02. Subjects that were randomized to placebo will receive active therapy during CLN100P.02.

Conditions

• Refractory Epilepsy

Interventions

Type	Name	Description
COMBINATION_PRODUCT	ICV delivery of CT-010 via an implantable pump and a cranial port and dual lumen catheter (CIC)	Subjects will be implanted with a drug delivery pump and catheters leading to the ICV space allowing delivery of CT-010.
COMBINATION_PRODUCT	Placebo delivery via an implantable pump and a cranial port and dual lumen catheter (CIC)	Subjects will be implanted with a drug delivery pump and catheters leading to the ICV space allowing delivery of a placebo (saline).

Timeline

• Start date: 2020-08-21
• Primary completion: 2024-03-01
• Completion: 2024-03-01
• First posted: 2019-11-06
• Last updated: 2024-03-13

Locations

11 sites across 2 countries: Australia, Israel

Regulatory

• FDA-regulated drug study