NCT05422482

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ

An Open-Label, Phase 1 and Extension Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ Who Have Central Nervous System Involvement and Are Receiving Treatment with Intravenous Drug

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: GC Biopharma Corp · Industry
• Sex: All
• Age: 18 Months – 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intracerebroventricular GC1123 in patients with MPS Ⅱ who have central nervous system involvement and are receiving treatment with intravenous drug

Detailed description

This study is designed as prospective, open-label, phase I and extension study. Safety, tolerability, pharmacokinetic, and pharmacodynamic properties of repeat-dose treatment of ICV-administered investigational product will be studied in patients undergoing standard treatments. Patients will undergo cerebrospinal fluid (CSF) reservoir device implantation surgery on their scalps, and the reservoirs will be used to administer GC1123 to the cerebral ventricles monthly (every 28 days). The planned administering dose is 30 mg. After the 2nd dose on the 6th patient, Data and Safety Monitoring Boards (DSMB) will evaluate the safety and tolerability data of GC1123. The planned duration of the sutdy is total about 2 years (phase I and extension)

Conditions

• Mucopolysaccharidosis II
• Hunter Syndrome

Interventions

Type	Name	Description
BIOLOGICAL	GC1123	ICV-administered Hunterase, Idursulfase-ß

Timeline

• Start date: 2022-09-20
• Primary completion: 2027-06-01
• Completion: 2027-06-01
• First posted: 2022-06-16
• Last updated: 2024-10-15

Locations

3 sites across 1 country: South Korea