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Clinical Trials in South Korea / NCT05422482
Active, not recruiting Phase 1

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

NCT05422482 · tracked via the Priya Life Science South Korea tracker
Phase
Phase 1
Started
2022-09-20
Last updated
2024-10-15

Condition(s) studied

Mucopolysaccharidosis IIHunter Syndrome

Investigational drug(s) / intervention(s)

GC1123

GC1123: ICV-administered Hunterase, Idursulfase-ß

Study 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

Eligibility

Sex
ALL
Min age
18 Months
Max age
18 Years
Healthy volunteers
No
Inclusion Criteria: 1. Patient who has been diagnosed with severe MPS Ⅱ (Hunter syndrome) 2. Patient, aged 1.5 years (18 months) to 18 years at the time of the screening 3. Patient who has received and tolerated a minimum of 12 weeks of treatment with weekly intravenous treatment, and who has received 80% of the total planned infusions within that time frame. 4. Patient who is capable of undergoing neurosurgery, which has been confirmed by neurosurgeons and anesthesiologist. 5. Patient eligible to execute patient evaluation activities during the clinical trial period, as assessed by the investigator 6. Patient whose parents or legal representative are willing to participate in this clinical trial and provide written informed consent form Exclusion Criteria: 1. Patient who has been administered with intrathecal Idursulfase in the past 2. Patient with a history of bone marrow transplantation or cord blood transplant 3. Patient with a history of ventriculoperitoneal shunt or other intracranial surgeries 4. Patient with end-stage multiple organ dysfunction syndrome or other severe diseases 5. Patient who is exposed to malignant neoplasm 6. Patient who has received treatment with any investigational drug or device within 30 days prior to study entry 7. Patient who have experience of hypersensitivity or anaphylaxis to ingredients of the investigational product at the time of screening 8. Patient with a history of bronchotomy/tracheostomy, or patient with acute respiratory disease at the time of screening 9. Patient who is ineligible to participate in the clinical trial due to laboratory test results or other reasons, as determined by the investigator

Primary outcome measure(s)

Trial sites (3)

FacilityCityRegionStatus
Pusan National University Yangsan Hospital Pusan South Korea
Seoul National University Seoul South Korea
Samsung Medical Center Seoul South Korea

More GC Biopharma Corp trials in South Korea

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05422482 on ClinicalTrials.gov ↗ ← All trials in South Korea