Status:
ACTIVE_NOT_RECRUITING
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ
Lead Sponsor:
GC Biopharma Corp
Conditions:
Mucopolysaccharidosis II
Hunter Syndrome
Eligibility:
All Genders
18-18 years
Phase:
PHASE1
Brief 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 involv...
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 inves...
Eligibility Criteria
Inclusion
- Patient who has been diagnosed with severe MPS Ⅱ (Hunter syndrome)
- Patient, aged 1.5 years (18 months) to 18 years at the time of the screening
- 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.
- Patient who is capable of undergoing neurosurgery, which has been confirmed by neurosurgeons and anesthesiologist.
- Patient eligible to execute patient evaluation activities during the clinical trial period, as assessed by the investigator
- Patient whose parents or legal representative are willing to participate in this clinical trial and provide written informed consent form
Exclusion
- Patient who has been administered with intrathecal Idursulfase in the past
- Patient with a history of bone marrow transplantation or cord blood transplant
- Patient with a history of ventriculoperitoneal shunt or other intracranial surgeries
- Patient with end-stage multiple organ dysfunction syndrome or other severe diseases
- Patient who is exposed to malignant neoplasm
- Patient who has received treatment with any investigational drug or device within 30 days prior to study entry
- Patient who have experience of hypersensitivity or anaphylaxis to ingredients of the investigational product at the time of screening
- Patient with a history of bronchotomy/tracheostomy, or patient with acute respiratory disease at the time of screening
- Patient who is ineligible to participate in the clinical trial due to laboratory test results or other reasons, as determined by the investigator
Key Trial Info
Start Date :
September 20 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 1 2027
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT05422482
Start Date
September 20 2022
End Date
June 1 2027
Last Update
October 15 2024
Active Locations (3)
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1
Pusan National University Yangsan Hospital
Pusan, South Korea, 50612
2
Seoul National University
Seoul, South Korea, 03080
3
Samsung Medical Center
Seoul, South Korea, 06351