A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ
Mucopolysaccharidosis II, Hunter Syndrome
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis II
Eligibility Criteria
Inclusion Criteria:
- 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 6 month 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 Criteria:
- 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 had experienced hypersensitivity or anaphylaxis to ingredients of the investigational product
- 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
Sites / Locations
- Pusan National University Yangsan HospitalRecruiting
- Seoul National UniversityRecruiting
- Samsung Medical CenterRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Experimental
GC1123 30mg
GC1123 30mg + GC1123 45mg
GC1123 45mg
30 mg of IP will be administered every 28 days for 6 patients enrolled in Group 1
30 mg of IP will be administered twice every 28 days for 2 patients enrolled in Group 2 45 mg of IP will be administered twice, and any DLT occurrence will be followed-up until 4 weeks after the 2nd 45 mg dose on the 2nd patient. DSMB will determine whether to proceed the administration after reviewing safety and tolerability data from 1) and 2). Group 2 will continue to receive 45 mg of IP administration
45 mg of IP will be administered every 28 days for 4 patients enrolled in Group 3