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

NCT05422482 | Active Not Recruiting Phase 1 DMC

• 1 other identifier: GC1123_MPS2_P0101
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 3

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 involvement and are receiving treatment with intravenous drug

Conditions

Mucopolysaccharidosis II; Hunter Syndrome

Outcome Measures

Primary Outcomes (3)

• Incidence and frequency of serious adverse events (SAEs)

  Incidence and frequency of serious adverse events (SAEs) after administration of ICV-Hunterase (GC1123)

  Every 28 days from Week 1 through study completion (about 110 weeks)

• Frequency and characteristics (severity, outcome, etc.) of adverse events

  Frequency and characteristics (severity, outcome, etc.) of adverse events after administration of ICV-Hunterase (GC1123)

  Every 28 days from Week 1 through study completion (about 110 weeks)

• Presence of clinically significant abnormal echocardiography results

  Presence of clinically significant abnormal echocardiography results after administration of ICV-Hunterase (GC1123); phase I only

  Week 1 to Phase I study completion (about 26 weeks)

Secondary Outcomes (12)

• Pharmacokinetic (PK) parameters - Cmax

  Week 2 to Week 22

• Pharmacokinetic (PK) parameters - Tmax

  Week 2 to Week 22

• Pharmacokinetic (PK) parameters - AUClast

  Week 2 to Week 22

• Pharmacokinetic (PK) parameters - AUCinf

  Week 2 to Week 22

• Pharmacokinetic (PK) parameters - t1/2

  Week 2 to Week 22

Other Outcomes (4)

• Development Function assessed by Bayley Scales of Infant and Toddler Development-III and/or Kaufman Assessment Battery for Children-II (BSID-III/KABC-II)

  Approximately every 6 months (Week 1 [baseline], Week 26, Week 54, Week 82, Week 110)

• Adaptive Function assessed by Vineland Adaptive Behavior Scales 2nd Ed. (VABS-II)

  Approximately every 6 months (Week 1 [baseline], Week 26, Week 54, Week 82, Week 110)

• Quality of Life (Survey) assessed by Infant and Toddler Quality of Life Questionnaire (ITQOL) and/or Childhood Health Questionnaire parent form (CHQ-PF50)

  Approximately every 6 months (Week 1 [baseline], Week 26, Week 54, Week 82, Week 110)

Study Arms (1)

GC1123 30mg

EXPERIMENTAL

30 mg of IP will be administered every 28 days for all enrolled patients

Biological: GC1123

Interventions

GC1123 BIOLOGICAL

ICV-administered Hunterase, Idursulfase-ß

GC1123 30mg

Eligibility Criteria

• Age: 18 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• GC Biopharma Corp: lead

Study Sites (3)

Pusan National University Yangsan Hospital

Pusan, 50612, South Korea

Location

Seoul National University

Seoul, 03080, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

MeSH Terms

Conditions

Mucopolysaccharidosis II

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 31, 2022
• First Posted: June 16, 2022
• Study Start: September 20, 2022
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027
• Last Updated: October 15, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

KR (3)