A Study of GC1111 in Hunter Syndrom Patients

Phase 3, Double-blind, Randomized, Active-controlled (Part 1) and Open-labeled, Historical Placebo Controlled (Part 2) Study to Evaluate the Efficacy of Hunterase (Idursulfase-beta) in Hunter Syndrome (Mucopolysaccharidosis II) Patients

The objective of this study is to evaluate the efficacy of GC1111 in Hunter Syndrome Patients

Study Overview

• Status: Completed
• Conditions: Hunter Syndrome
• Intervention / Treatment: Combination product: GC1111; Combination product: Comparator

• Study Type: Interventional
• Enrollment (Actual): 32
• Phase: Phase 3

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Samsug Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patients diagnosed with Hunter syndrome
• Male at the age of ≥ 5
• Adequate abilities (including 6-MWT) to participate in this study in the opinion of the investigator.
• Voluntarily signed written informed consent to participation in this study
• Consent to contraception

Exclusion Criteria:

• Prior treatment with iduronate-2-sulfatase ERT
• History of bronchotomy, bone marrow trasplanation, or cord blood transplanation.
• Known hypersensitivity reactions to any of the components of the invetigational product
• Prior or planned administration of other investigational products within 30 days before treatment with the investigational product in this study or duirng this study.
• Unable to perform 6-MWT.
• Female

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: GC1111; In part 1, all participants who randomized into GC1111 arm should receive the GC1111 for 52 weeks. In part 2, all enrolled participants should receive the GC1111 for 52 weeks.	Combination product: GC1111; GC1111 is 0.5mg per kg of body weight once weekly to be administered slowly by intravenous infusion.
Active Comparator: Comparator (Part 1); In Part 1, all participants who randomized into comparator arm should receive the GC1111 for 52 weeks.	Combination product: Comparator; Comparator is 0.5mg per kg of body weight once weekly to be administered slowly by intravenous infusion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change in 6-MWT	at Week 53 from baseline

Collaborators and Investigators

• Sponsor: Green Cross Corporation
• Investigators: Principal Investigator: DongKyu Jin, M.D., Ph.D., Samsung Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): March 9, 2017
• Primary Completion (Actual): February 15, 2022
• Study Completion (Actual): February 15, 2022

Study Registration Dates

• First Submitted: April 16, 2019
• First Submitted That Met QC Criteria: April 16, 2019
• First Posted (Actual): April 19, 2019

Study Record Updates

• Last Update Posted (Actual): April 16, 2024
• Last Update Submitted That Met QC Criteria: April 14, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Mucopolysaccharidosis II; MPS II; GC1111; idursulfase-beta; IDS
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Syndrome; Mucopolysaccharidosis II; Mucopolysaccharidoses
• Other Study ID Numbers: GC1111_P3

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No