A Study of GC1111 in Hunter Syndrom Patients

NCT03920540 | Completed Phase 3

• 1 other identifier: GC1111_P3
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Hunter Syndrome

Keywords

GC1111; Mucopolysaccharidosis II; MPS II; idursulfase-beta; IDS

Outcome Measures

Primary Outcomes (1)

• Change in 6-MWT

  at Week 53 from baseline

Study Arms (2)

GC1111

EXPERIMENTAL

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

Comparator (Part 1)

ACTIVE COMPARATOR

In Part 1, all participants who randomized into comparator arm should receive the GC1111 for 52 weeks.

Combination Product: Comparator

Interventions

GC1111 COMBINATION_PRODUCT

GC1111 is 0.5mg per kg of body weight once weekly to be administered slowly by intravenous infusion.

GC1111

Comparator COMBINATION_PRODUCT

Comparator is 0.5mg per kg of body weight once weekly to be administered slowly by intravenous infusion.

Comparator (Part 1)

Eligibility Criteria

• Age: 5 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Green Cross Corporation: lead

Study Sites (1)

Samsug Medical Center

Seoul, South Korea

Location

MeSH Terms

Conditions

Mucopolysaccharidosis II; Sudden Infant Death

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; Death, Sudden; Death; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Infant Death

Study Officials

• DongKyu Jin, M.D., Ph.D.

  Samsung Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 16, 2019
• First Posted: April 19, 2019
• Study Start: March 9, 2017
• Primary Completion: February 15, 2022
• Study Completion: February 15, 2022
• Last Updated: April 16, 2024

Record last verified: 2024-04

Locations

KR (1)