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

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03920540

Enrollment

Registered

2019-04-19

Start date

2017-03-09

Completion date

2022-02-15

Last updated

2024-04-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hunter Syndrome

Keywords

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

Brief summary

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

Interventions

COMBINATION_PRODUCTGC1111

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

COMBINATION_PRODUCTComparator

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

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

DOUBLE (Subject, Investigator)

Eligibility

Sex/Gender

MALE

Age

5 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

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

Countries

South Korea

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026