To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients

Randomized, Single-blind, Active-controlled, Phase 1/2 Study to Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) Patients

Status

Completed

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01301898

Enrollment

Registered

2011-02-23

Start date

2010-05-31

Completion date

2011-03-31

Last updated

2012-02-14

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

Conditions

Mucopolysaccharidosis II

Keywords

Hunter syndrome, MPS II, Idursulfase

Brief summary

The purpose of this study is to evaluate the safety and efficacy of GC1111 (recombinant human iduronate-w-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) patients

Interventions

DRUGGC1111_0.5mg/kg

DRUGGC1111_1.0mg/kg

DRUGElaprase_0.5mg/kg

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

SINGLE (Subject)

Eligibility

Sex/Gender

MALE

Age

6 Years to 35 Years

Healthy volunteers

Inclusion criteria

1. Patients with diagnosis of MPS II based on both clinical and biochemical criteria 2. Male, ages 6 to 35 years old 3. Patients who are able to comply with the study requirements 4. Patients who have given voluntary written consent to participate in the study 5. Patients who is acceptable for using an appropriate method of contraception

Exclusion criteria

1. History of a tracheostomy or a bone marrow transplant 2. Known hypersensitivity to idursulfase 3. Known shock to idursulfase 4. History of receiving treatment with another investigational therapy within the past 30 days 5. History of a stem cell transplant 6. Known hypersensitivity to any of the components of idursulfase 7. Female

Design outcomes

Primary

Measure	Time frame	Description
Primary Outcome	baseline, every 4 weeks	measurement of % change of Urine GAG

Secondary

Measure	Time frame	Description
Secondary Outcome	baseline, every 12 weeks	* Measurements of the six-minute-walk test (6-MWT) * Measurements of liver volume * Measurements of heart size and heart function * Measurements of joint range of motion * Measurements of urine GAG levels * Measurements of pulmonary function * Evaluate the safety: vital signs, physical examination, laboratory tests, adverse events, immunogenecity

Countries

South Korea

Outcome results

None listed

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