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

Phase 1

Completed

• Conditions: Mucopolysaccharidosis II
• Interventions: Drug: GC1111_1.0mg/kg; Drug: GC1111_0.5mg/kg; Drug: Elaprase_0.5mg/kg

• Registration Number: NCT01301898
• Lead Sponsor: Green Cross Corporation

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

Detailed Description

Not available

Study Timeline

• Study Start: 2010-05
• Study First Submitted: 2011-02-22
• Study First Submitted QC: 2011-02-22
• Study First Posted: 2011-02-23
• Primary Completion: 2011-03
• Study Completion: 2011-03
• Status Verified: 2012-02
• Last Update Submitted: 2012-02-12
• Last Update Posted: 2012-02-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 31

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
GC1111_1.0mg/kg	GC1111_1.0mg/kg	-
GC1111_0.5mg/kg	GC1111_0.5mg/kg	-
Elaprase_0.5mg/kg	Elaprase_0.5mg/kg	-

Primary Outcome Measures

Name	Time	Method
Primary Outcome	baseline, every 4 weeks	measurement of % change of Urine GAG

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Samsung Medical Center; 🇰🇷 Seoul, Irwon-dong, Gangnam-Gu, Korea, Republic of