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

Mucopolysaccharidosis II Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01301898
• Other study ID #: GC1111_P1/2
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: February 22, 2011
• Last updated: February 12, 2012
• Start date: May 2010
• Est. completion date: March 2011

Study information

• Verified date: February 2012
• Source: Green Cross Corporation
• Contact: n/a
• Is FDA regulated: No
• Health authority: Korea: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 31
• Est. completion date: March 2011
• Est. primary completion date: March 2011
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years to 35 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Subject), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis II

Intervention

Drug:
• GC1111_0.5mg/kg

• GC1111_1.0mg/kg

• Elaprase_0.5mg/kg

Locations

Country	Name	City	State
Korea, Republic of	Samsung Medical Center	Seoul	Irwon-dong, Gangnam-Gu

Sponsors (1)

Lead Sponsor	Collaborator
Green Cross Corporation

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Primary Outcome	measurement of % change of Urine GAG	baseline, every 4 weeks	No
Secondary	Secondary Outcome	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	baseline, every 12 weeks	Yes