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

Green Cross Corporation

Status and phase: Completed; Phase 2; Phase 1

Conditions: Mucopolysaccharidosis II

Treatments: Drug: GC1111_1.0mg/kg; Drug: GC1111_0.5mg/kg; Drug: Elaprase_0.5mg/kg

Study type: Interventional

Funder types: Industry

Identifiers

NCT01301898

GC1111_P1/2

Details and patient eligibility

About

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

Enrollment: 31 patients

Sex: Male

Ages: 6 to 35 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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31 participants in 3 patient groups

GC1111_0.5mg/kg

Experimental group

Treatment:

Drug: GC1111_0.5mg/kg

GC1111_1.0mg/kg

Experimental group

Treatment:

Drug: GC1111_1.0mg/kg

Elaprase_0.5mg/kg

Active Comparator group

Treatment:

Drug: Elaprase_0.5mg/kg