Study of Idursulfase-beta (GC1111) in Hunter Syndrome

NCT ID: NCT02663024

Last Updated: 2016-01-26

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-12-31
• Study Completion Date: 2020-06-30

Brief Summary

This study evaluates the efficacy and safety of three doses of GC1111 in patients with Hunter Syndrome. Participants will be randomized to one of three doses of GC1111 or comparator.

Detailed Description

This is a randomized, double-blind, active-controlled, dose-ranging study, where patient will receive one of the three doses of GC1111 (0.5 mg/kg, 1.0 mg/kg, and 1.5 mg/kg) or ELAPRASE 0.5 mg/kg. Approximately 20 patients will be administrated each study drug once every week as an iv infusion for 24 weeks. Efficacy of GC1111 will be evaluated in Six-Minute Walk Test (6MWT), urine Glycosaminoglycans(uGAG), liver and spleen volume, percent predicted Forced Vital Capacity(FVC), and cardiac size and function. Also immunogenicity, Pharmacokinetics(PK) and safety will be evaluated.

Conditions

Mucopolysaccharidosis II

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Arm 1

0.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks

Group Type: EXPERIMENTAL

idursulfase beta

Intervention Type: BIOLOGICAL

IV, weekly infusion for 24 weeks

Arm 2

1.0 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks

Group Type: EXPERIMENTAL

idursulfase beta

Intervention Type: BIOLOGICAL

IV, weekly infusion for 24 weeks

Arm 3

1.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks

Group Type: EXPERIMENTAL

idursulfase beta

Intervention Type: BIOLOGICAL

IV, weekly infusion for 24 weeks

Arm 4

0.5mg/kg, iv, weekly infusion of idursulfase for 24 weeks

Group Type: ACTIVE_COMPARATOR

idursulfase

Intervention Type: BIOLOGICAL

0.5 mg/kg, iv, weekly infusion for 24 weeks

Interventions

idursulfase beta

IV, weekly infusion for 24 weeks

Intervention Type: BIOLOGICAL

idursulfase

0.5 mg/kg, iv, weekly infusion for 24 weeks

Intervention Type: BIOLOGICAL

Other Intervention Names

GC1111; Elaprase

Eligibility Criteria

Inclusion Criteria

• Male patients between 5 and 35 years of age
• Informed consent form signed
• Patients diagnosed with hunter syndrome
• Previously untreated with an enzyme replacement therapy

Exclusion Criteria

• History of tracheostomy, bone marrow transplant, or cord blood transplant
• Treatment with another investigational product within 30 days prior to the start of study drug
• Known hypersensitivity of any of the ingredients of study drug
• Patient with severe hunter syndrome who cannot perform 6MWT
• Female patients

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 35 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Green Cross Corporation

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Central Contacts

Sangmi Kang

Role: CONTACT

sm6220@greencross.com

82-31-260-1957

Other Identifiers

GC1111B_P2

Identifier Type: -

Identifier Source: org_study_id