Study of Idursulfase-beta (GC1111) in Hunter Syndrome

Green Cross Corporation

Status and phase: Unknown; Phase 2

Conditions: Mucopolysaccharidosis II

Treatments: Biological: idursulfase; Biological: idursulfase beta

Study type: Interventional

Funder types: Industry

Identifiers

NCT02663024

GC1111B_P2

Details and patient eligibility

About

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.

Full 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.

Enrollment: 20 estimated patients

Sex: Male

Ages: 5 to 35 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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20 participants in 4 patient groups

Arm 1

Experimental group

Description:

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

Treatment:

Biological: idursulfase beta

Arm 2

Experimental group

Description:

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

Treatment:

Biological: idursulfase beta

Arm 3

Experimental group

Description:

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

Treatment:

Biological: idursulfase beta

Arm 4

Active Comparator group

Description:

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

Treatment:

Biological: idursulfase