Safety and Efficacy of Hunterase (GC1111)

To Evaluate the Safety and Efficacy of Hunterase(Idursulfase-beta) in Hunter Syndrome Patients < 6 Years of Age Receiving Idursulfase Enzyme Replacement Therapy

The objective of this study is to determine the safety and efficacy of once weekly dosing of idursulfase-beta 0.5mg/kg administered by intravenous(IV) infusion for Hunter syndrome patients < 6 years old.

Study Overview

• Status: Completed
• Conditions: Hunter Syndrome
• Intervention / Treatment: Biological: Hunterase

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 3

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Samsug Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 5 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

Inclusion Criteria:

1. The patient has a diagnosis of Hunter syndrome based upon biochemical criteria:

   • as measured in plasma, leukocytes, or fibroblasts,

     • a deficiency in iduronate-2-sulfatase (I2S) enzyme activity of ≤ 10 % of the lower limit of the normal range

   • That corresponds to one or more of the following:

     • a normal enzyme activity level of one other sulfatase
     • Confirmed as MPS2 by genetic test results
     • shows clinical symptoms/ visible signs of MPS2
2. < 6 years old and male
3. Patients who are able to comply with the study requirements
4. The patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study

Exclusion Criteria:

1. The patient has had a tracheostomy
2. The patient has known severe hypersensitivity or shock to any of the components of idursulfase
3. The patient has received treatment with another investigational therapy within 30 days prior to enrollment
4. History of a stem cell transplant
5. The patient has known severe hypersensitivity or shock to any of the components of test drug(excipient etc)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Test drug; Idursulfase-beta	Biological: Hunterase; once weekly, 0.5mg/kg IV infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of adverse events	One year

Secondary Outcome Measures

Outcome Measure	Time Frame
change of anti-idursulfase-beta antibody status	baseline and one year
Percent Change of Urine GAG	baseline to 53 weeks

Collaborators and Investigators

• Sponsor: Green Cross Corporation
• Investigators: Principal Investigator: Dong-Kyu Jin, Samsung Medical Center, Seoul, Republic of Korea

Study record dates

Study Major Dates

• Study Start: July 1, 2012
• Primary Completion (Actual): September 1, 2013
• Study Completion (Actual): September 1, 2013

Study Registration Dates

• First Submitted: July 16, 2012
• First Submitted That Met QC Criteria: July 19, 2012
• First Posted (Estimate): July 20, 2012

Study Record Updates

• Last Update Posted (Estimate): July 8, 2014
• Last Update Submitted That Met QC Criteria: July 7, 2014
• Last Verified: July 1, 2014

More Information

Terms related to this study

• Keywords: Hunter syndrome; GC1111; idursulfase-beta; Hunterase
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidoses; Syndrome; Mucopolysaccharidosis II
• Other Study ID Numbers: GC1111C