Safety and Efficacy of Hunterase

Hunter Syndrome Clinical Trial

— GC1111  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01645189
• Other study ID #: GC1111C
• Status: Completed
• Phase: Phase 3
• First received: July 16, 2012
• Last updated: July 7, 2014
• Start date: July 2012
• Est. completion date: September 2013

Study information

• Verified date: July 2014
• 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 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: September 2013
• Est. primary completion date: September 2013
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 5 Years

Eligibility

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 Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hunter Syndrome
• Mucopolysaccharidosis II

Intervention

Biological:
• Hunterase
once weekly, 0.5mg/kg IV infusion

Locations

Country	Name	City	State
Korea, Republic of	Samsug Medical Center	Seoul

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	Incidence of adverse events		One year	Yes
Secondary	change of anti-idursulfase-beta antibody status		baseline and one year	Yes
Secondary	Percent Change of Urine GAG		baseline to 53 weeks	No