Safety and Efficacy of Hunterase

Phase 3

Completed

• Conditions: Hunter Syndrome
• Interventions: Biological: Hunterase

• Registration Number: NCT01645189
• Lead Sponsor: Green Cross Corporation

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2012-07
• Study First Submitted: 2012-07-16
• Study First Submitted QC: 2012-07-19
• Study First Posted: 2012-07-20
• Primary Completion: 2013-09
• Study Completion: 2013-09
• Status Verified: 2014-07
• Last Update Submitted: 2014-07-07
• Last Update Posted: 2014-07-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 6

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

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Test drug	Hunterase	Idursulfase-beta

Primary Outcome Measures

Name	Time	Method
Incidence of adverse events	One year

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Samsug Medical Center; 🇰🇷 Seoul, Korea, Republic of