Safety and Efficacy of Hunterase

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01645189
• Date of registration: 16/07/2012
• Prospective Registration: No
• Primary sponsor: Green Cross Corporation
• Public title: Safety and Efficacy of Hunterase GC1111
• Scientific title: To Evaluate the Safety and Efficacy of Hunterase(Idursulfase-beta) in Hunter Syndrome Patients < 6 Years of Age Receiving Idursulfase Enzyme Replacement Therapy
• Date of first enrolment: July 2012
• Target sample size: 6
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01645189
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

Korea, Republic of

Contacts

• Name: Dong-Kyu Jin
• Affiliation: Samsung medical center, Seoul, Republic of Korea

Key inclusion & exclusion criteria

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)



Age minimum: N/A
Age maximum: 5 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Hunter Syndrome

Intervention(s)

Biological: Hunterase

Primary Outcome(s)

Incidence of adverse events [Time Frame: One year]

Secondary Outcome(s)

change of anti-idursulfase-beta antibody status [Time Frame: baseline and one year]

Percent Change of Urine GAG [Time Frame: baseline to 53 weeks]

Secondary ID(s)

GC1111C

Source(s) of Monetary Support

Please refer to primary and secondary sponsors