Safety and Efficacy of Hunterase

NCT ID: NCT01645189

Last Updated: 2014-07-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-07-31

Study Completion Date

2013-09-30

Brief Summary

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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

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Conditions

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Hunter Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Test drug

Idursulfase-beta

Group Type EXPERIMENTAL

Hunterase

Intervention Type BIOLOGICAL

once weekly, 0.5mg/kg IV infusion

Interventions

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Hunterase

once weekly, 0.5mg/kg IV infusion

Intervention Type BIOLOGICAL

Eligibility Criteria

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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)
Maximum Eligible Age

5 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Green Cross Corporation

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Dong-Kyu Jin

Role: PRINCIPAL_INVESTIGATOR

Samsung medical center, Seoul, Republic of Korea

Locations

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Samsug Medical Center

Seoul, , South Korea

Site Status

Countries

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South Korea

Other Identifiers

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GC1111C

Identifier Type: -

Identifier Source: org_study_id

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