Safety and Efficacy of Hunterase

NCT01645189 | Completed Phase 3

• 1 other identifier: GC1111C
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Hunter Syndrome

Keywords

Hunter syndrome; idursulfase-beta; Hunterase; GC1111

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events

  One year

Secondary Outcomes (2)

• change of anti-idursulfase-beta antibody status

  baseline and one year

• Percent Change of Urine GAG

  baseline to 53 weeks

Study Arms (1)

Test drug

EXPERIMENTAL

Idursulfase-beta

Biological: Hunterase

Interventions

Hunterase BIOLOGICAL

once weekly, 0.5mg/kg IV infusion

Test drug

Eligibility Criteria

• Age: Up to 5 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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
• \< 6 years old and male
• Patients who are able to comply with the study requirements
• The patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study

You may not qualify if:

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

Sponsors & Collaborators

• Green Cross Corporation: lead

Study Sites (1)

Samsug Medical Center

Seoul, South Korea

Location

MeSH Terms

Conditions

Mucopolysaccharidosis II

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Dong-Kyu Jin

  Samsung medical center, Seoul, Republic of Korea

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 16, 2012
• First Posted: July 20, 2012
• Study Start: July 1, 2012
• Primary Completion: September 1, 2013
• Study Completion: September 1, 2013
• Last Updated: July 8, 2014

Record last verified: 2014-07

Locations

KR (1)