To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
Randomized, Single-blind, Active-controlled, Phase 1/2 Study to Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) Patients
1 other identifier
interventional
31
1 country
1
Brief Summary
The purpose of this study is to evaluate the safety and efficacy of GC1111 (recombinant human iduronate-w-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) patients
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started May 2010
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 1, 2010
CompletedFirst Submitted
Initial submission to the registry
February 22, 2011
CompletedFirst Posted
Study publicly available on registry
February 23, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2011
CompletedFebruary 14, 2012
February 1, 2012
10 months
February 22, 2011
February 12, 2012
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Primary Outcome
measurement of % change of Urine GAG
baseline, every 4 weeks
Secondary Outcomes (1)
Secondary Outcome
baseline, every 12 weeks
Study Arms (3)
GC1111_0.5mg/kg
EXPERIMENTALGC1111_1.0mg/kg
EXPERIMENTALElaprase_0.5mg/kg
ACTIVE COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- Patients with diagnosis of MPS II based on both clinical and biochemical criteria
- Male, ages 6 to 35 years old
- Patients who are able to comply with the study requirements
- Patients who have given voluntary written consent to participate in the study
- Patients who is acceptable for using an appropriate method of contraception
You may not qualify if:
- History of a tracheostomy or a bone marrow transplant
- Known hypersensitivity to idursulfase
- Known shock to idursulfase
- History of receiving treatment with another investigational therapy within the past 30 days
- History of a stem cell transplant
- Known hypersensitivity to any of the components of idursulfase
- Female
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Samsung Medical Center
Seoul, Irwon-dong, Gangnam-Gu, 135-710, South Korea
Related Publications (1)
Sohn YB, Cho SY, Park SW, Kim SJ, Ko AR, Kwon EK, Han SJ, Jin DK. Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter syndrome). Orphanet J Rare Dis. 2013 Mar 18;8:42. doi: 10.1186/1750-1172-8-42.
PMID: 23497636DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- SINGLE
- Who Masked
- PARTICIPANT
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 22, 2011
First Posted
February 23, 2011
Study Start
May 1, 2010
Primary Completion
March 1, 2011
Study Completion
March 1, 2011
Last Updated
February 14, 2012
Record last verified: 2012-02