NCT00607386

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 28 (actual)
• Sponsor: Shire · Industry
• Sex: Male
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

The objective of this study is to determine the safety of once weekly dosing of idursulfase 0.5 mg/kg administered by intravenous (IV) infusion for male Hunter syndrome patients ≤ 5 years old.

Detailed description

This study will provide a basis for evaluating the safety of idursulfase administered to Hunter syndrome patients who are ≤ 5 years old. Additionally, this study will provide a basis for evaluating the idursulfase single- and repeated-dose pharmacokinetic profiles as well as the pharmacodynamic effect (as measured by urinary GAG excretion) in this pediatric population. Additional exploratory measures will include abdominal ultrasound measurements of liver and spleen volumes, assessments of growth with comparisons to normal population growth data, assessments of annualized growth velocity, assessments of routine developmental milestones using the Denver II, and assessments of clinical events, including the first occurrence of certain hearing-related events (e.g., hearing loss, otitis media), respiratory-related events (e.g., upper and lower respiratory infections), and specific surgical procedures (e.g., adenoidectomy, placement of PE tubes). All patients in this open-label study will receive once-weekly infusions of idursulfase at a dose of 0.5 mg/kg.

Conditions

• Hunter Syndrome
• Mucopolysaccharidosis II
• MPS II

Interventions

Type	Name	Description
BIOLOGICAL	Idursulfase	Solution for intravenous infusion, 0.5 mg/kg weekly

Timeline

• Start date: 2007-12-31
• Primary completion: 2011-07-08
• Completion: 2011-07-08
• First posted: 2008-02-05
• Last updated: 2021-06-08
• Results posted: 2013-11-07

Locations

3 sites across 3 countries: Brazil, Poland, Taiwan