NCT02455622

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and Weight in Patients With MPS II Who Are Receiving Elaprase and Started Treatment With Elaprase at <6 Years of Age

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Shire · Industry
• Sex: Male
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

This long-term study will provide Elaprase treatment to children enrolled in this study and will utilize data from both enrolled patients and Hunter Outcome Survey (HOS) patient registry data to conduct the primary growth analysis to assess changes in height and weight in patients with Mucopolysaccharidosis II (Hunter syndrome) MPS II.

Conditions

• Hunter Syndrome

Interventions

Type	Name	Description
DRUG	Elaprase for intravenous (IV) infusion	Patients enrolled in this study will receive once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and will be followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reach their 10th birthday, whichever is longer.Height and weight data from HOS will be utilized in the Primary Growth Analysis for this study.

Timeline

• Start date: 2015-10-28
• Primary completion: 2025-07-29
• Completion: 2025-07-29
• First posted: 2015-05-28
• Last updated: 2026-04-02
• Results posted: 2026-04-02

Locations

8 sites across 8 countries: United States, Dominican Republic, Germany, Malaysia, Philippines, Serbia, Thailand, Vietnam