NCT02412787

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction With Elaprase® in Patients With Hunter Syndrome and Cognitive Impairment

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 56 (actual)
• Sponsor: Shire · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This extension study will allow participants that completed Study HGT-HIT-094 to continue receiving Elaprase treatment in conjunction with idursulfase IT or to continue receiving Elaprase treatment and begin concurrent IT treatment for those that did not receive idursulfase IT treatment in Study HGT-HIT-094.

Conditions

• Hunter Syndrome

Interventions

Type	Name	Description
DRUG	Idursulfase-IT	Participants received 10 mg of idursulfase-IT intrathecally via IDDD or LP once every 28 days. Participants who were younger than 3 years of age received an adjusted dose of 7.5 mg (\>8 months to 30 months of age) and 10 mg (\>30 months to 3 years of age).
DRUG	Elaprase	Participants received intravenous (IV) Elaprase infusions at a minimum of 48 hours after IT administration of idursulfase-IT.

Timeline

• Start date: 2015-04-14
• Primary completion: 2024-04-18
• Completion: 2024-04-18
• First posted: 2015-04-09
• Last updated: 2025-06-19
• Results posted: 2025-06-19

Locations

9 sites across 7 countries: United States, Australia, Canada, France, Mexico, Spain, United Kingdom