NCT01506141

An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment

An Open-Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Pediatric Patients With Hunter Syndrome and Cognitive Impairment

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Takeda · Industry
• Sex: Male
• Age: 3 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

This extension study of HGT-HIT-045 is designed to collect long-term safety data in pediatric participants with Hunter syndrome and cognitive impairment who are receiving intrathecal (IT) idursulfase-IT and intravenous (IV) Elaprase enzyme replacement therapy.

Conditions

• Hunter Syndrome

Interventions

Type	Name	Description
DRUG	Idursulfase-IT	Idursulfase-IT once monthly via IDDD.
DRUG	Elaprase	Weekly IV infusions of commercially available Elaprase.

Timeline

• Start date: 2010-08-01
• Primary completion: 2024-04-30
• Completion: 2024-04-30
• First posted: 2012-01-09
• Last updated: 2025-08-06
• Results posted: 2025-08-06

Locations

9 sites across 3 countries: United States, Canada, United Kingdom

Regulatory

• FDA-regulated drug study