NCT06031259

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

An Open-Label Extension for Subjects in Studies HGT-HIT-046 and SHP609-302 Evaluating Long-Term Safety of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Subjects With Hunter Syndrome and Cognitive Impairment

• Status: Active Not Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Takeda · Industry
• Sex: Male
• Age: 3 Years – 19 Years
• Healthy volunteers: Not accepted

Summary

The study is an extension of two previous studies (HGT-HIT-046 \[NCT01506141\] and SHP609-302 \[NCT02412787\]). Participants must have completed one of the previous studies. The main aim of this study is to collect more information about the safety of the treatments, idursulfase-IT and elaprase, in children and adults with Hunter syndrome and cognitive impairment. Participants will receive the same treatment as in the previous studies.

Detailed description

The drug being tested in this study is called idursulfase. Idursulfase is being tested for long term safety in participants with Hunter syndrome and cognitive impairment. The study will enroll up to approximately 8 patients. Participants will receive idursulfase-IT via intrathecal drug delivery device (IDDD) once monthly along with elaprase intravenous (IV) infusion, weekly. This multi-center trial will be conducted in France and Canada. The overall time to participate in this study is approximately 4.8 years.

Conditions

• Hunter Syndrome

Interventions

Type	Name	Description
DRUG	Idursulfase-IT	Idursulfase-IT intrathecally via IDDD.
DRUG	Elaprase	Elaprase IV infusion.

Timeline

• Start date: 2024-03-05
• Primary completion: 2029-01-01
• Completion: 2029-01-01
• First posted: 2023-09-11
• Last updated: 2025-11-06

Locations

4 sites across 2 countries: Canada, France

Regulatory

• FDA-regulated drug study