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