Extension Paediatric stdy of Intrathecal IdursulfaseIT with Elaprase®
Research type
Research Study
Full title
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
IRAS ID
169136
Contact name
Simon Allan Jones
Contact email
Sponsor organisation
Shire Human Genetic Therapies, Inc
Eudract number
2014-004143-13
Duration of Study in the UK
4 years, 4 months, 30 days
Research summary
This study is to provide ongoing treatment with the study drug (idursulfase- IT) to patients who received idursulfase -IT in the main Study and to initiate treatment in patients who received no idursulfase IT in the main Study.
The long term safety of the study drug administered in conjunction with Elaprase® on the severe form of Hunter syndrome when given directly into the cerebrospinal fluid (CSF) will be evaluated.
MPS II is a rare, inherited disease that affects mainly males, in which an enzyme (iduronate-2-sulfatase, I2S) is missing or not working properly, leading to progressive, serious, life limiting disease. In addition to the physical problems, about two thirds of MPS II patients develop problems in the central nervous system (CNS, the brain and spinal cord) and are characterised as severe.
The currently approved therapy for Hunter syndrome is Elaprase, which has shown to provide some clinical benefit. It can’t enter the Blood brain barrier and so has limited action on the CNS.
Idursulfase-IT was developed specifically for delivery into the CSF to access CNS tissues.
Approximately 42 male paediatric participants who completed Study HGT-HIT-094 will take part in this study for up to about 3 years.
Participants who provide written consent and meet all eligibility criteria may enrol into the study to receive the treatment (idursulfase-IT) and will continue to receive weekly IV Elaprase as part of standard of care.
After screening to see if patients are suitable, participants who have not received the study drug in the main study will undergo surgery to have a port implanted to allow for idursulfase-IT to be given monthly into the CSF.
Study procedures include, among other tests, physical exams, blood and urine tests, brain MRI, and questionnaires.
REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
15/YH/0017
Date of REC Opinion
5 Feb 2015
REC opinion
Further Information Favourable Opinion