Intrathecal Enzyme Replacement Therapy for MPS II Extension Study

• Research type

  Research Study

• Full title

  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

• IRAS ID

  72798

• Contact name

  Suresh Vijay

• Contact email

  suresh.vijay@bch.nhs.uk

• Sponsor organisation

  Shire Human Genetic Therapies Incorporated

• Eudract number

  2011-000212-25

• Clinicaltrials.gov Identifier

  100,610, IND

• Duration of Study in the UK

  3 years, 0 months, 1 days

• Research summary

  This is an open-label extension of Study HGT-HIT-045 designed to evaluate the long-term safety and clinical outcomes of monthly intrathecal (IT) injection of idursulfase-IT via an IDDD in conjunction with weekly intravenous (IV) infusion of Elaprase® in patients with Hunter syndrome and cognitive impairment.

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  11/NW/0773

• Date of REC Opinion

  5 Jan 2012

• REC opinion

  Further Information Favourable Opinion