Intrathecal Enzyme Replacement therapy for MPSII

• Research type

  Research Study

• Full title

  A Phase I/II, Randomized, Safety and Ascending Dose Ranging Study of Intrathecal Idursulfase-IT Administered in Conjunction with Intravenous Elaprase® in Pediatric Patients with Hunter Syndrome and Cognitive Impairment

• IRAS ID

  53547

• Contact name

  Chris Hendriksz

• Eudract number

  2010-020048-36

• Clinicaltrials.gov Identifier

  NCT00920647

• Research summary

  This is a randomized, multicenter, multiple-dose, time-lagged, dose escalation study designed to evaluate the safety, tolerability and clinical activity of up to 3 dose levels of idursulfase-IT administered via an IDDD ( intra thecal drug delivery device) over 6 months in conjunction with weekly Elaprase 0.5 mg/kg IV in patients with Hunter syndrome who have cognitive impairment.

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  10/H1008/128

• Date of REC Opinion

  31 Jan 2011

• REC opinion

  Further Information Favourable Opinion