Phase 1/2 Study of UX003 in Patients with MPS 7

• Research type

  Research Study

• Full title

  An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7

• IRAS ID

  132352

• Contact name

  Simon Jones

• Contact email

  Simon.Jones@cmft.nhs.uk

• Eudract number

  2013-001152-35

• Clinicaltrials.gov Identifier

  NCT01359319

• Research summary

  UX003-CL201 is an open-label Phase 1/2 study to assess the safety, efficacy, and dose of UX003 in MPS 7 subjects via IV administration every other week (QOW) for 36 weeks with up to an additional 36 weeks from the optional continuation period. Up to 5 subjects, who are between 5 and 30 years of age inclusive, will be enrolled and treated with UX003.

  The initial 12-week treatment period will be followed by a 24-week forced dose titration period to assess the optimal dose.

• REC name

  Yorkshire & The Humber - Leeds East Research Ethics Committee

• REC reference

  13/YH/0188

• Date of REC Opinion

  25 Jul 2013

• REC opinion

  Further Information Favourable Opinion