HGT-1410 in Pediatric Patients with Early Stage MPS IIIA

• Research type

  Research Study

• Full title

  A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Early Stage Mucopolysaccharidosis Type III A Disease

• IRAS ID

  137186

• Contact name

  Maureen Cleary

• Contact email

  Maureen.Cleary@gosh.nhs.uk

• Sponsor organisation

  Shire Human Genetic Therapies, Inc.

• Eudract number

  2013-003450-24

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  13/LO/1694

• Date of REC Opinion

  23 Dec 2013

• REC opinion

  Further Information Favourable Opinion