A Phase I/II Safety, Tolerability, Ascending Dose and Dose Frequency Study of Recombinant Human Heparan N-Sulfatase (rhHNS) Intrathecal Administration Via an Intrathecal Drug Delivery Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Trial Profile

A Phase I/II Safety, Tolerability, Ascending Dose and Dose Frequency Study of Recombinant Human Heparan N-Sulfatase (rhHNS) Intrathecal Administration Via an Intrathecal Drug Delivery Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Completed
Phase of Trial: Phase I/II

Latest Information Update: 26 Jul 2016

At a glance

  • Drugs SHP 610 (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Adverse reactions; First in man
  • Sponsors Shire
  • Most Recent Events

    • 25 Jun 2014 New trial record
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