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

Trial Profile

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

Completed
Phase of Trial: Phase II

Latest Information Update: 19 May 2017

At a glance

  • Drugs SHP 610 (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Therapeutic Use
  • Sponsors Shire
  • Most Recent Events

    • 01 Nov 2016 Primary endpoint has not been met. (A maximum decline in DQ (Developmental Quotient) of 10 points over 48 weeks, assessed by neurocognitive testing), as per Shire media release.
    • 15 Jun 2016 Status changed from active, no longer recruiting to completed.
    • 18 Apr 2016 Planned End Date changed from 1 Mar 2016 to 1 May 2016.
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