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
Phase of Trial: Phase II
Latest Information Update: 30 Sep 2017
At a glance
- Drugs SHP 610 (Primary)
- Indications Mucopolysaccharidosis III
- Focus Therapeutic Use
- Sponsors Shire
- 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.