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
Status:
Completed
Phase of Trial:
Phase II
Latest Information Update: 08 Nov 2023
Price :
$35
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At a glance
- Drugs SHP 610 (Primary)
- Indications Mucopolysaccharidosis III
- Focus Therapeutic Use
- Sponsors Shire
- 08 Nov 2023 This trial has been discontinued in France (Date of the global end of the trial: 01-Jun-2023), according to the European Clinical Trials Database record.
- 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.