Trial Profile
A Single Center, Open-Label, Non-Randomized, Uncontrolled, Multiple-Dose, Dose Escalation Study of the Safety, Pharmacokinetics, Efficacy and Long Term Safety of HGT-1111 (Recombinant Human Arylsulfatase A [rhASA, Metazym]) for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Status:
Completed
Phase of Trial:
Phase I/II
Latest Information Update: 15 Jun 2022
Price :
$35
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At a glance
- Drugs Cerebroside-sulfatase (Primary)
- Indications Metachromatic leukodystrophy
- Focus Therapeutic Use
- Sponsors Shire
- 26 Apr 2022 Using data from 3 studies (NCT00418561; NCT00633139; NCT00681811) were used to evaluate the toxic effects of sulfatide and lysosulfatide on the central (CNS) and peripheral (PNS) nervous systems in children with metachromatic leukodystrophy (MLD) and to explore the impact of treatment with intravenous recombinant human arylsulfatase A (rhASA) on myelin turnover, presented at the 74th Annual Meeting of the American Academy of Neurology 2022.
- 07 May 2014 New trial record