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An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy
Sponsor: Shire
Summary
The purpose of this study is to collect long-term safety data in participants with metachromatic leukodystrophy (MLD) who are receiving HGT-1110 and have participated in Study HGT-MLD-070 (NCT01510028) through Week 40.
Official title: An Open-Label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients With Metachromatic Leukodystrophy
Key Details
Gender
All
Age Range
0 Years - 13 Years
Study Type
INTERVENTIONAL
Enrollment
24
Start Date
2013-05-23
Completion Date
2026-03-31
Last Updated
2026-01-09
Healthy Volunteers
No
Conditions
Interventions
HGT-1110
Participants will receive IT injection of HGT-1110.
Locations (15)
The Children's Hospital at Westmead
Westmead, Australia
Hospital de Clinicas de Porto Alegre
Porto Alegre, Brazil
Detska Interni Klinika, Lf Mu A Fn Brno
Brno, Czechia
Rigshospitalet
Copenhagen, Denmark
Hopital Femme Mere Enfant
Bron, France
Hopital Gui de Chauliac - CHRU de Montpellier
Montpellier, France
CHU de Nantes
Nantes, France
CHR Orleans - Hopital La Source
Orléans, France
Hôpital de Bicêtre
Le Kremlin-Bicêtre, Île-de-France Region, France
Center for Pediatric Clinical Studies (CPCS)
Tübingen, Baden-Wurttemberg, Germany
Klinikum Oldenburg
Oldenburg, Germany
Marien-Hospital Wesel gGmbh
Wesel, Germany
Kitakyushu Municipal Yahata Hospital
Fukuoka, Japan
Kurashiki Central Hospital
Okayama Prefecture, Japan
Osaka University Hospital
Osaka, Japan