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ACTIVE NOT RECRUITING
NCT01887938
PHASE1/PHASE2

An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy

Sponsor: Shire

View on ClinicalTrials.gov

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

Interventions

BIOLOGICAL

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