Extended trial tests potential treatment for rare childhood brain disease
NCT ID NCT01887938
Summary
This study continues testing an experimental treatment called HGT-1110 in children with metachromatic leukodystrophy (MLD), a rare and serious genetic brain disease. It follows 24 children who participated in an earlier trial, giving them regular spinal injections of the treatment to monitor long-term safety and see if it helps maintain motor skills and daily functioning. The main goal is to understand the treatment's safety profile over an extended period while also checking if it provides any benefit.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for METACHROMATIC LEUKODYSTROPHY (MLD) are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
CHR Orleans - Hopital La Source
Orléans, 45067, France
-
CHU de Nantes
Nantes, 44093, France
-
Center for Pediatric Clinical Studies (CPCS)
Tübingen, Baden-Wurttemberg, 72076, Germany
-
Detska Interni Klinika, Lf Mu A Fn Brno
Brno, 61300, Czechia
-
Hopital Femme Mere Enfant
Bron, 69 677, France
-
Hopital Gui de Chauliac - CHRU de Montpellier
Montpellier, 34000, France
-
Hospital de Clinicas de Porto Alegre
Porto Alegre, 90035-003, Brazil
-
Hôpital de Bicêtre
Le Kremlin-Bicêtre, Île-de-France Region, 94275, France
-
Kitakyushu Municipal Yahata Hospital
Fukuoka, 805-8534, Japan
-
Klinikum Oldenburg
Oldenburg, 26133, Germany
-
Kurashiki Central Hospital
Okayama Prefecture, 710-8602, Japan
-
Marien-Hospital Wesel gGmbh
Wesel, 46483, Germany
-
Osaka University Hospital
Osaka, 565-0871, Japan
-
Rigshospitalet
Copenhagen, 2100, Denmark
-
The Children's Hospital at Westmead
Westmead, 2145, Australia
Conditions
Explore the condition pages connected to this study.