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Leukodystrophy

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Clinical Trials

This information is provided by ClinicalTrials.gov

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Displaying 1-10 of 21 results.
Natural History, Diagnosis, and Outcomes for Leukodystrophies
Status: Recruiting
Last Changed: Jan 28, 2020
First Received: Aug 21, 2018
Disease(s): Leukodystrophy
Locations: Primary Children's Hospital, Salt Lake City, Utah, United States
Lentiviral Gene Therapy for MLD
Status: Recruiting
Last Changed: Sep 19, 2019
First Received: Oct 31, 2018
Disease(s): Metachromatic Leukodystrophy (MLD)
Intervention(s): Lentivirus-mediated delivery of ARSA to the CNS.
Locations: Lung-Ji Chang, Shenzhen, Guangdong, China
Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
Status: Recruiting
Last Changed: Sep 24, 2015
First Received: Sep 24, 2015
Disease(s): Metachromatic Leukodystrophy, Adrenoleukodystrophy
Intervention(s): transduced CD34+ hematopoietic stem cell
Locations: Shenzhen Second People's Hospital, The First Affiliated Hospital of Shenzhen University, Shenzhen, Guangdong, China
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
Status: Recruiting
Last Changed: Feb 27, 2020
First Received: Feb 25, 2020
Disease(s): Lysosomal Storage Diseases, Metachromatic Leukodystrophy
Intervention(s): OTL-200
Locations: Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET), Milan, Italy
A Study of Intrathecal SHP611 in Participants With Late Infantile Metachromatic Leukodystrophy
Status: Recruiting
Last Changed: Feb 13, 2020
First Received: Dec 11, 2018
Disease(s): Metachromatic Leukodystrophy (MLD)
Intervention(s): SHP611
Locations: Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, California, United States
Children's Hospital Colorado, Aurora, Colorado, United States
University of Miami Miller School of Medicine, Miami, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
University of Iowa Stead Family Children's Hospital, Iowa City, Iowa, United States
... and 20 other locations.
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Status: Recruiting
Last Changed: Dec 02, 2019
First Received: Mar 04, 2016
Disease(s): Leukodystrophy, White Matter Disease
Locations: The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)
Status: Recruiting
Last Changed: Feb 17, 2020
First Received: Jan 08, 2018
Disease(s): Lysosomal Storage Disease, Metachromatic Leukodystrophy
Intervention(s): OTL-200
Locations: Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET), Milan, Italy
Biomarker for Metachromatic Leukodystrophy (BioMeta) Disease
Status: Recruiting
Last Changed: Sep 17, 2019
First Received: Feb 22, 2012
Disease(s): Peripheral Neuropathy, Muscle Weakness
Locations: Children Hospital, Faculty of Medicine, Cairo University, Cairo, Egypt
Centogene AG, Rostock, Germany
Amrita Institute of Medical Sciences & Research Centre, Cochin, Kerala, India
Navi Mumbai Institute of Research In Mental And Neurological Handicap (NIRMAN), Mumbai, India
Childrens Hospital and Institute of Child Health, Ferozepur Road, Lahore, Pakistan
... and 1 other locations.
The Myelin Disorders Biorepository Project
Status: Recruiting
Last Changed: Apr 05, 2019
First Received: Feb 09, 2017
Disease(s): Leukodystrophy, White Matter Disease, Leukoencephalopathies
Locations: The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
Krabbe Disease Global Patient Registry
Status: Recruiting
Last Changed: Apr 05, 2019
First Received: Dec 15, 2016
Disease(s): Krabbe Disease
Locations: State University of New York at Buffalo, Buffalo, New York, United States