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Metachromatic leukodystrophy

MLDArylsulfatase A DeficiencyARSA deficiencyGreenfield's disease
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Clinical Trials

This information is provided by ClinicalTrials.gov

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Displaying 1-9 of 9 results.
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: Oct 06, 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
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
The Natural History of Metachromatic Leukodystrophy Study (HOME Study)
Status: Recruiting
Last Changed: Nov 13, 2020
First Received: Nov 13, 2020
Disease(s): Metachromatic Leukodystrophy
Locations: National Organization for Rare Disorders, Danbury, Connecticut, United States
A Study of Intrathecal SHP611 in Participants With Late Infantile Metachromatic Leukodystrophy
Status: Recruiting
Last Changed: Oct 22, 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
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
Mayo Clinic - PPDS, Rochester, Minnesota, United States
New York University Langone Medical Center, New York, New York, United States
... and 17 other locations.
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Status: Recruiting
Last Changed: Sep 30, 2020
First Received: Jun 23, 2014
Disease(s): Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Intervention(s): Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Locations: Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Status: Recruiting
Last Changed: Dec 03, 2019
First Received: Oct 02, 2014
Disease(s): Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Intervention(s): DUOC-01
Locations: Duke University Medical Center, Durham, North Carolina, United States
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Status: Recruiting
Last Changed: Oct 08, 2020
First Received: Aug 27, 2020
Disease(s): Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Intervention(s): data collection
Locations: UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Status: Recruiting
Last Changed: Jun 04, 2020
First Received: Oct 14, 2013
Disease(s): Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Intervention(s): Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Locations: UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States