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Demyelinating Diseases

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Clinical Trials

This information is provided by ClinicalTrials.gov

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Displaying 21-30 of 541 results.
Evaluation of Outcome Metrics in Alexander Disease
Status: Recruiting
Last Changed: Jul 29, 2020
First Received: Mar 21, 2016
Disease(s): Alexander Disease
Locations: 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
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
Status: Recruiting
Last Changed: Mar 24, 2020
First Received: Dec 08, 2017
Disease(s): Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy, Inherited Metabolic Disorders
Intervention(s): Blood and Marrow Transplant
Locations: Masonic Caner Center at 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
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
Status: Recruiting
Last Changed: Aug 25, 2020
First Received: May 01, 2018
Disease(s): Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
Intervention(s): Thiotepa--single daily dose, Thiotepa--escalated dose
Locations: UF Health Shands Children's Hospital, Gainesville, Florida, United States
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
Status: Recruiting
Last Changed: Jul 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
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
Longitudinal Study of Neurodegenerative Disorders
Status: Recruiting
Last Changed: Nov 05, 2019
First Received: Nov 06, 2017
Disease(s): MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Intervention(s): Palliative Care, Hematopoetic Stem Cell Transplantation
Locations: The Program for the Study of Neurodevelopment in Rare Disorders, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States
Plasma Exchange With Albumin in AMN Patients
Status: Not yet recruiting
Last Changed: Mar 11, 2020
First Received: Mar 11, 2020
Disease(s): Adrenomyeloneuropathy, Adrenoleukodystrophy
Intervention(s): Albumin solution
Locations: Bellvitge University Hospital, L'Hospitalet de Llobregat, Barcelona, Spain
Precision Exercise in Children With Malignant Hemopathies
Status: Recruiting
Last Changed: Sep 30, 2019
First Received: Sep 16, 2019
Disease(s): Leukemia, Myeloid, Acute, Leukemia, B-cell, Leukemia, T Cell, Lymphoma, Hodgkin, Lymphoma, Non-Hodgkin, Drepanocytosis, Thalassemia Major, Adrenoleukodystrophy
Intervention(s): Sport Therapy
Locations: Maria Letizia Verga Center, Pediatric Clinic, University of Milan Bicocca, at the Foundation for the Mother and Her Child, Monza, MB, Italy