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Clinical Trials

This information is provided by ClinicalTrials.gov

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Displaying 1-8 of 8 results.
Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis
Status: Not yet recruiting
Last Changed: Jul 24, 2019
First Received: Jul 24, 2019
Disease(s): Alpha-Mannosidosis
Intervention(s): Velmanase Alfa, Placebo
Biomarker for Mannosidosis Disease (BioMannosidosis)
Status: Recruiting
Last Changed: Sep 17, 2019
First Received: Aug 28, 2017
Disease(s): Alpha-Mannosidase B Deficiency, Lysosomal Alpha B Mannosidosis, Alpha-Mannosidase Deficiency
Locations: Children Hospital, Faculty of Medicine, Cairo University, Cairo, Egypt
Centogene AG, Rostock, Germany
Navi Mumbai Institute of Research In Mental And Neurological Handicap (NIRMAN), Mumbai, India
Childrens Hospital and Institute of Child Health, Ferozepur Road, Lahore, Pakistan
European Alpha-Mannosidosis Patient Epidemiological Program (EUMAP)
Status: Recruiting
Last Changed: Nov 20, 2019
First Received: Aug 29, 2018
Disease(s): Immunodeficiency, Skeletal Abnormalities, Deafness, Mental Retardation, Gingival Hypertrophy, Facial Dysmorphism
Locations: Charité - Universitätsmedizin Berlin, Leitung Sozialpädiatrisches Zentrum, Klinik für Pädiatrie m.S. Neurologie, Berlin, Germany
Carl-Thiem-Klinikum Cottbus GmbH, Sozialpädiatrisches Zentrum, Cottbus, Germany
Carl-Thiem-Klinikum Cottbus GmbH, Klinik für Kinder und Jugendmedizin, Cottbus, Germany
Universitätsklinikum Essen, Klinik für Hals-Nasen und Ohrenheilkunde, Essen, Germany
Universitätsklinikum Gießen, Zentrum für Kinderheilkunde und Jugendmedizin, Abteilung für Kinderneurologie, Sozialpädiatrie und Epileptologie, Gießen, Germany
... and 4 other locations.
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Status: Recruiting
Last Changed: Sep 26, 2019
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
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
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
Status: Recruiting
Last Changed: May 21, 2019
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
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Status: Recruiting
Last Changed: Oct 24, 2019
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: Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States