167 studies found for:    Open Studies | "Brain Diseases, Metabolic, Inborn"
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Open Studies | "Brain Diseases, Metabolic, Inborn" (167 records)
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Rank Status Study
1 Recruiting Natural History Study of Children With Metachromatic Leukodystrophy
Conditions: Leukodystrophy, Metachromatic;   Hereditary Central Nervous System Demyelinating Diseases;   Brain Diseases, Metabolic, Inborn;   Brain Diseases, Metabolic;   Brain Diseases;   Central Nervous System Diseases;   Nervous System Diseases;   Sulfatidosis;   Sphingolipidoses;   Lysosomal Storage Diseases, Nervous System;   Leukoencephalopathies;   Demyelinating Diseases;   Metabolism, Inborn Errors;   Genetic Diseases, Inborn;   Lipidoses;   Lipid Metabolism, Inborn Errors;   Lysosomal Storage Diseases;   Metabolic Diseases;   Lipid Metabolism Disorders
Intervention: Other: Natural History Study of Children With Metachromatic Leukodystrophy
2 Recruiting Biomarker for Metachromatic Leukodystrophy Disease
Conditions: Leukodystrophy, Metachromatic;   Hereditary Central Nervous System Demyelinating Diseases;   Brain Diseases, Metabolic, Inborn;   Sphingolipidoses;   Lysosomal Storage Diseases, Nervous System
Intervention:
3 Recruiting Longitudinal Study of Urea Cycle Disorders
Conditions: Brain Diseases, Metabolic, Inborn;   Amino Acid Metabolism, Inborn Errors;   Urea Cycle Disorders
Intervention:
4 Recruiting UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Conditions: 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
Intervention: Biological: DUOC-01
5 Recruiting Biomarker for Krabbe Disease
Conditions: Lysosomal Storage Diseases;   Krabbe Disease
Intervention:
6 Recruiting Home Therapy With VPRIV in Gaucher's Disease
Condition: Gaucher's Disease
Intervention:
7 Not yet recruiting Betaine METABOLISM OF PATIENTS With Homocystinuria
Condition: Homocystinuria
Intervention: Drug: Betaine
8 Recruiting Phenylbutyrate Therapy for Maple Syrup Urine Disease
Condition: Maple Syrup Urine Disease
Interventions: Drug: Phenylbutyrate;   Drug: Placebo powder
9 Recruiting Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease
Condition: Fabry's Disease
Intervention: Drug: GZ/SAR402671
10 Recruiting Genes Involved in Lipid Disorders
Condition: Dyslipidemia
Intervention:
11 Recruiting Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
Condition: Niemann-Pick Disease, Type C1
Intervention: Drug: 2-hydroxypropyl-beta-cyclodextrin
12 Recruiting Investigating Lysosomal Storage Diseases in Minority Groups
Conditions: Lysosomal Storage Disorders;   Gaucher Disease;   Fabry Disease;   Pompe Disease;   Niemann-Pick Disease
Intervention:
13 Unknown  Myocardial Affectation in Patients With Fabry Disease Without Phenotypic Manifestation. Diagnostic Value of Biomarkers
Conditions: Fabry Disease, Cardiac Variant;   Right Ventricular Hypertrophy
Intervention:
14 Unknown  Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease
Condition: Fabry Disease
Intervention: Drug: recombinant alpha-galactosidase A
15 Recruiting Protein Sorbent Properties of Montmorillonite in Vitro and in Vivo Models
Conditions: Urea Cycle Disorders, Inborn;   Other Metabolic Diseases
Interventions: Dietary Supplement: montmorillonite 5 g;   Dietary Supplement: montmorillonite 3 g;   Dietary Supplement: Montmorillonite 1 g
16 Not yet recruiting A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
Condition: Niemann-Pick Disease, Type C
Intervention:
17 Unknown  The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease
Condition: Gaucher Disease
Interventions: Drug: Cerezyme®;   Drug: ISU302
18 Unknown  Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
Conditions: Sickle Cell Disease;   Thalassemia;   Anemia;   Granuloma;   Wiskott-Aldrich Syndrome;   Chediak Higashi Syndrome;   Osteopetrosis;   Neutropenia;   Thrombocytopenia;   Hurler Disease;   Niemann-Pick Disease;   Fucosidosis
Intervention: Procedure: Hematopoietic stem cell transplantation
19 Recruiting Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
Condition: Urea Cycle Disorder
Intervention:
20 Recruiting An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
Condition: Phenylketonuria
Intervention: Drug: BMN 165

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