182 studies found for:    Open Studies | "Brain Diseases, Metabolic, Inborn"
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Rank Status Study
1 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
2 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
3 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
4 Recruiting Longitudinal Study of Urea Cycle Disorders
Conditions: Brain Diseases, Metabolic, Inborn;   Amino Acid Metabolism, Inborn Errors;   Urea Cycle Disorders
5 Recruiting Biomarker for Krabbe Disease
Conditions: Lysosomal Storage Diseases;   Krabbe Disease
6 Not yet recruiting Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
Condition: Pyruvate Dehydrogenase Complex Deficiency
Interventions: Drug: Dichloroacetate (DCA);   Other: Placebo;   Genetic: Genotype
7 Not yet recruiting Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C
Condition: Niemann-Pick Disease, Type C
Interventions: Drug: arimoclomol;   Drug: Placebo
8 Recruiting SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease
Condition: Gaucher Disease
9 Recruiting Compare Pharmacokinetic(PK) Profiles of XZK vs Lovastatin in Healthy Male Volunteers
Condition: Lipid Metabolism Disorder
Interventions: Drug: XueZhiKang;   Drug: Lovastatin
10 Not yet recruiting A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Dietary Supplement: vitamin D3
11 Recruiting Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)
Condition: Gaucher Disease Type 1
Intervention: Drug: N-acetylcysteine
12 Not yet recruiting This Study is to Evaluate the Effect of Velaglucerase Alfa (VPRIV®) on Patients With Type 1 Gaucher Disease Through the IV Administration of VPRIV® Over 2 Years
Condition: Type 1 Gaucher Disease
Intervention: Drug: Velaglucerase alfa
13 Recruiting Metabolic Complications Following Knee Injury in Young and Middle Aged Patients
Conditions: Glucose Metabolism Disorders;   Lipid Metabolism Disorders;   Sedentary Lifestyle
Interventions: Biological: Blood and Urine sampling;   Radiation: DXA scan;   Biological: OGTT
14 Recruiting Evaluating the Potential of the Diet as Disease Modifier in Amyotrophic Lateral Sclerosis (JERN_ALS)
Conditions: Amyotrophic Lateral Sclerosis;   Dietary Habits;   Disorder of Fatty Acid Metabolism
15 Recruiting Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
Conditions: Metachromatic Leukodystrophy;   Adrenoleukodystrophy
Intervention: Genetic: transduced CD34+ hematopoietic stem cell
16 Not yet recruiting Simplified Diet Approach in Phenylketonuria
Condition: Phenylketonuria
Intervention: Behavioral: Dietary Protein Counting and Free Fruits and Vegetables
17 Recruiting Flank Study With Crown Cooling Insert
Condition: Body Fat Disorder
Intervention: Device: The ZELTIQ System
18 Not yet recruiting WILSTIM - DBS (WILson STIMulation - Deep Brain Stimulation)
Conditions: Severe Dystonia;   Wilson's Disease
Interventions: Device: Medtronic, Activa® PC "on";   Device: Medtronic, Activa® PC "off"
19 Recruiting A Dose-escalating Clinical Trial With KH176
Conditions: MELAS;   LHON;   Leigh Syndrome;   Mitochondrial Disease;   Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
Interventions: Drug: KH176;   Drug: placebo
20 Recruiting A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
Condition: Gaucher Disease
Intervention: Drug: eliglustat

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Indicates status has not been verified in more than two years