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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableStudy completedNCT04577170
What this trial is testing

Pulsatility Index, Vasomotor Reactivity and Leukoencephalopathy in Fabry Patients

Who this might be right for
Fabry Disease
National and Kapodistrian University of Athens 45
Large-scale testing (Phase 3)Ended earlyNCT05764239
What this trial is testing

Efficacy and Safety of SYNB1934 in Patients With PKU (SYNPHENY-3)

Who this might be right for
Phenylketonuria
Synlogic 35
Not applicableUnknownNCT05914727
What this trial is testing

Biomarkers Of Lung InVolvement In ASMD

Who this might be right for
Asmd, Visceral Type
Eline C. B. Eskes 68
Testing effectiveness (Phase 2)Study completedNCT00897221
What this trial is testing

A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia

Who this might be right for
Friedreich's Ataxia
ApoPharma 36
Early research (Phase 1)Study completedNCT00151216
What this trial is testing

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

Who this might be right for
Batten DiseaseLate Infantile Neuronal Ceroid Lipofuscinosis
Weill Medical College of Cornell University 10
Not applicableWithdrawnNCT04055532
What this trial is testing

Biomarkers in Neurodegenerative Diseases

Who this might be right for
Mild Cognitive Impairment (MCI)Alzheimer Disease (AD)Dementia With Lewy Bodies (DLB)+8 more
University of Minnesota
Not applicableLooking for participantsNCT05848271
What this trial is testing

Natural History Study of Patients with HPDL Mutations

Who this might be right for
Mitochondrial EncephalomyopathiesHereditary Spastic ParaplegiaSpastic Paraplegia+4 more
University of California, San Diego 50
Not applicableLooking for participantsNCT07728032
What this trial is testing

Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU

Who this might be right for
Phenylketonuria
Birmingham Women's and Children's NHS Foundation Trust 148
Not applicableStudy completedNCT04943393
What this trial is testing

Remote Neurocognitive and Psychological Assessment in PKU

Who this might be right for
Phenylketonurias
University of Missouri-Columbia 84
Not applicableStudy completedNCT00001972
What this trial is testing

PET Scan of Brain Metabolism in Relation to Age and Disease

Who this might be right for
Alzheimer's DiseaseBrain NeoplasmNiemann Pick Disease
National Institute of Neurological Disorders and Stroke (NINDS) 123
Not applicableUnknownNCT06324877
What this trial is testing

Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With Nicotinamide Riboside

Who this might be right for
Ataxia Telangiectasia
The University of Queensland 10
Not applicableUnknownNCT04991701
What this trial is testing

A National Retrospective Population Based Cohort Study of the Natural History of Ataxia Telangiectasia

Who this might be right for
Ataxia Telangiectasia
University of Nottingham 178
Not applicableNot Yet RecruitingNCT04252001
What this trial is testing

Growing up With the Young Endocrine Support System (YESS!)

Who this might be right for
Congenital Adrenal HyperplasiaHypogonadotropic HypogonadismGrowth Hormone Deficiency+6 more
dr. Laura C. G. de Graaff-Herder 160
Not applicableStudy completedNCT03620071
What this trial is testing

GoalKeeper: Intelligent Information Sharing for Children With Medical Complexity

Who this might be right for
Childhood CancerCerebral PalsyChronic Lung Disease+3 more
Stanford University 67
Not applicableUnknownNCT02859363
What this trial is testing

Fabry Disease in Cerebrovascular Disease

Who this might be right for
Fabry Disease
Chang Gung Memorial Hospital 700
Not applicableStudy completedNCT05330039
What this trial is testing

Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)

Who this might be right for
Inborn Errors of MetabolismPropionic AciduriaMethylmalonic Aciduria+1 more
Emma Marie Caroline Slack 22
Early research (Phase 1)Looking for participantsNCT02254863
What this trial is testing

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Who this might be right for
AdrenoleukodystrophyBatten DiseaseMucopolysaccharidosis II+9 more
Joanne Kurtzberg, MD 40
Testing effectiveness (Phase 2)Study completedNCT03519711
What this trial is testing

PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia

Who this might be right for
BH4 DeficiencyHyperphenylalaninemia
PTC Therapeutics 8
Large-scale testing (Phase 3)Study completedNCT01842841
What this trial is testing

Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Shire 5
Not applicableUnknownNCT06065605
What this trial is testing

Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

Who this might be right for
Fabry Disease
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 40
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