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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Ended earlyNCT01751802
What this trial is testing

Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease

Who this might be right for
Lesch-Nyhan DiseaseSelf-injurious Behavior
Emalex Biosciences Inc. 9
Large-scale testing (Phase 3)Study completedNCT00891202
What this trial is testing

Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

Who this might be right for
Gaucher Disease, Type 1
Genzyme, a Sanofi Company 40
Testing effectiveness (Phase 2)Ended earlyNCT03952156
What this trial is testing

Gene Therapy Clinical Study in Adult PKU

Who this might be right for
PhenylketonuriasPAH Deficiency
Homology Medicines, Inc 10
Testing effectiveness (Phase 2)Study completedNCT04513002
What this trial is testing

Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis

Who this might be right for
Ataxia Telangiectasia
The University of Queensland 30
Testing effectiveness (Phase 2)Ended earlyNCT01195753
What this trial is testing

Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders

Who this might be right for
Urea Cycle Disorders
Cytonet GmbH & Co. KG 10
Early research (Phase 1)Study completedNCT04560816
What this trial is testing

The Cardiac Effects of ALXN1840 in Healthy Adults

Who this might be right for
Healthy
Alexion Pharmaceuticals, Inc. 57
Post-approval studies (Phase 4)Active Not RecruitingNCT06780332
What this trial is testing

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

Who this might be right for
PhenylketonuriaPKU
BioMarin Pharmaceutical 9
Not applicableUnknownNCT04113772
What this trial is testing

Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine

Who this might be right for
Hereditary Tyrosinemia, Type I
Sutphin Drugs 4
Not applicableNot Yet RecruitingNCT06985212
What this trial is testing

National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children

Who this might be right for
Acid Sphingomyelinase Deficiency (ASMD)Niemann Pick Disease
Wladimir MAUHIN, Dr 200
Not applicableNo Longer AvailableNCT02233257
What this trial is testing

Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
University of Minnesota
Not applicableStudy completedNCT01198899
What this trial is testing

Belgian Screening Project for the Detection of Anderson-Fabry Disease in Hypertrophic Cardiomyopathy

Who this might be right for
Left Ventricular Hypertrophy
University Hospital, Ghent 540
Not applicableStudy completedNCT04375592
What this trial is testing

Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of Phenylketonuria

Who this might be right for
PhenylketonuriasHyperphenylalaninaemiaTetrahydrobiopterin Deficiency
metaX Institut fuer Diatetik GmbH 10
Not applicableStudy completedNCT07532564
What this trial is testing

Risk Factors, Costs, and Impacts of ED Boarding

Who this might be right for
Stroke (CVA) or TIADiabetesAsthma (Diagnosis)+9 more
University of Maryland, Baltimore 30,486
Not applicableStudy completedNCT02285348
What this trial is testing

Oxidative Stress, Low Grade Inflammation, Tissue Breakdown and Biomarkers in Cerebrospinal Fluid of A-T

Who this might be right for
Ataxia Telangiectasia
Johann Wolfgang Goethe University Hospital 18
Early research (Phase 1)Study completedNCT01586455
What this trial is testing

Human Placental-Derived Stem Cell Transplantation

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIAdrenoleukodystrophy+13 more
New York Medical College 43
Early research (Phase 1)Ended earlyNCT01345578
What this trial is testing

Hepatocyte Transplantation for Liver Based Metabolic Disorders

Who this might be right for
Metabolic Diseases
Ira Fox 5
Not applicableStudy completedNCT03678324
What this trial is testing

Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease

Who this might be right for
Fabry Disease
University of Utah 40
Testing effectiveness (Phase 2)Ended earlyNCT00837824
What this trial is testing

Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

Who this might be right for
Fabry DiseaseChronic Kidney Disease, Stage IV (Severe)
Genzyme, a Sanofi Company 20
Testing effectiveness (Phase 2)Looking for participantsNCT07123155
What this trial is testing

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 45
Not applicableEnrolling By InvitationNCT06627543
What this trial is testing

Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3

Who this might be right for
Gaucher Disease Type 3
Noha Osman 44
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