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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT07301216
What this trial is testing

Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study

Who this might be right for
Wilson Disease
Yale University 30
Large-scale testing (Phase 3)Study completedNCT02770625
What this trial is testing

Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1
ISU Abxis Co., Ltd. 8
Not applicableStudy completedNCT01389947
What this trial is testing

Microcirculation During Extracorporeal Circulation

Who this might be right for
Mesenteric Ischemia
Centre Hospitalier Universitaire de Besancon 66
Testing effectiveness (Phase 2)Looking for participantsNCT06687733
What this trial is testing

Safety and Efficacy Study of NGGT002 in Adult Patients With Phenylketonuria

Who this might be right for
Phenylketonurias
NGGT (Suzhou) Biotechnology Co., Ltd. 18
Post-approval studies (Phase 4)Active Not RecruitingNCT05164055
What this trial is testing

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 17
Large-scale testing (Phase 3)Study completedNCT00272792
What this trial is testing

Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

Who this might be right for
Phenylketonurias
BioMarin Pharmaceutical 45
Not applicableStudy completedNCT00688844
What this trial is testing

Nutritional and Neurotransmitter Changes in PKU Subjects on BH4

Who this might be right for
Phenylketonuria
Emory University 58
Testing effectiveness (Phase 2)Looking for participantsNCT07215416
What this trial is testing

Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)

Who this might be right for
Ataxia Telangiectasia
Timothy Yu 10
Not applicableStudy completedNCT01431326
What this trial is testing

Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care

Who this might be right for
AdenovirusAnesthesiaAnxiety+56 more
Daniel Benjamin 3,520
Not applicableUnknownNCT01506258
What this trial is testing

Autologous Stem Cells in Newborns With Oxygen Deprivation

Who this might be right for
Apgar; 0-3 at 1 MinuteMetabolic AcidosisHypoxia, Brain+1 more
Hospital Universitario Dr. Jose E. Gonzalez 20
Not applicableLooking for participantsNCT03137355
What this trial is testing

The International Registry for Leigh Syndrome

Who this might be right for
Leigh SyndromeLeigh DiseaseLeigh's Necrotizing Encephalopathy+2 more
The University of Texas Health Science Center, Houston 200
Large-scale testing (Phase 3)Study completedNCT00176904
What this trial is testing

Stem Cell Transplant for Inborn Errors of Metabolism

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more
Masonic Cancer Center, University of Minnesota 135
Not applicableLooking for participantsNCT05659901
What this trial is testing

Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

Who this might be right for
Pelizaeus-Merzbacher Disease
Ionis Pharmaceuticals, Inc. 32
Not applicableLooking for participantsNCT07130565
What this trial is testing

Pan-Amyloid PET/CT in Various Amyloid-Related Disease

Who this might be right for
AmyloidCardiac AmyloidosisAmyloidosis
Tianjin Medical University 500
Testing effectiveness (Phase 2)Study completedNCT06644534
What this trial is testing

Assess TTI-0102 vs Placebo in MELAS Patients

Who this might be right for
MELAS SyndromeMitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
Thiogenesis Therapeutics, Inc. 9
Not applicableStudy completedNCT02255981
What this trial is testing

Efficacy of Acupuncture in Macular Diseases

Who this might be right for
Age-related Macular DegenerationStargardt DiseaseMacular Dystrophy
Escuela Neijing 33
Not applicableStudy completedNCT00728364
What this trial is testing

Anderson-Fabry Disease in Chronic Kidney Disease Patients Not on Renal Replacement Therapy

Who this might be right for
Focus of Study: Prevalence of Fabry Disease in CKD Population
Klinikum Wels-Grieskirchen 4,353
Post-approval studies (Phase 4)Study completedNCT03910621
What this trial is testing

Safety and Efficacy of Miglustat in Chinese NPC Patients

Who this might be right for
Niemann-Pick Disease, Type C
Actelion 17
Large-scale testing (Phase 3)Ended earlyNCT05676853
What this trial is testing

Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency

Who this might be right for
Arginase I DeficiencyHyperargininemia
Aeglea Biotherapeutics 3
Testing effectiveness (Phase 2)Study completedNCT01962363
What this trial is testing

EPI-743 in Friedreich's Ataxia Point Mutations

Who this might be right for
Friedreich's Ataxia
University of South Florida 4
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