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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Very early researchLooking for participantsNCT07240896
What this trial is testing

A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)

Who this might be right for
Wilsons Disease
DSciLab Co., Ltd. 18
Testing effectiveness (Phase 2)Ended earlyNCT00681811
What this trial is testing

Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD

Who this might be right for
Late Infantile Metachromatic Leukodystrophy
Shire 11
Large-scale testing (Phase 3)Study completedNCT00074971
What this trial is testing

The Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 58
Testing effectiveness (Phase 2)Active Not RecruitingNCT06961344
What this trial is testing

An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)

Who this might be right for
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
Tisento Therapeutics 44
Not applicableStudy completedNCT04106544
What this trial is testing

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

Who this might be right for
Sphingomyelin Lipidosis
Sanofi 84
Not applicableStudy completedNCT00065299
What this trial is testing

Low Phenylalanine Diet for Mothers With Phenylketonuria (PKU)

Who this might be right for
Phenylketonuria
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 572
Not applicableAvailableNCT01881334
What this trial is testing

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

Who this might be right for
Hematologic MalignanciesInborn Errors of Metabolism DisordersImmune Deficiencies
Joanne Kurtzberg, MD
Not applicableWithdrawnNCT00639132
What this trial is testing

The Natural History of Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy
University of Pittsburgh
Not applicableUnknownNCT06072287
What this trial is testing

The Living With a Long-Term Condition Study

Who this might be right for
AsthmaAtrial FibrillationCancer+42 more
King's College London 600
Not applicableStudy completedNCT00983879
What this trial is testing

The Natural History of Infantile Globoid Cell Leukodystrophy

Who this might be right for
Infantile Globoid Cell Leukodystrophy
Zymenex A/S 6
Not applicableStudy completedNCT04602364
What this trial is testing

French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat

Who this might be right for
Fabry Disease
Amicus Therapeutics France SAS 48
Large-scale testing (Phase 3)Study completedNCT03852498
What this trial is testing

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Who this might be right for
Cerebral Adrenoleukodystrophy (CALD)
Genetix Biotherapeutics Inc. 35
Not applicableStudy completedNCT03168399
What this trial is testing

Evaluation of PKU Explore

Who this might be right for
PhenylketonuriaInborn Errors of Metabolism
Vitaflo International, Ltd 22
Not applicableNot Yet RecruitingNCT06664840
What this trial is testing

MyRareDiet A Novel Diet Tracking Tool

Who this might be right for
Urea Cycle DisorderPropionic AciduriaMaple Syrup Urine Disease+1 more
Oregon Health and Science University 60
Not applicableStudy completedNCT03097250
What this trial is testing

MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria

Who this might be right for
Phenylketonuria
Boston Children's Hospital 10
Not applicableNot Yet RecruitingNCT05470478
What this trial is testing

IBCI Optimization for Veterans With Paralysis

Who this might be right for
Spinal Cord InjuryAmyotrophic Lateral SclerosisBrain Stem Infarctions+2 more
VA Office of Research and Development 2
Not applicableStudy completedNCT00458991
What this trial is testing

RhGH Therapy on Hepatic Drug Metabolism

Who this might be right for
Growth Hormone Deficiency, Dwarfism
University of Louisville 9
Not applicableLooking for participantsNCT03334292
What this trial is testing

Natural History of Wilson Disease

Who this might be right for
Wilson Disease
Yale University 300
Early research (Phase 1)Study completedNCT03888716
What this trial is testing

A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease

Who this might be right for
Mitochondrial DiseasesMitochondrial Respiratory Chain DeficienciesMELAS Syndrome+1 more
Abliva AB 72
Not applicableLooking for participantsNCT06337864
What this trial is testing

Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria

Who this might be right for
Brain DiseasesBrain Diseases, MetabolicBrain Diseases, Metabolic, Inborn+5 more
Rigshospitalet, Denmark 30
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