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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Not applicableActive Not RecruitingNCT06573866
What this trial is testing

Enhancement of Quality of Work And Life

Who this might be right for
Parkinson DiseaseHereditary Spastic ParaparesisCerebellar Ataxia+2 more
Radboud University Medical Center 124
Not applicableLooking for participantsNCT00196742
What this trial is testing

Fabry Disease Registry & Pregnancy Sub-registry

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 9,000
Not applicableActive Not RecruitingNCT07506083
What this trial is testing

Natural History in Fabry Disease With IVS4+919G>A Mutations

Who this might be right for
Fabry Disease
Chinese University of Hong Kong 31
Not applicableStudy completedNCT02418546
What this trial is testing

Electronic-health Application To Measure Outcomes REmotely Clinical Trial

Who this might be right for
Neurodegenerative DiseaseWeight LossAmyotrophic Lateral Sclerosis+3 more
Massachusetts General Hospital 78
Large-scale testing (Phase 3)Looking for participantsNCT05163288
What this trial is testing

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Who this might be right for
Niemann-Pick Disease, Type C
IntraBio Inc 53
Testing effectiveness (Phase 2)Study completedNCT03197662
What this trial is testing

Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome

Who this might be right for
Prader-Willi SyndromeHyperphagia
Eric Hollander 32
Not applicableNo Longer AvailableNCT05317780
What this trial is testing

Canavan-Single Patient IND

Who this might be right for
Canavan Disease
University of Florida
Not applicableStudy completedNCT01451879
What this trial is testing

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

Who this might be right for
Pompe Disease
University of Florida 11
Not applicableLooking for participantsNCT04388969
What this trial is testing

World Data on Ambroxol for Patients With GD and GBA Related PD

Who this might be right for
Gaucher DiseaseParkinson DiseaseGBA Gene Mutation
Shaare Zedek Medical Center 300
Not applicableStudy completedNCT00077662
What this trial is testing

A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Genzyme, a Sanofi Company 61
Large-scale testing (Phase 3)Ended earlyNCT04958642
What this trial is testing

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

Who this might be right for
Niemann-Pick Disease, Type C
Mandos LLC 66
Not applicableLooking for participantsNCT07648303
What this trial is testing

Prevalence of Cardiac Thrombi in Cardiac Amyloidosis

Who this might be right for
Cardiac Amyloidosis
Centre Hospitalier Universitaire Dijon 200
Not applicableStudy completedNCT01080859
What this trial is testing

Vessel Wall Response of the Bio-Active-stent and Everolimus-Eluting Stent Assessed By Optical Coherence Tomography

Who this might be right for
Acute Coronary Syndrome
The Hospital District of Satakunta 40
Not applicableEnded earlyNCT00004442
What this trial is testing

Study of Bile Acids in Patients With Peroxisomal Disorders

Who this might be right for
Infantile Refsum's DiseaseZellweger SyndromeBifunctional Enzyme Deficiency+1 more
University of Cincinnati 25
Not applicableStudy completedNCT00758108
What this trial is testing

Characterization of WAGR Syndrome and Other Chromosome 11 Gene Deletions

Who this might be right for
WAGR SyndromeWilm's TumorAniridia+2 more
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 197
Not applicableStudy completedNCT05242913
What this trial is testing

Effects of Resistant Potato Starch on the Gut Microbiota

Who this might be right for
Microbiota
MSP Starch Products Inc. 75
Testing effectiveness (Phase 2)Study completedNCT01838655
What this trial is testing

Nitisinone for Type 1B Oculocutaneous Albinism

Who this might be right for
AlbinismVision Loss
National Eye Institute (NEI) 5
Not applicableStudy completedNCT04268147
What this trial is testing

Instrumented Data Exchange for Ataxia Study

Who this might be right for
Spinocerebellar Ataxia Type 1Spinocerebellar Ataxia Type 2Spinocerebellar Ataxia Type 3+2 more
University of Chicago 128
Not applicableStudy completedNCT06484478
What this trial is testing

A Real-world Wearables Study in Fabry Disease.

Who this might be right for
Fabry Disease
Royal Free Hospital NHS Foundation Trust 100
Large-scale testing (Phase 3)Study completedNCT00811785
What this trial is testing

Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency

Who this might be right for
Menkes DiseaseOccipital Horn SyndromeUnexplained Copper Deficiency
Cyprium Therapeutics, Inc. 93
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