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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableUnknownNCT05692622
What this trial is testing

Home-based Complex Intervention for Children With Ataxia Telangiectasia

Who this might be right for
Ataxia Telangiectasia in ChildrenAtaxia Telangiectasia
University of Plymouth 40
Not applicableUnknownNCT06186167
What this trial is testing

Amyloidosis Incidence in High-Risk Cardiac Device Patients

Who this might be right for
AmyloidCardiac AmyloidosisAmyloidosis Cardiac+4 more
Midwest Heart & Vascular Specialists 100
Post-approval studies (Phase 4)UnknownNCT00487630
What this trial is testing

Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease

Who this might be right for
Fabry Disease
Assistance Publique - Hôpitaux de Paris 34
Testing effectiveness (Phase 2)Study completedNCT00925054
What this trial is testing

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 40
Not applicableStudy completedNCT00071903
What this trial is testing

The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study

Who this might be right for
Pseudotumor CerebriCystinosis
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 18
Not applicableStudy completedNCT01472874
What this trial is testing

Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease

Who this might be right for
Wilson Disease
Yale University 8
Large-scale testing (Phase 3)Active Not RecruitingNCT04910776
What this trial is testing

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

Who this might be right for
Glycogen Storage Disease Type II
Sanofi 17
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Not applicableUnknownNCT02761421
What this trial is testing

Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT

Who this might be right for
Glycogen Storage Disease Type II
Taipei Veterans General Hospital, Taiwan 15
Testing effectiveness (Phase 2)Active Not RecruitingNCT05739643
What this trial is testing

Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT

Who this might be right for
Krabbe Disease
Forge Biologics, Inc 9
Not applicableStudy completedNCT00728676
What this trial is testing

Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors

Who this might be right for
Phenylketonuria
Shoji Yano 10
Testing effectiveness (Phase 2)Study completedNCT00004333
What this trial is testing

Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I

Who this might be right for
Tyrosinemia, Type I
National Center for Research Resources (NCRR)
Not applicableLooking for participantsNCT05443906
What this trial is testing

Home Exercise for Individuals With Neurodegenerative Disease

Who this might be right for
Neurodegenerative DiseasesLeukodystrophyAtaxia+5 more
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 30
Testing effectiveness (Phase 2)Study completedNCT03471143
What this trial is testing

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

Who this might be right for
Niemann-Pick Disease, Type C
Washington University School of Medicine 4
Very early researchEnded earlyNCT02357225
What this trial is testing

A Pilot Study of Pyridostigmine in Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 2
Large-scale testing (Phase 3)Study completedNCT01994889
What this trial is testing

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

Who this might be right for
Transthyretin (TTR) Amyloid Cardiomyopathy
Pfizer 441
Not applicableStudy completedNCT01339130
What this trial is testing

Assessment of Social-emotional Functioning in Neurological Diseases

Who this might be right for
Alzheimer DiseaseStrokeParkinson's Disease+2 more
Centre Hospitalier Universitaire, Amiens 177
Testing effectiveness (Phase 2)Ended earlyNCT03759678
What this trial is testing

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

Who this might be right for
Ataxia Telangiectasia
IntraBio Inc 17
Not applicableStudy completedNCT05471310
What this trial is testing

Videoocular Assessment of Eye Movement Activity in an Ataxia Telangiectasia

Who this might be right for
Ataxia TelangiectasiaDysmetria
Federal Research Institute of Pediatric Hematology, Oncology and Immunology 5
Not applicableStudy completedNCT00001774
What this trial is testing

Vasodilation in Patients With Fabry's Disease

Who this might be right for
Cerebrovascular AccidentFabry DiseaseHealthy
National Institute of Neurological Disorders and Stroke (NINDS) 48
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