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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableUnknownNCT03324906
What this trial is testing

Effects of Transcranial Direct Current Stimulation (tDCS) on Individuals With Prader-Willi Syndrome

Who this might be right for
Prader-Willi SyndromeObesityDepressive Disorder+1 more
Federal University of São Paulo 40
Not applicableStudy completedNCT02719249
What this trial is testing

Fabry Disease Screening in ESRD Patients in West of France

Who this might be right for
ESRD
Rennes University Hospital 500
Not applicableEnrolling By InvitationNCT06940193
What this trial is testing

A Self-test Home-use Blood Phenylalanine Monitoring System Under the Brand Name Egoo Phe System Has Been Developed for Measurement of Phenylalanine (Phe) in Individuals Diagnosed With Phenylketonuria (PKU): The Study Purpose is to Evaluate Accuracy and Usability

Who this might be right for
Phenylketonuria (PKU)
Egoo Health Aps 20
Testing effectiveness (Phase 2)WithdrawnNCT03687476
What this trial is testing

Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease

Who this might be right for
Niemann-Pick Disease, Type C
Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
Testing effectiveness (Phase 2)Study completedNCT01720316
What this trial is testing

Neurobiology of a Mutation in Glycine Metabolism in Psychotic Disorders

Who this might be right for
Schizo-affective DisorderBipolar Disorder
Mclean Hospital 2
Not applicableStudy completedNCT00004378
What this trial is testing

Stem Cell Transplantation (SCT) for Genetic Diseases

Who this might be right for
ThrombocytopeniaMetachromatic LeukodystrophyFanconi's Anemia+3 more
National Center for Research Resources (NCRR)
Not applicableEnded earlyNCT00001178
What this trial is testing

Brain Study of Patients With Frontal Lobe Dementia and Parkinsonian Disorders

Who this might be right for
Alzheimer's DiseaseDementiaDown's Syndrome
National Institute of Neurological Disorders and Stroke (NINDS) 597
Testing effectiveness (Phase 2)Looking for participantsNCT05092685
What this trial is testing

Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn

Who this might be right for
Ornithine Transcarbamylase Deficiency
University College, London 12
Not applicableUnknownNCT03045042
What this trial is testing

Search for Serum/Plasma Biomarkers in Pompe's Disease

Who this might be right for
Late Onset Pompe DiseasePompe Disease
Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau 35
Early research (Phase 1)Study completedNCT06128954
What this trial is testing

Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily).

Who this might be right for
Wilson's Disease
Orphalan 26
Not applicableUnknownNCT06497829
What this trial is testing

Geranylgeraniol Supplementation in Patients With Mevalonate Kinase Deficiency

Who this might be right for
Mevalonate Kinase Deficiency
University Hospital, Motol 6
Post-approval studies (Phase 4)Study completedNCT02574286
What this trial is testing

Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
Shire 21
Not applicableLooking for participantsNCT07336394
What this trial is testing

Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques

Who this might be right for
Danon DiseaseFabry DiseaseCardiac Amyloidosis+4 more
Chinese Academy of Medical Sciences, Fuwai Hospital 1,000
Not applicableUnknownNCT03812042
What this trial is testing

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

Who this might be right for
Lysosomal Storage Diseases
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 100,000
Testing effectiveness (Phase 2)Looking for participantsNCT04998396
What this trial is testing

AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

Who this might be right for
Canavan Disease
Aspa Therapeutics 26
Not applicableEnrolling By InvitationNCT07703020
What this trial is testing

Prevalence and Determinants of Obesity in PKU Patients

Who this might be right for
Phenylketonuria (PKU)
Christel Tran 100
Not applicableLooking for participantsNCT03409003
What this trial is testing

Orphan Europe Carbaglu® Surveillance Protocol

Who this might be right for
N-acetylglutamate Synthase (NAGS) Deficiency
Nicholas Ah Mew 30
Not applicableLooking for participantsNCT04880356
What this trial is testing

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Who this might be right for
Inherited DiseaseRare DiseasesMetabolic Disease+3 more
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100
Testing effectiveness (Phase 2)Study completedNCT00355264
What this trial is testing

Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency

Who this might be right for
Tetrahydrobiopterin DeficienciesHyperphenylalaninemia, Non-Phenylketonuric
BioMarin Pharmaceutical 12
Not applicableNot Yet RecruitingNCT06956573
What this trial is testing

Aortic Dimensions in Patients With Fabry Disease

Who this might be right for
Fabry Disease
University Hospital, Caen 200
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