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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableNot Yet RecruitingNCT03725670
What this trial is testing

Direct Lentiviral Injection Gene Therapy for MLD

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shenzhen Geno-Immune Medical Institute 10
Not applicableStudy completedNCT02468570
What this trial is testing

Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 9
Not applicableStudy completedNCT01002469
What this trial is testing

Study to Evaluate 13 C Isotope Ratio Measurement for Urea Cycle Capacity Assessment

Who this might be right for
Urea Cycle Disorders
Cytonet GmbH & Co. KG 10
Not applicableStudy completedNCT03299829
What this trial is testing

A Retrospective Study to Assess the Clinical Efficacy and Safety of Trientine in Wilson's Disease Patients

Who this might be right for
Trientine Treatment for Wilson's Disease
Excelsior 48
Not applicableStudy completedNCT04050137
What this trial is testing

Therapeutic Exercise to Treat Neuropathic Pain

Who this might be right for
Gaucher DiseaseFabry Disease
Universidad de Zaragoza 5
Not applicableLooking for participantsNCT07701694
What this trial is testing

Functional Evaluation in Patients With Urea Cycle Disorders (UCD) During a Driving Task

Who this might be right for
Urea Cycle Disorder
St. Jude Children's Research Hospital 60
Large-scale testing (Phase 3)Study completedNCT01896102
What this trial is testing

The Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Who this might be right for
Cerebral Adrenoleukodystrophy (CALD)
Genetix Biotherapeutics Inc. 32
Large-scale testing (Phase 3)Active Not RecruitingNCT06673056
What this trial is testing

A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)

Who this might be right for
Ataxia-TelangiectasiaAtaxia-Telangiectasia (A-T)
IntraBio Inc 60
Not applicableLooking for participantsNCT07484945
What this trial is testing

Multiomics Approach in Adult Patients With Phenylketonuria

Who this might be right for
Phenylketonuria (PKU)
University Hospital, Tours 149
Not applicableActive Not RecruitingNCT05692596
What this trial is testing

The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel Therapeutics

Who this might be right for
Pancreatic Ductal AdenocarcinomaPancreatic CystChronic Pancreatitis+10 more
H. Lee Moffitt Cancer Center and Research Institute 317
Large-scale testing (Phase 3)Looking for participantsNCT06953583
What this trial is testing

Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
Biogen 255
Large-scale testing (Phase 3)Ended earlyNCT03643562
What this trial is testing

Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

Who this might be right for
Niemann-Pick Type C Disease
Mandos LLC 27
Not applicableStudy completedNCT00414583
What this trial is testing

Stroke in Young Fabry Patients (sifap1): Frequency of Fabry Disease in Young Stroke Patients

Who this might be right for
Cerebrovascular Accident
CENTOGENE GmbH Rostock 5,000
Not applicableLooking for participantsNCT07235709
What this trial is testing

Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study

Who this might be right for
Fabry Disease
Yonsei University 25
Not applicableLooking for participantsNCT04675749
What this trial is testing

Quality of Life in Women With X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Leipzig University Medical Center 200
Not applicableActive Not RecruitingNCT06239064
What this trial is testing

Early Genetic Identification of Obesity

Who this might be right for
Obesity, ChildhoodHyperphagiaRetinopathy+5 more
Rolfs Consulting und Verwaltungs-GmbH (RCV) 1,000
Large-scale testing (Phase 3)Study completedNCT01104649
What this trial is testing

Efficacy of Riluzole in Hereditary Cerebellar Ataxia

Who this might be right for
Cerebellar Ataxia
S. Andrea Hospital 60
Testing effectiveness (Phase 2)Study completedNCT03962114
What this trial is testing

Effects of Vitamin B3 in Patients With Ataxia Telangiectasia

Who this might be right for
Ataxia TelangiectasiaATM Gene Mutation
Radboud University Medical Center 24
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07410455
What this trial is testing

An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome

Who this might be right for
Lowe Syndrome
Can-Fite BioPharma 5
Not applicableLooking for participantsNCT02795052
What this trial is testing

Neurologic Stem Cell Treatment Study

Who this might be right for
Neurologic DisordersNervous System DiseasesNeurodegenerative Diseases+23 more
MD Stem Cells 500
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