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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableUnknownNCT03357978
What this trial is testing

Susceptibility to Infections, Tumor Risk and Liver Disease in Patients With Ataxia Telangiectasia

Who this might be right for
Ataxia Telangiectasia
Johann Wolfgang Goethe University Hospital 30
Large-scale testing (Phase 3)Active Not RecruitingNCT05222906
What this trial is testing

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Who this might be right for
Gaucher's Disease Type III
Sanofi 43
Not applicableLooking for participantsNCT06289348
What this trial is testing

Announcement of Rare Metabolic Diseases in Systematic Newborn Screening: the Phenylketonuria Experience.

Who this might be right for
Phenylketonuria
Assistance Publique - Hôpitaux de Paris 80
Not applicableLooking for participantsNCT06213090
What this trial is testing

Patterns of Neurodevelopmental Disorders

Who this might be right for
Neurodevelopmental DisordersAutism Spectrum DisorderPediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal Infection+5 more
Richard Frye 1,000
Not applicableUnknownNCT05536388
What this trial is testing

Drug Discovery for Parkinson's With Mutations in the GBA Gene

Who this might be right for
Parkinson DiseaseGaucher DiseaseHealthy+1 more
New York Stem Cell Foundation Research Institute 60
Testing effectiveness (Phase 2)Study completedNCT01303146
What this trial is testing

Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation

Who this might be right for
Metachromatic Leukodystrophy
Assistance Publique - Hôpitaux de Paris 1
Post-approval studies (Phase 4)Study completedNCT00486889
What this trial is testing

Growth and Development Study of Alglucosidase Alfa

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease
Genzyme, a Sanofi Company 12
Early research (Phase 1)UnknownNCT05531890
What this trial is testing

Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects

Who this might be right for
Ataxia Telangiectasia
Grace Therapeutics Inc. 48
Not applicableStudy completedNCT03548779
What this trial is testing

North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2

Who this might be right for
Epilepsy; SeizureNeuromuscular DiseasesBrain Malformation+14 more
University of North Carolina, Chapel Hill 548
Not applicableUnknownNCT04624789
What this trial is testing

Registry Gangliosidoses

Who this might be right for
GangliosidosesGM1 GangliosidosisMorquio B Disease+6 more
SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) 40
Large-scale testing (Phase 3)WithdrawnNCT02921620
What this trial is testing

Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease

Who this might be right for
Fabry Disease
Protalix
Testing effectiveness (Phase 2)Study completedNCT04046224
What this trial is testing

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

Who this might be right for
Fabry Disease
Sangamo Therapeutics 36
Not applicableLooking for participantsNCT06224413
What this trial is testing

Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

Who this might be right for
Cerebral Adrenoleukodystrophy (CALD)
Genetix Biotherapeutics Inc. 120
Not applicableStudy completedNCT05249218
What this trial is testing

Impact of Fruit and Vegetables in PKU

Who this might be right for
Phenylketonurias
Alex Pinto 16
Not applicableLooking for participantsNCT04573660
What this trial is testing

Abbott Vascular Medical Device Registry

Who this might be right for
Acute Myocardial InfarctionRestenoses, CoronaryCoronary Artery Lesions+2 more
Abbott Medical Devices 3,784
Not applicableActive Not RecruitingNCT00209235
What this trial is testing

Albright Hereditary Osteodystrophy: Natural History, Growth, and Cognitive/Behavioral Assessments

Who this might be right for
Pseudohypoparathyroidism Type 1AAlbright Hereditary OsteodystrophyPseudopseudohypoparathyroidism
Connecticut Children's Medical Center 600
Not applicableUnknownNCT02120235
What this trial is testing

Investigating Lysosomal Storage Diseases in Minority Groups

Who this might be right for
Lysosomal Storage DisordersGaucher DiseaseFabry Disease+2 more
O & O Alpan LLC 20,000
Not applicableStudy completedNCT00964236
What this trial is testing

The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)

Who this might be right for
Phenylketonuria
University of Missouri-Columbia 20
Testing effectiveness (Phase 2)UnknownNCT00004293
What this trial is testing

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

Who this might be right for
Gaucher's Disease
National Center for Research Resources (NCRR) 24
Not applicableStudy completedNCT00001771
What this trial is testing

I-123 Brain Studies of Serotonin Metabolism in Psychiatric Patients and Normal Volunteers

Who this might be right for
HealthyMental DisorderObsessive Compulsive Disorder+2 more
National Institute of Mental Health (NIMH) 112
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