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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Not applicableStudy completedNCT02053896
What this trial is testing

A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
ISU Abxis Co., Ltd. 5
Not applicableWithdrawnNCT04272736
What this trial is testing

PKU Low Calorie Drink Study

Who this might be right for
PhenylketonuriasHyperphenylalaninaemia
Nutricia UK Ltd
Not applicableLooking for participantsNCT03789721
What this trial is testing

Adrenoleukodystrophy National Registry Study

Who this might be right for
ALD (Adrenoleukodystrophy)AdrenoleukodystrophyCerebral Adrenoleukodystrophy
Masonic Cancer Center, University of Minnesota 1,000
Large-scale testing (Phase 3)Looking for participantsNCT06302348
What this trial is testing

Sepiapterin in Participants With Phenylketonuria (PKU)

Who this might be right for
Phenylketonuria
PTC Therapeutics 56
Testing effectiveness (Phase 2)Study completedNCT01339884
What this trial is testing

Resveratrol as Treatment for Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
Murdoch Childrens Research Institute 27
Not applicableStudy completedNCT02769949
What this trial is testing

Pediatric Patients With Metabolic or Other Genetic Disorders

Who this might be right for
Genetic DisorderAsperger DisorderAutism Spectrum Disorder+2 more
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 96
Not applicableNot Yet RecruitingNCT07075887
What this trial is testing

Optic Nerve Head Evaluation Through Multimodal Blood Flow Analysis: a Prospective Observational, Multi-center Study

Who this might be right for
Neuro-OphthalmologyOptic NeuropathiesOptic Atrophy, Hereditary, Leber+3 more
Barboni 70
Not applicableLooking for participantsNCT07382128
What this trial is testing

Myocardial Perfusion CMR for Differentiating and Characterizing Hypertrophic Cardiomyopathy Phenotypes

Who this might be right for
HCM - Hypertrophic CardiomyopathyAnderson Fabry DiseaseCardiac Magnetic Resonance Imaging+1 more
IRCCS Azienda Ospedaliero-Universitaria di Bologna 250
Not applicableUnknownNCT04032639
What this trial is testing

Stress and Brain Response Using MEG in PWS

Who this might be right for
Prader-Willi Syndrome
The Hospital for Sick Children 48
Large-scale testing (Phase 3)Ended earlyNCT04378075
What this trial is testing

Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

Who this might be right for
Mitochondrial DiseasesDrug Resistant EpilepsyLeigh Disease+5 more
PTC Therapeutics 68
Not applicableStudy completedNCT01820702
What this trial is testing

Short Term Bed Rest Study: Evaluation of the Use of Artificial Gravity, Induced by Short-arm Centrifugation

Who this might be right for
Bone Diseases, MetabolicAcid-Base ImbalanceMuscular Atrophy+4 more
DLR German Aerospace Center 10
Testing effectiveness (Phase 2)Ended earlyNCT02898753
What this trial is testing

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease
Valerion Therapeutics, LLC 12
Not applicableUnknownNCT04055831
What this trial is testing

Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease Type 1
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 40
Testing effectiveness (Phase 2)UnknownNCT06114329
What this trial is testing

Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease

Who this might be right for
Fabry Disease
AceLink Therapeutics, Inc. 16
Not applicableLooking for participantsNCT02132741
What this trial is testing

Optical Coherence Tomography And NEphropathy: The OCTANE Study

Who this might be right for
HealthHypertensionChronic Kidney Disease
University of Edinburgh 150
Not applicableStudy completedNCT04041102
What this trial is testing

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

Who this might be right for
GM1 Gangliosidosis
University of Pennsylvania 31
Post-approval studies (Phase 4)Study completedNCT01257737
What this trial is testing

To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)

Who this might be right for
Urea Cycle Disorders
Amgen 88
Not applicableStudy completedNCT00343577
What this trial is testing

Antiproteinuric Agents and Fabry Disease

Who this might be right for
Fabry DiseaseProteinuria
University of Alabama at Birmingham 12
Not applicableLooking for participantsNCT06887283
What this trial is testing

Essen Amyloidosis Registry

Who this might be right for
Amyloidosis; SystemicAmyloidosis Cardiac
University Hospital, Essen 400
Testing effectiveness (Phase 2)Study completedNCT04571970
What this trial is testing

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Who this might be right for
Mucopolysaccharidosis Type II (MPS II)
REGENXBIO Inc. 6
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