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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Not Yet RecruitingNCT07681713
What this trial is testing

Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)

Who this might be right for
Friedreich's Ataxia
PTC Therapeutics 120
Post-approval studies (Phase 4)Ended earlyNCT01597596
What this trial is testing

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Who this might be right for
Pompe Disease (Infantile-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenosis 2+1 more
Genzyme, a Sanofi Company 4
Not applicableUnknownNCT04320329
What this trial is testing

Natural History of Morquio B and Late-Onset of GM1 Gangliosidosis

Who this might be right for
Morquio B DiseaseGM1 Gangliosidosis Type III
University of British Columbia 30
Large-scale testing (Phase 3)Study completedNCT03694353
What this trial is testing

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

Who this might be right for
Phenylketonuria (PKU)
BioMarin Pharmaceutical 37
Not applicableNot Yet RecruitingNCT06258577
What this trial is testing

Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

Who this might be right for
Gaucher Disease
Chung-Hsing Wang 50
Large-scale testing (Phase 3)Ended earlyNCT01458119
What this trial is testing

Open-Label Phase 3 Long-Term Safety Study of Migalastat

Who this might be right for
Fabry Disease
Amicus Therapeutics 85
Testing effectiveness (Phase 2)Looking for participantsNCT07667387
What this trial is testing

Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders

Who this might be right for
Urea Cycle DisordersCarbamoyl-Phosphate Synthase I Deficiency
Rebecca Ahrens-Nicklas 7
Large-scale testing (Phase 3)Active Not RecruitingNCT05345171
What this trial is testing

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

Who this might be right for
OTC Deficiency
Ultragenyx Pharmaceutical Inc 37
Early research (Phase 1)Ended earlyNCT00004498
What this trial is testing

Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency

Who this might be right for
Ornithine Transcarbamylase Deficiency Disease
University of Pennsylvania 21
Not applicableNot Yet RecruitingNCT06743906
What this trial is testing

Role of OCT & OCT Angiography in Patients With Posterior at the Uveitis Clinic of Assiut University Hospital.

Who this might be right for
Uveitis, Posterior
Assiut University 25
Not applicableStudy completedNCT03470584
What this trial is testing

Vegetarian Diet and Chronic Degenerative Diseases

Who this might be right for
DementiaDepressionCardiovascular Diseases+5 more
Dalin Tzu Chi General Hospital 18,064
Large-scale testing (Phase 3)Study completedNCT00430625
What this trial is testing

Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1
Shire 25
Not applicableLooking for participantsNCT06162338
What this trial is testing

The Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I

Who this might be right for
Gaucher Disease Type I
He Huang 3
Not applicableLooking for participantsNCT00358943
What this trial is testing

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

Who this might be right for
Gaucher DiseaseCerebroside Lipidosis SyndromeGlucocerebrosidase Deficiency Disease+1 more
Genzyme, a Sanofi Company 12,000
Testing effectiveness (Phase 2)Active Not RecruitingNCT04127578
What this trial is testing

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

Who this might be right for
Parkinson Disease
Prevail Therapeutics 32
Not applicableStudy completedNCT01592552
What this trial is testing

A Biospecimen and Clinical Data Study on Patients for Drug & Biomarker Discovery

Who this might be right for
Alzheimer's DiseaseMultiple SclerosisParkinson's Disease+2 more
Sanguine Biosciences 20
Testing effectiveness (Phase 2)Study completedNCT00041535
What this trial is testing

OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease

Who this might be right for
Gaucher Disease
National Institute of Neurological Disorders and Stroke (NINDS) 30
Not applicableStudy completedNCT04569604
What this trial is testing

QoL and Cognitive Function in Patients With Hypoparathyroidism

Who this might be right for
HypoparathyroidismPseudo Hypoparathyroidism
University of Aarhus 64
Not applicableStudy completedNCT02445521
What this trial is testing

Testing of Four Home Phenylalanine Monitoring Prototype Devices

Who this might be right for
Phenylketonurias
Emory University 9
Not applicableStudy completedNCT04469894
What this trial is testing

Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick

Who this might be right for
Niemann-Pick Diseases
National Niemann-Pick Disease Foundation 76
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