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Results for “Brain Diseases, Metabolic, Inborn”

Showing 20 of 0 results

Not applicableLooking for participantsNCT05671666
What this trial is testing

Ureagenesis Analysis in Healthy Subjects and in Urea Cycle Disorder Patients

Who this might be right for
Urea Cycle Disorders
University Children's Hospital, Zurich 100
Not applicableStudy completedNCT03838237
What this trial is testing

Effect of Migalastat on Cardiac Involvement in Fabry Disease

Who this might be right for
Fabry DiseaseHeart Diseases
Ospedale San Donato 18
Not applicableNo Longer AvailableNCT00962260
What this trial is testing

Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Pfizer
Testing effectiveness (Phase 2)Study completedNCT02292654
What this trial is testing

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

Who this might be right for
Sphingomyelin Lipidosis
Genzyme, a Sanofi Company 20
Not applicableUnknownNCT03811496
What this trial is testing

Biomarker Analysis for GBA Associated Parkinson's Disease

Who this might be right for
Parkinson DiseaseGaucher Disease
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 100
Not applicableNot Yet RecruitingNCT06196931
What this trial is testing

Clinical Value of DWI-ADC Matching in the Short-term Prognosis of Wilson's Disease

Who this might be right for
Hepatolenticular Degeneration; Wilson
Hospital Affiliated to the Institute of Neurology, Anhui University of Chinese Medicine 200
Not applicableStudy completedNCT03233841
What this trial is testing

Farber Disease Natural History Study

Who this might be right for
Farber DiseaseFarber's DiseaseFarber Lipogranulomatosis+4 more
Sumitomo Pharma Switzerland GmbH 45
Large-scale testing (Phase 3)Study completedNCT04577352
What this trial is testing

Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
PTC Therapeutics 146
Testing effectiveness (Phase 2)WithdrawnNCT03228940
What this trial is testing

Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease

Who this might be right for
Fabry Disease
Resverlogix Corp
Not applicableStudy completedNCT00683189
What this trial is testing

Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy
The Cooper Health System 10
Testing effectiveness (Phase 2)Study completedNCT00283959
What this trial is testing

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 4
Not applicableLooking for participantsNCT07095062
What this trial is testing

Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.

Who this might be right for
Friedreich's AtaxiaMotor Disorders
IRCCS Eugenio Medea 30
Large-scale testing (Phase 3)WithdrawnNCT04882735
What this trial is testing

Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

Who this might be right for
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Eidos Therapeutics, a BridgeBio company
Testing effectiveness (Phase 2)UnknownNCT00068913
What this trial is testing

Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome

Who this might be right for
MELAS Syndrome
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 35
Testing effectiveness (Phase 2)Study completedNCT02228460
What this trial is testing

Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 11
Testing effectiveness (Phase 2)Temporarily pausedNCT01463215
What this trial is testing

Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease

Who this might be right for
Type I Gaucher Disease
Exsar Corporation 20
Not applicableUnknownNCT03230591
What this trial is testing

Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy

Who this might be right for
Fabry Disease
Yonsei University 25
Not applicableStudy completedNCT02838368
What this trial is testing

Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices

Who this might be right for
Pompe Disease
University of California, Irvine 921
Large-scale testing (Phase 3)Study completedNCT03566017
What this trial is testing

Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Chiesi Farmaceutici S.p.A. 97
Not applicableStudy completedNCT00001491
What this trial is testing

Analysis of the Nervous System in Patients With Fabry's Disease

Who this might be right for
Fabry's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 325
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