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Results for “Brain Diseases, Metabolic, Inborn”

1,552 trials

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Showing 20 of 1,552 results

Large-scale testing (Phase 3)Study completedNCT00478647
What this trial is testing

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

Who this might be right for
Gaucher Disease
Shire 40
Not applicableLooking for participantsNCT03564561
What this trial is testing

Natural History of Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II, Adult
Assistance Publique - Hôpitaux de Paris 20
Not applicableUnknownNCT02000310
What this trial is testing

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Who this might be right for
Lysosomal Storage Disorders
O & O Alpan LLC 80
Not applicableEnded earlyNCT04008849
What this trial is testing

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 3
Not applicableActive Not RecruitingNCT04848779
What this trial is testing

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

Who this might be right for
Glycogen Storage Disease Type II
Sanofi 16
Testing effectiveness (Phase 2)WithdrawnNCT02715505
What this trial is testing

Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)

Who this might be right for
Inherited Metabolic Disorders IMD
Novartis Pharmaceuticals
Not applicableStudy completedNCT04804566
What this trial is testing

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

Who this might be right for
Fabry Disease
Amicus Therapeutics 44
Not applicableUnknownNCT04476550
What this trial is testing

Clinical Specimen Collection From Pompe Disease Patients

Who this might be right for
Pompe Disease
Serhat Gumrukcu, MD PhD 12
Not applicableApproved For MarketingNCT00484991
What this trial is testing

Sapropterin Expanded Access Program

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical
Large-scale testing (Phase 3)Study completedNCT01994889
What this trial is testing

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

Who this might be right for
Transthyretin (TTR) Amyloid Cardiomyopathy
Pfizer 441
Not applicableStudy completedNCT04141891
What this trial is testing

Advancing Understanding of Transportation Options

Who this might be right for
Diabetic RetinopathyMacular DegenerationGlaucoma+29 more
University of Colorado, Denver 529
Not applicableLooking for participantsNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Testing effectiveness (Phase 2)Study completedNCT03378531
What this trial is testing

AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency

Who this might be right for
Arginase I DeficiencyHyperargininemia
Aeglea Biotherapeutics 14
Post-approval studies (Phase 4)Ended earlyNCT00230607
What this trial is testing

Study of the Effects of Fabrazyme Treatment on Lactation and Infants

Who this might be right for
Fabry DiseaseAlpha Galactosidase A Deficiency
Genzyme, a Sanofi Company 7
Not applicableLooking for participantsNCT06967831
What this trial is testing

Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells

Who this might be right for
Leigh Syndrome (Maternally Inherited, MILS)Leigh Syndrome (AR, AD, XR)
Charite University, Berlin, Germany 80
Not applicableStudy completedNCT00233870
What this trial is testing

A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease.

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 405
Testing effectiveness (Phase 2)Study completedNCT04455230
What this trial is testing

A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190

Who this might be right for
Fabry DiseaseLysosomal Storage Diseases
Spur Therapeutics 3
Not applicableApproved For MarketingNCT05183802
What this trial is testing

An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)

Who this might be right for
Bardet-Biedl Syndrome (BBS)Obesity
Rhythm Pharmaceuticals, Inc.
Not applicableUnknownNCT02702375
What this trial is testing

Meta-analyses of Impotrant Food Sources of Sugars and Incident Cardiometabolic Diseases

Who this might be right for
Diabetes Mellitus, Type 2Metabolic Syndrome XHypertension+3 more
University of Toronto 1
Not applicableLooking for participantsNCT03131427
What this trial is testing

China Registry for Genetic / Metabolic Liver Diseases

Who this might be right for
Genetic/Metabolic Liver Diseases
Beijing Friendship Hospital 20,000
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