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Results for “Genetic Inborn Errors of Metabolism”

3,356 trials

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Showing 20 of 3,356 results

Not applicableStudy completedNCT02638220
What this trial is testing

Cerebrotendinous Xanthomatosis (CTX) Prevalence Study

Who this might be right for
Cerebrotendinous Xanthomatosis (CTX)
Travere Therapeutics, Inc. 442
Not applicableStudy completedNCT02851862
What this trial is testing

A Natural History of Late Onset Tay-Sachs Disease

Who this might be right for
GM2 Gangliosidosis
Massachusetts General Hospital 10
Not applicableStudy completedNCT01685424
What this trial is testing

Etoricoxib Prescribing Patterns and Adverse Events of Interest in Primary Care in the United Kingdom (MK-0663-162 AM1)

Who this might be right for
OsteoarthritisRheumatoid ArthritisAnkylosing Spondylitis+2 more
Organon and Co 79,189
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 applicableUnknownNCT02702479
What this trial is testing

Meta-analyses of the Effect of Sucrose Versus High Fructose Corn Syrup on Cardiometabolic Risk

Who this might be right for
ObesityDyslipidemiaPrediabetes+5 more
University of Toronto 1
Testing effectiveness (Phase 2)Study completedNCT03580122
What this trial is testing

The Effect of Arginine on Classic Galactosemia

Who this might be right for
Classic Galactosemia
Academisch Ziekenhuis Maastricht 4
Early research (Phase 1)Study completedNCT00738842
What this trial is testing

Uric Acid Study in Healthy Male Volunteers

Who this might be right for
GoutCoronary Artery Disease
AstraZeneca 24
Not applicableUnknownNCT04388969
What this trial is testing

World Data on Ambroxol for Patients With GD and GBA Related PD

Who this might be right for
Gaucher DiseaseParkinson DiseaseGBA Gene Mutation
Shaare Zedek Medical Center 300
Testing effectiveness (Phase 2)Ended earlyNCT03848182
What this trial is testing

Analyzing Childhood Recall Antigens in Patients With Pancreatic Cancer

Who this might be right for
Pancreatic Cancer
Albert Einstein College of Medicine 10
Not applicableStudy completedNCT00328159
What this trial is testing

Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids

Who this might be right for
Inborn Errors of Metabolism
Assistance Publique - Hôpitaux de Paris 20
Early research (Phase 1)Study completedNCT00768183
What this trial is testing

Comparison of KADIAN 100 mg When Dosed With Alcohol Under Fasting and Fed Conditions Compared to Water

Who this might be right for
Healthy
Actavis Inc. 32
Not applicableStudy completedNCT04353466
What this trial is testing

Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs

Who this might be right for
Gaucher Disease, Type 1
Shaare Zedek Medical Center 30
Not applicableNo Longer AvailableNCT03544060
What this trial is testing

Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

Who this might be right for
Familial Chylomicronemia
Akcea Therapeutics
Testing effectiveness (Phase 2)Study completedNCT00001596
What this trial is testing

Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

Who this might be right for
AlbinismInborn Errors of MetabolismOculocutaneous Albinism+2 more
William Gahl, M.D. 35
Not applicableWithdrawnNCT02385162
What this trial is testing

Biomarker for Glycogen Storage Diseases (BioGlycogen)

Who this might be right for
Fructose Metabolism, Inborn ErrorsGlycogen Storage DiseaseGlycogen Storage Disease Type I+7 more
CENTOGENE GmbH Rostock
Testing effectiveness (Phase 2)Study completedNCT00925054
What this trial is testing

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 40
Not applicableStudy completedNCT03871673
What this trial is testing

The Use of Uncooked Sweet Manioc Starch to Treat Hepatic Glycogen Storage Diseases

Who this might be right for
Glycogen Storage Disease Type I
Hospital de Clinicas de Porto Alegre 11
Large-scale testing (Phase 3)Study completedNCT02319005
What this trial is testing

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

Who this might be right for
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)Amyloidosis, HereditaryAmyloid Neuropathies, Familial+3 more
Alnylam Pharmaceuticals 206
Not applicableUnknownNCT03875105
What this trial is testing

Daily Living Activities and Exercise Capacity in Lipid Storage Diseases

Who this might be right for
Lipid Storage Disease
Istanbul University 25
Not applicableUnknownNCT02870660
What this trial is testing

Familial Hypercholesterolemia Amongst Patients With Acute Coronary Syndrome

Who this might be right for
Familial HypercholesterolemiaCardiac EventAcute Coronary Syndrome
Isfahan University of Medical Sciences 500
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