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Metabolism, Inborn Errors

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Metabolism, Inborn Errors

Explore clinical trials studying Metabolism, Inborn Errors. Study availability and eligibility vary by location and protocol.

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Metabolism, Inborn Errors trial results

Completed

Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II

NCT06022380

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
Locations: Manchester University NHS Foundation Trust Manchester, Lancashire, United Kingdom

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Completed

Screening for Hypercholesterolemia in Children Using Dried Blood Spot

NCT05191355

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Genetic Diseases, Inborn, +9 more
Locations: Hospices Civils de Lyon, Dept of Endocrinology Bron, France

Hospices Civils de Lyon, Service d'Hépatologie et de Gastro-Entérologie et Nutrition Pédiatrique Bron, France

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Completed

Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants

NCT04526210

Conditions: Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations: Clinical Trial Site Austin, Texas, United States

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Completed

Examining the Effect of Burosumab on Muscle Function

NCT04146935

Conditions: Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +26 more
Locations: Yale University School of Medicine New Haven, Connecticut, United States

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Completed

Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.

NCT04526197

Conditions: Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations: Clinical Trial Site Austin, Texas, United States

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Completed

Comparative Bioavailability Study of TAH3311 5 mg Oral Dissolving Film vs ELIQUIS® 5 mg Tablet in Healthy Volunteers

NCT05995119

Conditions: Behavior, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, +13 more
Locations: Novum Pharmaceutical Research Services Las Vegas, Nevada, United States

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Completed

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

NCT04106544

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations: Investigational Site Number :8400002 Atlanta, Georgia, United States

Investigational Site Number :8400003 The Bronx, New York, United States

Investigational Site Number :8400001 Valhalla, New York, United States

Investigational Site Number :0320002 CABA, Argentina

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Completed

A Study of the Cardiac Effects of ALXN1840 in Healthy Adults

NCT04560816

Conditions: Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations: PPD Development, LP Austin, Texas, United States

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Completed

Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders

NCT01274026

Conditions: Amino Acid Metabolism, Inborn Errors, Behavior, Behavior and Behavior Mechanisms, +13 more
Locations: Tulane University Health Science Center New Orleans, Louisiana, United States

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Completed

A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial Hypercholesterolemia

NCT04031742

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Genetic Diseases, Inborn, +9 more
Locations: Beijing Anzhen Hospital, Capital Medical University Beijing, China

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Metabolism, Inborn Errors

Metabolism, Inborn Errors trial results

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