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Clinical trial condition

Metabolism, Inborn Errors

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

2,493 public trials 456 recruiting studies
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Metabolism, Inborn Errors trial results

Recruiting

European Cystinosis Cohort

NCT05901077

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystinosis, Genetic Diseases, Inborn, +4 more
Locations
  • RaDiCo-ECYSCO Paris, Île-de-France Region, France
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Recruiting

GenSci144 Tablets Phase I Clinical Trial

NCT07685210

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • The First Affiliated Hospital of Shandong First Medical University (Shandong Qianfoshan Hospital). Jinan, Shandong, China
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Recruiting

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

NCT06636383

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +8 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Children's Hospital Colorado Denver, Colorado, United States
  • University of Connecticut Health Center Hartford, Connecticut, United States
  • University of Michigan Ann Arbor, Michigan, United States
View all 19 locations
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Recruiting

Functional Capacity in Anderson-Fabry Disease Patients

NCT06884358

Conditions
Anderson-Fabry Disease, Brain Diseases, Brain Diseases, Metabolic, +20 more
Locations
  • Spedali Civili Hospital Brescia, Italy
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan, Italy
  • IRCCS Policlinico San Donato San Donato Milanese, Milan, Italy
  • Fondazione IRCCS San Gerardo dei Tintori Monza, Italy
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Recruiting

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases

NCT06451757

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +22 more
Locations
  • Massachusetts General Hospital Boston, Massachusetts, United States
  • Cleveland Clinic Neurological Institute Mellen Center Cleveland, Ohio, United States
  • The University of Texas Health Science Center at Houston Houston, Texas, United States
  • Rigshospitalet, University of Copenhagen Kopenhagen, Region Sjælland, Denmark
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Recruiting

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

NCT07354724

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of California-Irvine Irvine, California, United States
  • Duke University School of Medicine - Early Phase Research Unit Durham, North Carolina, United States
  • The Lysosomal & Rare Disorders Research & Treatment Center Fairfax, Virginia, United States
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Recruiting

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

NCT06563895

Conditions
Alzheimer Disease, Amyloid Cardiomyopathy, Amyloid Neuropathies, +26 more
Locations
  • University of California, San Diego (UCSD) - Medical Center La Jolla, California, United States
  • University of California, Los Angeles (UCLA) - David Geffen School of Medicine Los Angeles, California, United States
  • University of California, San Francisco (UCSF) San Francisco, California, United States
  • Stanford University Stanford, California, United States
View all 103 locations
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Recruiting

An Extension Study to Evaluate Safety and Efficacy of Atumelnant in Participants With Congenital Adrenal Hyperplasia

NCT06712823

Conditions
Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, Adrenogenital Syndrome, +17 more
Locations
  • Crinetics Study Site, Minneapolis, Minnesota 55454 Minneapolis, Minnesota, United States
  • Crinetics Study Site Morehead City, North Carolina, United States
  • Crinetics Study Site Philadelphia, Pennsylvania, United States
  • Crinetics Study Site Córdoba, Córdoba Province, Argentina
View all 13 locations
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