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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.

2,496 public trials 457 recruiting studies
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Metabolism, Inborn Errors trial results

Completed

Artificial Intelligence Enhanced ECG to Detect Cardiac Amyloidosis

NCT05557162

Conditions
Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +13 more
Locations
  • Mayo Clinic in Arizona Scottsdale, Arizona, United States
  • Mayo Clinic in Florida Jacksonville, Florida, United States
  • Mayo Clinic in Rochester Rochester, Minnesota, United States
  • Mayo Clinic Health System - Eau Claire Eau Claire, Wisconsin, United States
View Trial Details
Completed

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

NCT04049760

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of South Florida Tampa, Florida, United States
  • The Emory Clinic Atlanta, Georgia, United States
  • University of Minnesota Masonic Children's Hospital and Clinics Minneapolis, Minnesota, United States
  • Cincinnati Children's Hospital Cincinnati, Ohio, United States
View Trial Details
Completed

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

NCT04281537

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Amicus Therapeutics, Inc. Philadelphia, Pennsylvania, United States
  • Instituto de Genética e Erros Inatos do Metabolismo (IGEIM) São Paulo, Brazil
  • Keio University Hospital Tokyo, Japan
  • Yokohama Municipal Citizen's Hospital Yokohama, Japan
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Completed

Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

NCT04601051

Conditions
Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +19 more
Locations
  • Clinical Trial Site Paris, France
  • Clinical Trial Site Auckland, New Zealand
  • Clinical Trial Site Umeå, Sweden
  • Clinical Trial Site London, United Kingdom
View Trial Details
Completed

OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)

NCT05972954

Conditions
Acid-Base Imbalance, Acidosis, Acidosis, Lactic, +44 more
Locations
  • Universitätsklinikum Bonn, Sektion Neuromuskuläre Erkrankungen, Klinik und Poliklinik für Neurologie, Venusberg-Campus 1, Gebäude 80, NPP Bonn, Germany
  • Medizinisch Fakultät der Martin-Luther-Universität Halle-Wittenberg Universitätsklinik und Poliklinik für Neurologie Universitätsklinikum , Ernst-Grube-Str. 40 Halle, Germany
  • Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik, Klinikum der Universität München, Ludwig-Maximilians-Universität (LMU Klinikum), Ziemssenstr. 1a Munich, Germany
  • IRCCS Institute of Neurological Science of Bologna, University of Bologna, Department of Biomedical and Neuromotor Science (DIBINEM), Ospedale Bellaria Via Altura, 3 Bologna, Italy
View Trial Details
Completed

Short Course Primaquine for the Radical Cure of P. Vivax Malaria - Indonesia

NCT05879224

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +18 more
Locations
  • Puskesmas Hanura Hanura, Lampung, Indonesia
  • Puskesmas Tanjung Leidong Labuhanbatu, North Sumatra, Indonesia
  • Puskesmas Bhintuka Mimika, Special Region of Papua, Indonesia
  • Puskesmas Pasar Sentral Mimika, Special Region of Papua, Indonesia
View Trial Details

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