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Condition: Inborn Errors Of Metabolism Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Inborn Errors Of Metabolism

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Exercise Training in Transthyretin Cardiac Amyloidosis
NA · 1 location(s) · Brigham and Women's Hospital · NCT05797857Cardiac Structure and Function in MPS
Phase N/A · 3 location(s) · Children's Hospital of Orange County · NCT07361536A Phase 2 Trial of Dotinurad in Xanthine Oxidase Inhibitor (XOI) Intolerant/Uricase Failure Gout Participants
Phase 2 · 17 location(s) · Crystalys Therapeutics · NCT07535034Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI
Phase 2 · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT06887777A Gaucher Disease Gene Therapy Trial With FLT201
Phase 3 · 33 location(s) · Spur Therapeutics · NCT07223944Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome
Phase N/A · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT00001456Assessment of Physician Consideration of EPRO's, from Patients with Gout, Rheumatoid Arthritis, Sjogren's Syndrome or Sy
Phase N/A · 1 location(s) · University Hospital, Strasbourg, France · NCT05927688An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Phase 1/Phase 2 · 3 location(s) · JCR Pharmaceuticals Co., Ltd. · NCT06488924Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome
Phase 2 · 1 location(s) · Genethon · NCT06518005Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreat
Phase 1 · 1 location(s) · GeneCradle Inc · NCT05860569Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing
Phase N/A · 1 location(s) · Eunice Kennedy Shriver National Institute of Child Health an · NCT06749366A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeedin
Phase N/A · 1 location(s) · Amicus Therapeutics · NCT04252066Physiologic Assessment of Microvascular Function in Patients With Cardiac Amyloidosis
Phase N/A · 1 location(s) · Samsung Medical Center · NCT02798705Dose Response of Exercise for Arthritis Management
NA · 1 location(s) · University of South Carolina · NCT06880653Registre HEAR, Healthcare European Amyloidosis Registry
Phase N/A · 1 location(s) · Saving Lives Matters · NCT05101304Krabbe Disease Global Patient Registry
Phase N/A · 1 location(s) · State University of New York at Buffalo · NCT02993796Natural History Study of Patients With Excess Androgen
Phase N/A · 2 location(s) · Eunice Kennedy Shriver National Institute of Child Health an · NCT00250159CHEST-CA: Study of Chest Pain and Hidden Cardiac Amyloidosis
NA · 1 location(s) · Rigshospitalet, Denmark · NCT07435168Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI
NA · 1 location(s) · University Health Network, Toronto · NCT04856059Ovarian Tissue Cryopreservation in Pre-Pubertal (OTC-Pre Pubertal)
Phase N/A · 1 location(s) · Erin Rowell · NCT05299710Clinical and Basic Investigations Into Known and Suspected Congenital Disorders of Glycosylation
Phase N/A · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT02089789ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
Phase N/A · 40 location(s) · Alnylam Pharmaceuticals · NCT04561518Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy
NA · 4 location(s) · Third Military Medical University · NCT06796920[18F]FT8 PET Imaging in Immunoglobulin Light Chain Amyloidosis
Phase N/A · 2 location(s) · Tianjin Medical University · NCT07232459Prevalence of Abnormalities in Ultrasonography of Joint and Tendons in Patients With Gout
Phase N/A · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT05546593GSD VI and GSD IX Natural History
Phase N/A · 1 location(s) · Duke University · NCT04454216Cardiac Involvement in Wilson's Disease
NA · 1 location(s) · Fondation Ophtalmologique Adolphe de Rothschild · NCT05493605Comprehensive Program for Hereditary Transthyretin Amyloidosis
Phase N/A · 1 location(s) · Hospital de Alta Complejidad en Red · NCT07213297Geranylgeraniol Supplementation in Patients With Mevalonate Kinase Deficiency
NA · 1 location(s) · University Hospital, Motol · NCT06497829Screening for Familial Hypercholesterolemia in Children
NA · 1 location(s) · Centre Hospitalier Universitaire, Amiens · NCT06555120
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.