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

Recruiting studies: Amino Acid Metabolism Inborn Errors

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 →

Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome
Phase N/A · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT00001456Ovarian Tissue Cryopreservation in Pre-Pubertal (OTC-Pre Pubertal)
Phase N/A · 1 location(s) · Erin Rowell · NCT05299710A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
Phase N/A · 11 location(s) · BioMarin Pharmaceutical · NCT06305234A Study of JNT-517 in Participants With Phenylketonuria (PKU)
Phase 3 · 26 location(s) · Otsuka Pharmaceutical Development & Commercialization, Inc. · NCT06971731A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
Phase 2 · 1 location(s) · Arcturus Therapeutics, Inc. · NCT06488313Functional Evaluation in Patients With Urea Cycle Disorders (UCD) During a Driving Task
Phase N/A · 1 location(s) · St. Jude Children's Research Hospital · NCT07701694Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
Phase N/A · 32 location(s) · RECORDATI GROUP · NCT04176523Comparing Regorafenib Combined With Envafolimab to Physician's Choice in Patients With Metastatic Gastrointestinal Strom
Phase 2 · 1 location(s) · Jian Li · NCT07559864Safety and Efficacy Study of NGGT002 in PKU Adult Subjects
EARLY_Phase 1 · 1 location(s) · The First Affiliated Hospital of Bengbu Medical University · NCT06061614FARD (RaDiCo Cohort) (RaDiCo-FARD)
Phase N/A · 15 location(s) · Institut National de la Santé Et de la Recherche Médicale, F · NCT05954416Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU
Phase N/A · 1 location(s) · Birmingham Women's and Children's NHS Foundation Trust · NCT07728032Hepatic Histopathology in Urea Cycle Disorders
Phase N/A · 2 location(s) · Baylor College of Medicine · NCT04908319Neonatal Screening of Biotinidase Deficiency: Genotype-phenotype Correlation and Clinical Follow-up
Phase N/A · 1 location(s) · IRCCS Azienda Ospedaliero-Universitaria di Bologna · NCT06723925An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Phase 1/Phase 2 · 12 location(s) · iECURE, Inc. · NCT06255782Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Phase N/A · 1 location(s) · iECURE, Inc. · NCT06805695AAVrh10-PCCA Gene Therapy for Propionic Acidemia
Phase 1 · 1 location(s) · Mayo Clinic · NCT07643844Prospective Research Rare Kidney Stones (ProRKS)
Phase N/A · 11 location(s) · Mayo Clinic · NCT02780297Walking Program in Fatty Liver Children With Phenylketonuria
NA · 1 location(s) · Cairo University · NCT06718842A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During P
Phase N/A · 4 location(s) · BioMarin Pharmaceutical · NCT05579548Ganciclovir Resistant/Refractory Cytomegalovirus Infection in SOT Recipients and HSCT Patients
Phase N/A · 23 location(s) · IRCCS Azienda Ospedaliero-Universitaria di Bologna · NCT05234723A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRN
Phase 1/Phase 2 · 22 location(s) · ModernaTX, Inc. · NCT05130437A Long-Term Study of JNT-517 in Participants With Phenylketonuria
Phase 3 · 12 location(s) · Otsuka Pharmaceutical Development & Commercialization, Inc. · NCT06628128GMP Powdered Substitutes in PKU and TYR
NA · 1 location(s) · Nutricia UK Ltd · NCT06941532Abbott Vascular Medical Device Registry
Phase N/A · 37 location(s) · Abbott Medical Devices · NCT04573660A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonu
Phase 1 · 4 location(s) · Agios Pharmaceuticals, Inc. · NCT07241234A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Ag
Phase 3 · 52 location(s) · Travere Therapeutics, Inc. · NCT06247085A Trial to Examine the Interaction of Repinatrabit With Ethinyl Estradiol/Norethindrone, Metformin,Carbamazepine, Rosuva
Phase 1 · 1 location(s) · Otsuka Pharmaceutical Development & Commercialization, Inc. · NCT07446400Orphan Europe Carbaglu® Surveillance Protocol
Phase N/A · 3 location(s) · Nicholas Ah Mew · NCT03409003Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
Phase N/A · 2 location(s) · National Human Genome Research Institute (NHGRI) · NCT02890342Allergy and Immunology Natural History Study
Phase N/A · 1 location(s) · Columbia University · NCT05584488
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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.