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5 recruiting studies matching your filters

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudi…

Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase Deficiency DiseaseUrea Cycle Disorders, Inborn

Estimated

~$10 – $1,050

iECURE, Inc.NCT06805695

Phase 1

Master Protocol for a Phase I/II Open-label Safety and Efficacy Study of LNP.UCD.ABE, a Lipid Nanoparticle-delivered Base Editing Therapy, in Patients With Urea Cycle Disorders Due to Variants Amenable to Corrective Editing by LNP.UCD.ABE

This is a single-site Phase 1/2 open-label umbrella clinical trial designed to evaluate the safety, tolerability, and efficacy of a single intravenous dose of …

Urea Cycle DisordersCarbamoyl-Phosphate Synthase I Deficiency

Estimated

~$10 – $7,500

Rebecca Ahrens-NicklasNCT07667387

Observational

Pilot Study of At Home Ammonia Monitoring in Patients With an Inborn Error of Ammonia Metabolism

The goal of this observational study is to learn if people with certain ammonia metabolism disorders will measure their ammonia levels at home. The main quest…

Urea Cycle DisordersOrganic AcidemiasFatty Acid Oxidation Disorder+1 more

Estimated

~$10 – $8,750

Sequitur Health Corp.NCT06953505

Longitudinal Study of Urea Cycle Disorders

Urea cycle disorders (UCD) are a group of rare inherited metabolism disorders. Infants and children with UCD commonly experience episodes of vomiting, lethargy…

Brain Diseases, Metabolic, InbornAmino Acid Metabolism, Inborn ErrorsUrea Cycle Disorders

Estimated

~$10 – $1,125

Andrea GropmanNCT00237315

Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders

In proximal urea cycle disorders (UCD), particularly ornithine transcarbamylase deficiency (OTCD), hyperammonemia (HA) causes increased brain glutamine (Gln) w…

Urea Cycle Disorders

Estimated

~$10 – $675

Children's National Research InstituteNCT02935283