OTC Deficiency

Clinical trial pipeline · Data from ClinicalTrials.gov

See which OTC Deficiency trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 11 trials

RecruitingPhase 1/2

NCT06255782

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzym…

United States20 participantsStarted 2024-04-08

Treatment: ECUR-506

RecruitingNot Applicable

NCT06864273

Assessing Pharmacy Technician Educational Training for the Provision of Over-the-Counter Hearing Aids in Rural Alabama a…

Hearing loss is a major public health concern due to its negative association with emotional well-being, cognition, and physical ailments, such as diabetes. Acc…

United States144 participantsStarted 2025-07-25

Treatment: Pharmacy Technician Educational Training

RecruitingNot Applicable

NCT04908319

Hepatic Histopathology in Urea Cycle Disorders

This is a multi-site, retrospective chart review as well as a prospective study to evaluate histopathologic findings in liver samples from individuals with any…

United States70 participantsStarted 2022-02-24

RecruitingNot Applicable

NCT04573660

Abbott Vascular Medical Device Registry

The AV-MDR is a prospective, non-randomized, open-label, multi-center registry. The purpose of the AV-MDR study is to proactively collect and evaluate clinical…

United States3,784 participantsStarted 2020-10-25

Treatment: Coronary and peripheral stents, Pacing catheters

RecruitingNot Applicable

NCT02132741

Optical Coherence Tomography And NEphropathy: The OCTANE Study

Patients with high blood pressure (hypertension) and chronic kidney disease are at an increased risk of developing heart disease and strokes. Part of this risk…

United Kingdom150 participantsStarted 2014-05-16

RecruitingPhase 2

NCT06488313

A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD

Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.

United States9 participantsStarted 2024-11-04

Treatment: ARCT-810

All recent trials (60 shown)

Active — Not RecruitingNot Applicable

NCT04612764

Liver Disease in Urea Cycle Disorders

This is a multi-center, cross-sectional study to assess risk for liver fibrosis and hepatic injury in individuals with urea cycle disorders (UCDs) using serum b…

United States62 participantsStarted 2021-11-04

Active — Not RecruitingPhase 1

NCT06247670

Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype

The objective of this clinical study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple ascending doses of C…

Australia, Netherlands120 participantsStarted 2024-02-05

Treatment: CMP-CPS-001, Placebo

OTC Deficiency

Trial phase breakdown

Trial status

Actively recruiting — 11 trials

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

Assessing Pharmacy Technician Educational Training for the Provision of Over-the-Counter Hearing Aids in Rural Alabama a…

Hepatic Histopathology in Urea Cycle Disorders

Abbott Vascular Medical Device Registry

Optical Coherence Tomography And NEphropathy: The OCTANE Study

A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD

All recent trials (60 shown)

Liver Disease in Urea Cycle Disorders

Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype

Trial phase breakdown

Trial status

Actively recruiting — 11 trials

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

Assessing Pharmacy Technician Educational Training for the Provision of Over-the-Counter Hearing Aids in Rural Alabama a…

Hepatic Histopathology in Urea Cycle Disorders

Abbott Vascular Medical Device Registry

Optical Coherence Tomography And NEphropathy: The OCTANE Study

A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD

All recent trials (60 shown)

Liver Disease in Urea Cycle Disorders

Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype

Looking for VUS to Confirm Dominant Wolfram-like Syndrome Instead of Recessive Wolfram Syndrome

Outside the Cage (OTC) Robotic Esophagectomy

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

Epiretinal Membrane in Patients With DR.

Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-…

Radiomic Analysis in OCT Imaging of Acquired Vitelliform Lesions.

Baby Detect : Genomic Newborn Screening

Chuvashia Inappropriate Prescribing Study

Effect of Fixed Combination Citicoline Homotaurine and Pyrroloquinoline Quinone on Pattern-electroretinogram in Glaucoma

Role of OCT & OCT Angiography in Patients With Posterior at the Uveitis Clinic of Assiut University Hospital.

Early Check: Expanded Screening in Newborns

AI Quantitative Coronary Angiography Versus Optical Coherent Tomography Guidance for Coronary Stent Implantation

Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine…

Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)

Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI

Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects

Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan