ARCT-810-04

Exploring Innovative Treatments

Ornithine Transcarbamylase (OTC) Deficiency Research in Adolescent and Adult Patients

Study Drug:

ARCT-810

Age Group:

Adolescents and adults 12 years of age or older with confirmed OTC deficiency

Study Duration:

Approximately 16 weeks (including a screening period)

Visits:

About 13 visits over the 16-week study period (includes screening, 5 infusion visits, and follow-up assessments)

Location:

Uncommon Cures, Chevy Chase, MD

Compensation

Those who qualify receive all study-related laboratory tests, medical exams, and travel at no cost.

Study Overview

Uncommon Cures is conducting a clinical trial for adolescents and adults (12 years of age and older) with Ornithine Transcarbamylase (OTC) deficiency, a rare genetic disorder that impairs the body’s ability to process excess ammonia. This study is designed to test the safety and potential efficacy of an investigational mRNA drug, ARCT-810, developed by Arcturus Therapeutics, Inc. The trial takes place at our Chevy Chase, MD location. Participants will receive all study-related tests, medical exams, and travel at no cost.

Eligibility Criteria:

Age: 12 years and older
Diagnosis: Confirmed Ornithine Transcarbamylase (OTC) deficiency
Health History: Evidence of ammonia or glutamine elevations
Current Therapies: Must be on stable OTC deficiency therapy and/or dietary interventions
Other Requirements: Additional medical or study-specific criteria may apply

Study Process

Screening: A review of medical history, current condition, and eligibility.
Enrollment & Baseline Assessments: Participants complete physical exams, lab tests, and other evaluations before treatment begins.
Treatment Period: Five ARCT-810 infusions administered every two weeks over approximately 10 weeks (no placebo).
Follow-Up Evaluations: Additional visits for safety monitoring, lab tests, and clinical assessments (total of about 13 visits over a 16-week period).

Advance Rare Treatments.

Have a Question?

FAQ

We understand that participating in a clinical trial can raise a lot of questions. Here, you’ll find answers to some of the most common questions about our process, what to expect, and how we support our patients. If you don’t see the information you’re looking for, feel free to contact us directly for more details.

What is ARCT-810, and how does it work?

ARCT-810 is an investigational mRNA-based therapy designed to help the body produce the ornithine transcarbamylase (OTC) enzyme. It uses Arcturus Therapeutics’ LUNAR® lipid nanoparticle technology to deliver mRNA to the liver, where it may help restore urea cycle function in individuals with OTC deficiency.

Will I need to stay overnight for this study?

No, overnight stays are not required. However, you will have approximately 13 study visits over a 16-week period, including screenings, five infusion visits, and follow-up assessments.

What kind of follow-up care will I receive after the trial?

Participants will have follow-up study visits to monitor safety, track biomarkers, and evaluate treatment response. The study team will remain in contact with participants for any necessary medical support.

How is patient safety monitored during the trial?

Patient safety is the top priority. The study follows a rigorous protocol approved by regulatory authorities and an Ethics Committee to ensure participant well-being. Safety assessments, lab tests, and clinical evaluations will be conducted regularly throughout the study.

Can I speak to my doctor before enrolling?

Absolutely! We encourage all potential participants to discuss the trial with their healthcare provider before enrolling to ensure it aligns with their ongoing medical care.