Evaluating ARCT-810 for treating OTC deficiency

A Phase 2a, Open-label, Multiple Ascending Dose Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency

This study is testing a new treatment called ARCT-810 to see if it can help adolescents and adults with OTC deficiency feel better while they continue their usual care.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 9 (estimated)
• Ages: 12 Years and up
• Sex: All
• Sponsor: Arcturus Therapeutics, Inc. (industry)
• Locations: 1 site (Chevy Chase, Maryland)
• Trial ID: NCT06488313 on ClinicalTrials.gov

What this trial studies

This Phase 2a, open-label study evaluates the safety and pharmacodynamics of ARCT-810 in adolescents and adults aged 12 and older with OTC deficiency. Participants will undergo a diet stabilization period before receiving ARCT-810 every two weeks for up to five doses. The study includes multiple clinic visits for monitoring and assessment, while participants continue their current management for OTC deficiency. Dose escalation or cohort expansion may occur based on initial participant responses.

Who should consider this trial

Good fit: Ideal candidates are males and females aged 12 years and older with a documented diagnosis of OTC deficiency and stable clinical management.

Not a fit: Patients with uncontrolled hypertension or recent infections may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve the management of OTC deficiency and reduce the risk of hyperammonemia in affected patients.

How similar studies have performed: While this approach is being evaluated in this specific context, similar studies targeting metabolic disorders have shown promise in the past.

Eligibility criteria

Inclusion Criteria:

1. Willingness and ability to comply with all the protocol requirements, complete all study visits and sign informed consent.
2. Males and Females aged ≥12 years, at Screening.
3. Documented clinical diagnosis of OTC deficiency.
4. History of symptomatic hyperammonemia or elevated plasma ammonia or glutamine with clinical stability for at least 1 month prior to Screening.
5. Medically managed for OTC deficiency and receiving a stable protein-restricted diet, dietary supplements, and/or ammonia scavenger regimen (if applicable) for at least 28 days.
6. Good general health with no clinically significant abnormal findings that would interfere with study procedures (including plasma ammonia within participant's historical range).
7. Must be willing to adhere to contraception guidelines.

Exclusion Criteria:

1. Uncontrolled hypertension.
2. Symptoms of infection for at least 7 days prior to dosing.
3. Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
4. History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 2 years.
5. History of any organ transplant.
6. History of severe allergic reaction to a liposomal or PEG-containing product.
7. History of congenital or acquired cardiac disorders.
8. Abuse of medications, illicit drugs or alcohol.
9. Blood donation of 50 to 499 mL within 30 days of screening or of \>499 mL within 60 days of screening.
10. Clinically significant laboratory abnormalities on screening labs including INR \>1.5, eGFR\< 60 mL/min/1.73m2 or positive test results for HIV, HBV, or HCV.
11. Inadequately controlled diabetes.
12. Clinically significant anemia.
13. Changes in maintenance therapies for OTC deficiency with 28 days prior to dosing.
14. Medical history requiring continuous or intermittent systemic corticosteroid administration.
15. Receipt of inhibitors of urea synthesis or drugs that significantly affect renal clearance.
16. Recent treatment with another investigational drug, biological agent, or device.
17. Treatment with any oligonucleotide (siRNA or mRNA) within 6 months prior to screening. COVID-19 vaccines are not exclusionary.
18. Involved in study conduct or an immediate family member of an individual involved in the study.
19. Participated in another dosing cohort of the study.
20. Any other conditions, in the opinion of the investigator, that would interfere with participation.

Where this trial is running

Chevy Chase, Maryland

• Uncommon Cures — Chevy Chase, Maryland, United States (RECRUITING)

Conditions: OTC Deficiency, Ornithine Transcarbamylase Deficiency, OTCD