Actively Recruiting
A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
Led by Arcturus Therapeutics, Inc. · Updated on 2025-03-17
9
Participants Needed
1
Research Sites
95 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.
CONDITIONS
Official Title
A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Willingness and ability to comply with all protocol requirements, complete all study visits, and sign informed consent
- Males and females aged 12 years or older at screening
- Documented clinical diagnosis of OTC deficiency
- History of symptomatic hyperammonemia or elevated plasma ammonia or glutamine with clinical stability for at least 1 month prior to screening
- Medically managed for OTC deficiency with a stable protein-restricted diet, dietary supplements, and/or ammonia scavenger regimen for at least 28 days
- Good general health with no clinically significant abnormal findings interfering with study procedures, including plasma ammonia within participant's historical range
- Willingness to adhere to contraception guidelines
You will not qualify if you...
- Uncontrolled hypertension
- Symptoms of infection within 7 days prior to dosing
- Malignancy within 5 years, except successfully treated basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix
- History of any OTC gene therapy or liver-derived stem cell therapy within the past 2 years
- History of any organ transplant
- History of severe allergic reaction to liposomal or PEG-containing products
- History of congenital or acquired cardiac disorders
- Abuse of medications, illicit drugs, or alcohol
- Blood donation of 50 to 499 mL within 30 days or more than 499 mL within 60 days prior to screening
- Clinically significant laboratory abnormalities including INR >1.5, eGFR <60 mL/min/1.73m2, or positive tests for HIV, HBV, or HCV
- Inadequately controlled diabetes
- Clinically significant anemia
- Changes in maintenance therapies for OTC deficiency within 28 days prior to dosing
- Medical history requiring continuous or intermittent systemic corticosteroid use
- Receipt of inhibitors of urea synthesis or drugs affecting renal clearance
- Recent treatment with another investigational drug, biological agent, or device
- Treatment with any oligonucleotide (siRNA or mRNA) within 6 months prior to screening (COVID-19 vaccines allowed)
- Involvement in study conduct or immediate family member involvement
- Participation in another dosing cohort of this study
- Any other conditions that may interfere with participation according to the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Uncommon Cures
Chevy Chase, Maryland, United States, 20815
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here