NCT06488313 · Arcturus Therapeutics, Inc.
A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
What this study is about
Evaluate the safety and how the drug affects the body of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.
View original scientific description
Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.
Interventions
BIOLOGICAL
ARCT-810
ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) messenger RNA (mRNA) formulated in a lipid nanoparticle (LNP).
Primary outcome measures
Incidence, severity and dose-relationship of adverse events (AEs)
Time frame: Day 85
Safety and tolerability of ARCT-810 assessed by incidence, severity, and dose-relationship of AEs
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Willingness and ability to comply with all the protocol requirements, complete all study visits and sign informed consent.
- Males and Females aged ≥12 years, 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 and receiving a stable protein-restricted diet, dietary supplements, and/or ammonia scavenger regimen (if applicable) for at least 28 days.
- Good general health with no clinically significant abnormal findings that would interfere with study procedures (including plasma ammonia within participant's historical range).
- Must be willing to adhere to contraception guidelines.
Exclusion criteria
- Uncontrolled hypertension.
- Symptoms of infection for at least 7 days prior to dosing.
- 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.
- History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 2 years.
- History of any organ transplant.
- History of severe allergic reaction to a liposomal or PEG-containing product.
- History of congenital or acquired cardiac disorders.
- Abuse of medications, illicit drugs or alcohol.
- Blood donation of 50 to 499 mL within 30 days of screening or of \>499 mL within 60 days of screening.
- 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.
- Inadequately controlled diabetes.
- Clinically significant anemia.
- Changes in maintenance therapies for OTC deficiency with 28 days prior to dosing.
- Medical history requiring continuous or intermittent systemic corticosteroid administration.
- Receipt of inhibitors of urea synthesis or drugs that significantly affect 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 are not exclusionary.
- Involved in study conduct or an immediate family member of an individual involved in the study.
- Participated in another dosing cohort of the study.
- Any other conditions, in the opinion of the investigator, that would interfere with participation.
Where
- Chevy Chase, Maryland
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 17, 2025 · Source of record for eligibility and locations