Actively Recruiting
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
Led by Arcturus Therapeutics, Inc. · Updated on 2025-03-17
9
Participants Needed
1
Research Sites
13 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and pharmacodynamics of multiple doses of ARCT-810, an investigational drug, in adolescents and adults with Ornithine Transcarbamylase (OTC) deficiency. This Phase 2a, open-label study aims to better understand how ARCT-810 works and its safety profile in people aged 12 years and older who have this condition. Participants must have a documented diagnosis of OTC deficiency and be medically managed with a stable diet and supplements. Participants will receive up to five intravenous infusions of ARCT-810 every two weeks, following a diet stabilization period of at least four weeks. The study includes three different dose levels, and dose escalation or cohort expansion may occur after three participants complete each dose level. Participants will continue their usual clinical management for OTC deficiency throughout the study. During the study, participants will have clinic visits at screening and on Days 1, 15, 29, 36, 43, 57, 60, 71, and 85. Researchers will monitor adverse events, pharmacokinetics, fasting plasma ammonia, plasma glutamine, and stable isotope ureagenesis assay values. The primary outcome is the incidence, severity, and dose relationship of adverse events by Day 85. The total participation duration includes ongoing assessments up to Day 85 to evaluate safety and drug effects.
CONDITIONS
Brief 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 and complete all study visits
- 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 that would interfere with study procedures
- Willingness to follow contraception guidelines
You will not qualify if you...
- Uncontrolled hypertension
- Symptoms of infection for at least 7 days prior to dosing
- Malignancy within 5 years except certain successfully treated skin or cervical cancers
- History of any OTC gene therapy or liver-derived stem cell therapy within 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 lab abnormalities including INR >1.5, eGFR <60 mL/min/1.73m2, or positive HIV, HBV, or HCV tests
- 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 therapy within 6 months prior to screening (COVID-19 vaccines excluded)
- Involvement in study conduct or immediate family member involvement
- Prior participation in another dosing cohort of this study
- Any other conditions that may interfere with participation, as per investigator opinion
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 10 weeks
Participants receive up to 5 intravenous infusions of ARCT-810 every two weeks while remaining on their current clinical management for OTC deficiency.
Visits at Days 1, 15, 29, 36, 43, 57, 60, 71, and 85
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
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