Status:
RECRUITING
A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
Lead Sponsor:
Arcturus Therapeutics, Inc.
Conditions:
OTC Deficiency
Ornithine Transcarbamylase Deficiency
Eligibility:
All Genders
12+ years
Phase:
PHASE2
Brief Summary
Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.
Detailed Description
This a Phase 2a, open-label study of ARCT-810 in participants 12 years of age and older living with OTC deficiency. After a diet stabilization period of at least 4 weeks, all participants will be enro...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
November 4 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 1 2026
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT06488313
Start Date
November 4 2024
End Date
September 1 2026
Last Update
March 17 2025
Active Locations (1)
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1
Uncommon Cures
Chevy Chase, Maryland, United States, 20815