RecruitingPhase 2

A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD

United States9 participantsStarted 2024-11-04

Plain-language summary

Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.

Age range12 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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.

✕. 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.

Incidence, severity and dose-relationship of adverse events (AEs)

Timeframe: Day 85

NCT IDNCT06488313

SponsorArcturus Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-06-01

Who can participate

Age range12 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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.

✕. 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.