A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12+
SponsorArcturus Therapeutics, Inc.
About this trial
Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.
Eligibility criteria
Qualifiers
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- ARCT-810
Treatment groups
9 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov