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
This trial does not accept healthy volunteersQualifiers
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
Design model
Sequential
Treatments tested in this trial
ARCT-810
Biological/VaccineARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) messenger RNA (mRNA) formulated in a lipid nanoparticle (LNP).
Treatment groups
9 Participants
are divided into 1 treatment groupGroup A: ARCT-810Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Incidence, severity and dose-relationship of adverse events (AEs)
Safety and tolerability of ARCT-810 assessed by incidence, severity, and dose-relationship of AEs
Time frame
Day 85Secondary outcomes
1
Stable isotope ureagenesis assay values (AUC of first isotope)
Change from Baseline in stable isotope ureagenesis assay values (AUC of first isotope) following multiple doses of ARCT-810
Time frame
Up to Day 852
Stable isotope ureagenesis assay values (AUC of second isotope)
Change from Baseline in stable isotope ureagenesis assay values (AUC of second isotope) following mulitple doses of ARCT-810
Time frame
Up to Day 853
Fasting plasma ammonia
Proportion of participants maintaining normal morning fasting plasma ammonia
Time frame
Up to Day 854
Plasma Glutamine
Change from Baseline in plasma glutamine and labeled glutamine as available
Time frame
Up to Day 85Sponsors and contacts
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Source ClinicalTrials.gov