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ARCT-810

Phase 2

OTC Deficiency | Monoclonal antibody | Rare Disease |Arcturus Therapeutics Holdings Inc.|Last Updated: Mar 17, 2025

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment9

FDA Designations

No designations recorded

Clinical trial landscape

ARCT-810 · 3 trials · 3 indications

Phase 2 1Phase 1 2
NCT06488313A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCDOTC Deficiency
RECRUITING9 Analytics
PHASE2RECRUITING
A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
OTC DeficiencyUnlock trial analytics

Study Endpoints

Primary Endpoints

Incidence, severity and dose-relationship of adverse events (AEs)
Day 85

Safety and tolerability of ARCT-810 assessed by incidence, severity, and dose-relationship of AEs

Incidence, severity and dose-relationship of AEs
4 weeks

Safety and tolerability of ARCT-810 assessed by determining the incidence, severity and dose-relationship of AEs by dose

Secondary Endpoints

Stable isotope ureagenesis assay values (AUC of first isotope)
Up to Day 85
Stable isotope ureagenesis assay values (AUC of second isotope)
Up to Day 85
Fasting plasma ammonia
Up to Day 85
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ARCT-810EXPERIMENTALParticipants will receive up to 5 IV infusions of ARCT-810 administered at 14-day intervals.
PlaceboPLACEBO_COMPARATORSingle doses of 0.9% Saline administered intravenously

Interventions

NameTypeDescription
ARCT-810BIOLOGICALARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) messenger RNA (mRNA) formulated in a lipid nanoparticle (LNP).
PlaceboOTHERThe placebo for this study is 0.9% sterile saline.
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Eligibility Criteria

Age Range12 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Willingness and ability to comply with all the protocol requirements, complete all study visits and sign informed consent. 2. Males and Females aged ≥12 years, at Screening. 3. Documented clinical diagnosis of OTC deficiency. 4. History of symptomatic hyperammonemia or elevat...

Countries:United StatesNew Zealand
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Frequently asked questions about ARCT-810

What is ARCT-810 used for?

ARCT-810 is an investigational drug being developed for the treatment of Ornithine Transcarbamylase Deficiency (OTC Deficiency), a rare genetic disorder. It is currently in Phase 2 clinical development for this condition.

What does ARCT-810 target?

ARCT-810 is a monoclonal antibody being developed by Arcturus Therapeutics. The specific molecular target of ARCT-810 has not been disclosed in the available clinical trial information.

Who makes ARCT-810?

ARCT-810 is being developed by Arcturus Therapeutics Holdings Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol ARCT.

What phase is ARCT-810 in?

ARCT-810 is currently in Phase 2 clinical development. It has completed two Phase 1 trials and is now being evaluated in a Phase 2 study for Ornithine Transcarbamylase Deficiency.

What clinical trials is ARCT-810 in?

ARCT-810 has been studied in three clinical trials. Two Phase 1 trials (NCT04416126 and NCT04442347) are completed, and one Phase 2 trial (NCT06488313) is currently recruiting participants with OTC Deficiency.

Is ARCT-810 the same as any other drug?

ARCT-810 is not known by any alternative names. It is a distinct investigational drug being developed by Arcturus Therapeutics for the treatment of Ornithine Transcarbamylase Deficiency.