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ARCT-810 · 3 trials · 3 indications
Safety and tolerability of ARCT-810 assessed by incidence, severity, and dose-relationship of AEs
Safety and tolerability of ARCT-810 assessed by determining the incidence, severity and dose-relationship of AEs by dose
| Arm | Type | Description |
|---|---|---|
| ARCT-810 | EXPERIMENTAL | Participants will receive up to 5 IV infusions of ARCT-810 administered at 14-day intervals. |
| Placebo | PLACEBO_COMPARATOR | Single doses of 0.9% Saline administered intravenously |
| Name | Type | Description |
|---|---|---|
| ARCT-810 | BIOLOGICAL | ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) messenger RNA (mRNA) formulated in a lipid nanoparticle (LNP). |
| Placebo | OTHER | The placebo for this study is 0.9% sterile saline. |
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...
ARCT-810 is an investigational therapy being developed for ornithine transcarbamylase deficiency, or OTCD, a rare inherited urea cycle disorder. It is designed to address the underlying enzyme deficiency. Arcturus Therapeutics is evaluating it in clinical trials, including a Phase 2 study in participants with OTCD.
ARCT-810 targets ornithine transcarbamylase, the enzyme encoded by the OTC gene that is deficient in OTCD. By targeting OTC, the therapy aims to restore or replace the missing enzyme activity in patients. The drug is being developed as a treatment for this rare metabolic disorder.
ARCT-810 is being developed by Arcturus Therapeutics Holdings Inc., which trades on the Nasdaq under the ticker ARCT. The company is advancing the candidate for ornithine transcarbamylase deficiency and holds orphan drug, fast track, and rare pediatric disease designations from the FDA for the program.
ARCT-810 is in Phase 2 clinical development. A Phase 2 study evaluating pharmacodynamics and safety in participants with OTCD is currently recruiting. Earlier Phase 1 and Phase 1b studies in healthy adults and stable adult OTCD patients have been completed. The drug remains investigational and has not been approved by the FDA.
ARCT-810 has been studied in three registered trials. NCT06488313 is a recruiting Phase 2 study in participants with OTCD. NCT04442347 was a completed Phase 1b study in stable adults with OTCD, and NCT04416126 was a completed Phase 1 study in healthy adult volunteers. Total enrollment across these studies is 46 participants.