NCT05526066

Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Arcturus Therapeutics, Inc. · Industry
• Sex: All
• Age: 12 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The primary objective is to evaluate the safety and tolerability of repeated doses of intravenously administered ARCT-810.

Detailed description

This study is a Phase 2, randomized, placebo-controlled study of ARCT-810 in people living with OTC deficiency 12 years of age and older. After an at least 4 week screening and diet stabilization period, participants will be randomized 3:1 to receive ARCT-810 or placebo. Following the first dose and safety evaluation, participants will receive up to an additional 5 doses of ARCT-810 or placebo, each separated by 14 days. The treatment period is followed by a 12-week observation period.

Conditions

• Ornithine Transcarbamylase Deficiency
• OTC Deficiency
• OTCD

Interventions

Type	Name	Description
BIOLOGICAL	ARCT-810	ARCT-810 is messenger RNA (mRNA) coding for Ornithine Transcarbamylase (OTC) formulated in a lipid nanoparticle (LNP).
OTHER	Placebo	Normal Saline

Timeline

• Start date: 2022-10-17
• Primary completion: 2024-10-31
• Completion: 2024-10-31
• First posted: 2022-09-02
• Last updated: 2025-09-26

Locations

14 sites across 6 countries: Belgium, France, Italy, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study