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Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

Ornithine Transcarbamylase Deficiency Clinical Trial

Official title:
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

Clinical Trial Summary

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

Clinical Trial 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 a 4-6-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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05526066
Study type Interventional
Source Arcturus Therapeutics, Inc.
Contact David Geller, MD
Phone 224-727-7636
Email davidg@arcturusrx.com
Status Recruiting
Phase Phase 2
Start date July 6, 2022
Completion date June 2024
View Details
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