Trial intelligence / Profile preview

ARCT-810 phase 2 in adolescent and adult participants with ornithine transcarbamylase deficiency

2021-001081-38Phase 2Completed

Sponsor
Arcturus Therapeutics
Start
Aug 2021
Modality
Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems
Countries
1
01

Overview

Phase 2, randomised, double-blind trial in ornithine transcarbamylase deficiency and urea cycle disorder (UCD) comparing ARCT-810 with Placebo (2 arms). Primary endpoint: Safety and tolerability. Started Aug 2021; status: completed.

02

Drugs

ARCT-810
03

Conditions

ornithine transcarbamylase deficiencyurea cycle disorder (UCD)

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