NCT04442347 - Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

November 3, 2020

Primary Completion Date

August 30, 2023

Study Completion Date

April 30, 2024

Conditions

Ornithine Transcarbamylase Deficiency

Interventions

BIOLOGICAL

ARCT-810

ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) mRNA formulated in a lipid nanoparticle (LNP) under development.

OTHER

Placebo

The placebo for this study is 0.9% sterile saline.

Trial Locations (8)

10029

The Mount Sinai Hospital, New York

15224

Children's Hospital of Pittsburgh, Pittsburgh

32608

University of Florida, Gainesville

53226

Children's Wisconsin - Milwaukee Hospital, Milwaukee

55454

M Health Fairview Masonic Children's Hospital, Minneapolis

75390

University of Texas Southwestern Medical Center at Dallas, Dallas

76706

Baylor University, Waco

84112

University of Utah, Salt Lake City