Clinical Trials Logo

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


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

See also
  Status Clinical Trial Phase
Completed NCT01569568 - Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Terminated NCT04717453 - Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency
Recruiting NCT05910151 - Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Recruiting NCT04612764 - Liver Disease in Urea Cycle Disorders
Active, not recruiting NCT04442347 - Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency Phase 1
Not yet recruiting NCT02670889 - Urease Inhibitor Drug Treatment for Urea Cycle Disorders Phase 1/Phase 2
Recruiting NCT05092685 - Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn Phase 1/Phase 2
Completed NCT00472732 - Neurologic Injuries in Adults With Urea Cycle Disorders N/A
Terminated NCT04909346 - Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
Completed NCT04416126 - Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects Phase 1
Completed NCT00718627 - Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders Phase 2
Recruiting NCT04908319 - Hepatic Histopathology in Urea Cycle Disorders
Withdrawn NCT03767270 - Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency Phase 1/Phase 2
Completed NCT04269122 - A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects
Recruiting NCT06255782 - An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency (OTC-HOPE) Phase 1/Phase 2