Arcturus is conducting multiple studies to determine whether ARCT-810 can address the root cause of OTC deficiency and lessen the disease burden experienced by those living with the condition.
ARCT-810-04 (US)
Phase 2a multiple dose study of ARCT-810 in 12 years of age and older with OTC deficiency
The goal of the ARCT-810-04 study is to evaluate the safety and tolerability of ARCT-810 in people with OTC deficiency and its efficacy in restoring urea cycle function.
Now Enrolling
Female or male patients ages 12 years and above with confirmed OTCD*
Uncommon Cures | Chevy Chase, MD
Learn more
about this study on ClinicalTrials.gov
(NCT06488313)
This study is ~16-18 weeks in duration from start to finish.
Weeks 1–4
Screening & Diet Assessment
4-6 weeks
Weeks 5–12
Dosing
8 weeks
Weeks 13–18
Follow-up
4-6 weeks
Screening and Diet Assessment
During the 4- to 6-week screening period, study staff will collect information and perform blood tests, review diet and medications, and perform other clinical tests to determine eligibility for the study.
Study Drug Dosing Period
There is no placebo in this study. If eligible, participants will receive the first intravenous (IV) infusion of ARCT-810, along with additional tests during and after the infusion. Then, additional infusions may be given every two weeks up to 4 times at one of the three dose levels. Throughout the study, several assessments and tests will be performed to measure the safety of ACRT-810, urea cycle function, and ureagenesis.
Follow-Up Period
After completion of the dosing period, additional visits will occur over 4-6 weeks for tests and follow-up.
Additional study details will be provided by the study staff.
*Must understand the study requirements and sign a consent form to participate.
Those who qualify will receive all study-related laboratory tests, medical exams, and travel at no cost.
