At Arcturus, we strive to do something incredible everyday
Click the video below to learn more about our ongoing ARCT-810 clinical studies for OTC deficiency and the work Arcturus is doing to provide meaningful treatment options for people living with genetic conditions.
Kelsey Bobka, OTCD Carrier and Parent
Arcturus is conducting two OTC deficiency clinical studies. The aim of the studies is to learn more about our investigational treatment (ARCT-810) for people living with OTC deficiency.
ARCT-810-02 (US) – Fully enrolled
Phase 1B study of single doses of ARCT-810 in adults 18 years and older with clinically stable OTC deficiency
ARCT-810-03 (UK, EU)
Phase 2 study of repeated doses of ARCT-810 in 12- to 65-year-olds with OTC deficiency
The goal of the ARCT-810-02 study is to evaluate the safety and tolerability of single doses of ARCT-810 in adults with OTC deficiency.
The study will also measure the levels of ARCT-810 in the body over time (PK) and will explore whether a single dose of ARCT-810 can temporarily increase OTC activity in the body.
Fully Enrolled
Adults 18 years and older with confirmed OTC deficiency who are clinically stable
United States
New York, NY
Pittsburgh, PA
Gainesville, FL
Dallas, TX
Houston, TX
Minneapolis, MN
Milwaukee, WI
Salt Lake City, UT
Learn more
about this study on ClinicalTrials.gov
(NCT04442347)
This study is ~8 weeks in duration from start to finish.
Week 1
Week 2
Week 3
Week 4
Screening & Diet Assessment
4 weeks
Week 5
Dosing
1 Day
Week 6
Week 7
Week 8
Follow-up
4 weeks
Screening and Diet Assessment
During the 4-week screening period, study staff will collect information and perform blood tests, review diet and medications, and perform other clinical tests to determine if you are qualified to participate.
Study Drug Dosing Period
If eligible for the study, a urea cycle function test will be performed that takes about 4 hours to complete. After the screening period, a single intravenous (IV) dose of either ARCT-810 or placebo will be given. Then, continued observation at the study site will occur for about 4 hours.
Follow-Up Period
The day after the infusion, a urea cycle function test will be repeated. Then, there are 4 short follow-up visits to the study site over 4 weeks for clinical tests, blood work, dietitian sessions, and ammonia tests.
Additional study details will be provided by the study staff.
The goal of the ARCT-810-03 study is to evaluate the safety and tolerability of six doses of ARCT-810 in adolescents and adults with OTC deficiency. This study will measure whether repeated doses of ARCT-810 can increase the OTC activity and can improve urea cycle function in the body.
The study will also measure the levels of ARCT-810 in the body over time (PK).
Recruiting
People between 12–65 years of age with confirmed OTC deficiency†
United Kingdom
Spain
Belgium
France
Sweden
Italy
Learn more
about this study on ClinicalTrials.gov
(NCT05526066)
†Must understand the study requirements and sign a consent form to participate.
*Sites are located at the following institutions:
- UNITED KINGDOM
- London: Great Ormond Street Hospital for Children, University College London Hospitals;
- Birmingham: Queen Elizabeth Hospital Birmingham
- Salford: Coming soon
- SPAIN
- Barcelona: Hospital Clínic de Barcelona, Hospital Sant Joan de Déu;
- Madrid: Hospital Universitario 12 de Octubre;
- Santiago de Compostela: Complejo Hospitalario Universitario de Santiago (CHUS)
- BELGIUM
- Brussels: Cliniques Universitaires Saint-Luc
- FRANCE
- Paris: Hôpital Necker-Enfants Malades;
- Marseilles: Coming soon
- SWEDEN
- Stockholm: Karolinska Universitetssjukhuset
- ITALY
- Padova: Coming soon;
- Rome: Coming soon
This study is ~28–30 weeks in duration from start to finish.
Weeks 1–4
Screening & Diet Assessment
4-6 Weeks
Overnight
Testing
Weeks 5–16
Dosing Period
(Infusion every 2 weeks)
12 Weeks
Overnight
Testing
Weeks 17–28
Follow-up
12 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 of the study.
Study Drug Dosing Period
If eligible, participants will receive the first intravenous (IV) infusion of either ARCT-810 or placebo, along with additional tests during and after the infusion. Then, additional infusions may be given every 14 days up to 5 times. Throughout the study, several tests to measure safety and effectiveness of the study medicine will be performed. After the first and sixth infusion, participants are required to stay overnight for testing.
Follow-Up Period
After completion of the dosing period, additional visits will occur over 12 weeks (about 3 months) for tests and follow-up.
Additional study details will be provided by the study staff.
