Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency
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ClinicalTrials.gov Identifier: NCT04442347 |
Recruitment Status :
Recruiting
First Posted : June 22, 2020
Last Update Posted : October 8, 2020
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Condition or disease | Intervention/treatment | Phase |
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Ornithine Transcarbamylase Deficiency | Biological: ARCT-810 Other: Placebo | Phase 1 |
This is a single ascending dose study of ARCT-810 in which approximately 12 (up to a maximum of 20) subjects are planned to be enrolled. The length of each study participant is approximately 8 weeks from screening to last study visit. The study comprises an up to 4-week Screening and a 4-week Diet Run-in period to run concurrently followed by a 1-day dosing period and a 28-day Post Treatment Period.
Study participants will be allocated to one of the three different study groups (also called cohorts), to test different doses of ARCT-810. There will be 4 participants in each group. Within each cohort, subjects will be randomized 3 :1 to receive ARCT-810 or placebo as an IV infusion.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 12 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Double (Participant, Investigator) |
Masking Description: | Double Blinded, Placebo Controlled |
Primary Purpose: | Treatment |
Official Title: | A Phase 1b Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Clinically Stable Patients With Ornithine Transcarbamylase Deficiency |
Estimated Study Start Date : | October 2020 |
Estimated Primary Completion Date : | November 2021 |
Estimated Study Completion Date : | December 2021 |

Arm | Intervention/treatment |
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Experimental: ARCT-810
Ascending single doses of ARCT-810 administered intravenously
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Biological: ARCT-810
ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) mRNA formulated in a lipid nanoparticle (LNP) under development. |
Placebo Comparator: Placebo
Single doses of 0.9% Saline administered intravenously
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Other: Placebo
The placebo for this study is 0.9% sterile saline. |
- Incidence, severity and dose-relationship of adverse events (AEs) [ Time Frame: 4 weeks ]Safety and tolerability of ARCT-810 assessed by determining the incidence, severity and dose-relationship of AEs by dose
- Change in area under the curve after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]Area under the plasma concentration versus time curve (AUC) from time zero to the last quantifiable time point
- Maximum observed plasma concentration (Cmax) after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]The maximum observed plasma concentration (Cmax)
- Time at which Cmax occurred after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]The time at which Cmax occurred (Tmax)
- AUC0-inf after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]AUC from time zero extrapolated to infinity
- AUCExtrap after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]The relative portion of AUC0-inf extrapolated beyond AUC0-t
- T1/2 after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]Terminal half-life
- MRT0-inf after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]The mean residence time extrapolated to infinity
- CL after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]Total body clearance, calculated as dose divided by AUC0-inf
- Vss after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]Volume of distribution

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Males and females ≥18 years of age with documented diagnosis of late onset OTC deficiency confirmed with genetic testing.
- Subject's Ornithine Transcarbamylase deficiency (OTCD) is stable as evidenced by a) no clinical symptoms of hyperammonemia and b) an ammonia level <100 µmol/L (170 µg/dL) at the Screening evaluation. Subjects must remain free from symptoms of hyperammonemia throughout the screening period.
- If using nitrogen ammonia scavenger therapy, must be on a stable regimen for ≥ 28 days prior to signing informed consent and must remain on the same regimen throughout the screening period
- Must have maintained a stable protein restricted diet +/- amino acid supplementation for at least 28 days prior to signing informed consent and continue to maintain a stable diet for the duration of the study
- No clinically significant abnormal findings on medical history, clinical laboratory test results (other than ammonia) vital sign measurements, 12-lead ECG results, or physical examination
- Males must be surgically sterile or willing to use adequate contraception; females must be post-menopausal, surgically sterile or willing to use adequate contraception
Exclusion Criteria:
- History of clinically significant disease(s) (other than OTCD)
- Abnormal hepatic enzymes, significant renal impairment, clinically significant anemia or uncontrolled diabetes
- Blood Pressure greater than 160/100 mm Hg
- Malignancy within 5 years prior to study
- Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational drug, whichever is longer
- Gene therapy within 1 year prior to screening
- Prior organ transplant
- Positive viral serology test results for HIV type 1 or 2 antibodies, hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) antibody

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04442347
Contact: Steve Hughes, MD | 858-900-2660 | steveh@arcturusrx.com | |
Contact: Manely Yafeh | 858-900-2660 | Manely@arcturusrx.com |
United States, Texas | |
University of Texas Southwestern Medical Center at Dallas | Recruiting |
Dallas, Texas, United States, 75390 |
Responsible Party: | Arcturus Therapeutics, Inc. |
ClinicalTrials.gov Identifier: | NCT04442347 |
Other Study ID Numbers: |
ARCT- 810-02 |
First Posted: | June 22, 2020 Key Record Dates |
Last Update Posted: | October 8, 2020 |
Last Verified: | October 2020 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Ornithine Carbamoyltransferase Deficiency Disease Urea Cycle Disorders, Inborn Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases |
Nervous System Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Amino Acid Metabolism, Inborn Errors Metabolism, Inborn Errors Metabolic Diseases |