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Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in healthy adult subjects.
This is a single ascending dose study of ARCT-810 in which approximately 30 subjects are planned to be enrolled. The length of each study participant is approximately 8 weeks from screening to last study visit.
Study participants will be allocated to one of the five different study groups (also called cohorts), to test different doses of ARCT-810. There will be 6 participants in each group. Within each cohort, subjects will be randomized 2:1 to receive ARCT-810 or placebo as an IV infusion.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ARCT-810 | Experimental | Ascending single doses of ARCT-810 administered intravenously |
|
| Placebo | Placebo Comparator | Single doses of 0.9% Saline administered intravenously |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ARCT-810 | Biological | ARCT-810 is an investigational medicinal product comprising OTC mRNA formulated in a lipid nanoparticle (LNP) under development. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence, severity and dose-relationship of AEs | Safety and tolerability of ARCT-810 assessed by determining the incidence, severity and dose-relationship of AEs by dose | 4 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with changes in plasma pharmacokinetic parameters after single dose of ARCT-810 | The plasma pharmacokinetics (concentration-time results) of ARCT-810 will be assessed following single dose | Up to 15 days |
| Number of participants with changes in urine pharmacokinetic parameters after single dose of ARCT-810 |
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Inclusion Criteria:
Exclusion Criteria:
Clinically significant abnormalities in medical history
Screening laboratory results as follows:
Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed within 7 days prior to Study Day 1
Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B
Uncontrolled hypertension (BP > 160/100 mm Hg)
Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational drug, whichever is longer
Recent (within 1 year) history of, or current drug or alcohol abuse
Have any other conditions, which, in the opinion of the Investigator or Sponsor would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the Study
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| Name | Affiliation | Role |
|---|---|---|
| Christian Schwabe, MD | Auckland Clinical Studies (ACS) Ltd. | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Auckland Clinical Studies (ACS) Ltd. | Auckland | New Zealand |
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| ID | Term |
|---|---|
| D020163 | Ornithine Carbamoyltransferase Deficiency Disease |
| ID | Term |
|---|---|
| D056806 | Urea Cycle Disorders, Inborn |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
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Double blinded, Placebo Controlled
| Placebo | Other | The placebo for this study is 0.9% sterile saline. |
|
The urine pharmacokinetics (concentration-time results) of ARCT-810 will be assessed following single dose |
| Up to 24 hours |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |