Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects

NCT04416126 | Completed Phase 1 FDA Drug

• 1 other identifier: ARCT- 810-01
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in healthy adult subjects.

Conditions

Ornithine Transcarbamylase Deficiency

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (2)

• Number of participants with changes in plasma pharmacokinetic parameters after single dose of ARCT-810

  Up to 15 days

• Number of participants with changes in urine pharmacokinetic parameters after single dose of ARCT-810

  Up to 24 hours

Study Arms (2)

ARCT-810

EXPERIMENTAL

Ascending single doses of ARCT-810 administered intravenously

Biological: ARCT-810

Placebo

PLACEBO COMPARATOR

Single doses of 0.9% Saline administered intravenously

Other: Placebo

Interventions

ARCT-810 BIOLOGICAL

ARCT-810 is an investigational medicinal product comprising OTC mRNA formulated in a lipid nanoparticle (LNP) under development.

ARCT-810

Placebo OTHER

The placebo for this study is 0.9% sterile saline.

Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Healthy males or females aged 18 to 65 at the time of informed consent.
• Body weight ≤ 100Kg and body mass index \<35 kg/m2
• Willing to refrain from strenuous exercise/activity (for example heavy lifting, weight training, intense aerobics classes etc.) and alcohol for at least 72 hours prior to study visits and willing to refrain from taking protein supplements for the duration of the study.
• Willing and able to comply with protocol-defined procedures and complete all study visits
• Males must be surgically sterile or, if engaged in sexual relations with a female of child-bearing potential, the subject must be using an acceptable contraceptive method from the time of signing the informed consent form until at least 30 days after the last dose of Study Drug. Females: must be non-pregnant and non-lactating and either: i. surgically sterile or ii. post-menopausal

You may not qualify if:

• Clinically significant abnormalities in medical history
• Screening laboratory results as follows:
• ALT, AST, GGT, total bilirubin or alkaline phosphatase, \> ULN.
• Random blood glucose and/or HbA1c \> ULN
• Hemoglobin \< LLN
• Platelet count \< 100x109/L
• Estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73m2 calculated by Modification to Diet in Renal Disease \[MDRD\] study equation.
• Urine protein:creatinine ratio (UPCR) \> 50 mg/mmol
• 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

Sponsors & Collaborators

• Arcturus Therapeutics, Inc.: lead

Study Sites (1)

Auckland Clinical Studies (ACS) Ltd.

Auckland, New Zealand

Location

MeSH Terms

Conditions

Ornithine Carbamoyltransferase Deficiency Disease

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Christian Schwabe, MD

  Auckland Clinical Studies (ACS) Ltd.

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Double blinded, Placebo Controlled
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 28, 2020
• First Posted: June 4, 2020
• Study Start: June 1, 2020
• Primary Completion: December 9, 2020
• Study Completion: December 9, 2020
• Last Updated: November 8, 2023

Record last verified: 2023-11

Locations

NZ (1)