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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04416126




Registration number
NCT04416126
Ethics application status
Date submitted
28/05/2020
Date registered
4/06/2020
Date last updated
8/11/2023

Titles & IDs
Public title
Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects
Scientific title
A Phase 1 Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Healthy Adult Subjects
Secondary ID [1] 0 0
ARCT- 810-01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Ornithine Transcarbamylase Deficiency 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - ARCT-810
Other interventions - Placebo

Experimental: ARCT-810 - Ascending single doses of ARCT-810 administered intravenously

Placebo Comparator: Placebo - Single doses of 0.9% Saline administered intravenously


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

Other interventions: Placebo
The placebo for this study is 0.9% sterile saline.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence, severity and dose-relationship of AEs
Timepoint [1] 0 0
4 weeks
Secondary outcome [1] 0 0
Number of participants with changes in plasma pharmacokinetic parameters after single dose of ARCT-810
Timepoint [1] 0 0
Up to 15 days
Secondary outcome [2] 0 0
Number of participants with changes in urine pharmacokinetic parameters after single dose of ARCT-810
Timepoint [2] 0 0
Up to 24 hours

Eligibility
Key inclusion criteria
1. Healthy males or females aged 18 to 65 at the time of informed consent.

2. Body weight = 100Kg and body mass index <35 kg/m2

3. 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.

4. Willing and able to comply with protocol-defined procedures and complete all study
visits

5. 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
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. Clinically significant abnormalities in medical history

2. 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

3. Active infection requiring systemic antiviral or antimicrobial therapy that will not
be completed within 7 days prior to Study Day 1

4. Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C
or chronic hepatitis B

5. Uncontrolled hypertension (BP > 160/100 mm Hg)

6. Treatment with another investigational drug, biological agent, or device within one
month of screening, or 5 half-lives of investigational drug, whichever is longer

7. Recent (within 1 year) history of, or current drug or alcohol abuse

8. 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
9/12/2020
Sample size
Target
Accrual to date
Final
30
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Arcturus Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in
healthy adult subjects.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04416126
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Christian Schwabe, MD
Address 0 0
Auckland Clinical Studies (ACS) Ltd.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries