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

NCT ID: NCT04416126

Last Updated: 2023-11-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-06-01

Study Completion Date

2020-12-09

Brief Summary

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

Detailed Description

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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.

Conditions

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Ornithine Transcarbamylase Deficiency

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators
Double blinded, Placebo Controlled

Study Groups

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ARCT-810

Ascending single doses of ARCT-810 administered intravenously

Group Type EXPERIMENTAL

ARCT-810

Intervention Type BIOLOGICAL

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

Placebo

Single doses of 0.9% Saline administered intravenously

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

The placebo for this study is 0.9% sterile saline.

Interventions

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ARCT-810

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

Intervention Type BIOLOGICAL

Placebo

The placebo for this study is 0.9% sterile saline.

Intervention Type OTHER

Eligibility Criteria

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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

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Arcturus Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Christian Schwabe, MD

Role: PRINCIPAL_INVESTIGATOR

Auckland Clinical Studies (ACS) Ltd.

Locations

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Auckland Clinical Studies (ACS) Ltd.

Auckland, , New Zealand

Site Status

Countries

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New Zealand

Other Identifiers

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ARCT- 810-01

Identifier Type: -

Identifier Source: org_study_id

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