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

Primary Purpose

Ornithine Transcarbamylase Deficiency

Status

Completed

Phase

Phase 1

Locations

New Zealand

Study Type

Interventional

Intervention

ARCT-810

Placebo

About this trial

This is an interventional treatment trial for Ornithine Transcarbamylase Deficiency

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesAccepts Healthy Volunteers

Inclusion Criteria:

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

Exclusion Criteria:

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
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

Sites / Locations

Auckland Clinical Studies (ACS) Ltd.

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

ARCT-810

Placebo

Arm Description

Ascending single doses of ARCT-810 administered intravenously

Single doses of 0.9% Saline administered intravenously

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

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

The urine pharmacokinetics (concentration-time results) of ARCT-810 will be assessed following single dose

Full Information

NCT ID

NCT04416126

First Posted

May 28, 2020

Last Updated

April 6, 2023

Sponsor

Arcturus Therapeutics, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04416126

Brief Title

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

Official 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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Completed

Study Start Date

June 1, 2020 (Actual)

Primary Completion Date

December 9, 2020 (Actual)

Study Completion Date

December 9, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Arcturus Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Yes

5. Study Description

Brief Summary

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

Detailed Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Ornithine Transcarbamylase Deficiency

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Masking Description

Double blinded, Placebo Controlled

Allocation

Randomized

Enrollment

30 (Actual)

8. Arms, Groups, and Interventions

Arm Title

ARCT-810

Arm Type

Experimental

Arm Description

Ascending single doses of ARCT-810 administered intravenously

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Single doses of 0.9% Saline administered intravenously

Intervention Type

Biological

Intervention Name(s)

ARCT-810

Intervention Description

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

Intervention Type

Other

Intervention Name(s)

Placebo

Intervention Description

The placebo for this study is 0.9% sterile saline.

Primary Outcome Measure Information:

Title

Incidence, severity and dose-relationship of AEs

Description

Safety and tolerability of ARCT-810 assessed by determining the incidence, severity and dose-relationship of AEs by dose

Time Frame

4 weeks

Secondary Outcome Measure Information:

Title

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

Description

The plasma pharmacokinetics (concentration-time results) of ARCT-810 will be assessed following single dose

Time Frame

Up to 15 days

Title

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

Description

The urine pharmacokinetics (concentration-time results) of ARCT-810 will be assessed following single dose

Time Frame

Up to 24 hours

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 Exclusion Criteria: 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 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Christian Schwabe, MD

Organizational Affiliation

Auckland Clinical Studies (ACS) Ltd.

Official's Role

Principal Investigator

Facility Information:

Facility Name

Auckland Clinical Studies (ACS) Ltd.

City

Auckland

Country

New Zealand

12. IPD Sharing Statement

Learn more about this trial

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

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