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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04416126
Recruitment Status : Completed
First Posted : June 4, 2020
Last Update Posted : December 24, 2020
Sponsor:
Information provided by (Responsible Party):
Arcturus Therapeutics, Inc.

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

Condition or disease Intervention/treatment Phase
Ornithine Transcarbamylase Deficiency Biological: ARCT-810 Other: Placebo Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Masking Description: Double blinded, Placebo Controlled
Primary Purpose: Treatment
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
Actual Study Start Date : June 1, 2020
Actual Primary Completion Date : December 9, 2020
Actual Study Completion Date : December 9, 2020


Arm Intervention/treatment
Experimental: ARCT-810
Ascending single doses of ARCT-810 administered intravenously
Biological: ARCT-810
ARCT-810 is an investigational medicinal product comprising OTC mRNA formulated in a lipid nanoparticle (LNP) under development.

Placebo Comparator: Placebo
Single doses of 0.9% Saline administered intravenously
Other: Placebo
The placebo for this study is 0.9% sterile saline.




Primary Outcome Measures :
  1. Incidence, severity and dose-relationship of AEs [ Time Frame: 4 weeks ]
    Safety and tolerability of ARCT-810 assessed by determining the incidence, severity and dose-relationship of AEs by dose


Secondary Outcome Measures :
  1. Number of participants with changes in plasma pharmacokinetic parameters after single dose of ARCT-810 [ Time Frame: Up to 15 days ]
    The plasma pharmacokinetics (concentration-time results) of ARCT-810 will be assessed following single dose

  2. Number of participants with changes in urine pharmacokinetic parameters after single dose of ARCT-810 [ Time Frame: Up to 24 hours ]
    The urine pharmacokinetics (concentration-time results) of ARCT-810 will be assessed following single dose



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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04416126


Locations
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New Zealand
Auckland Clinical Studies (ACS) Ltd.
Auckland, New Zealand
Sponsors and Collaborators
Arcturus Therapeutics, Inc.
Investigators
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Principal Investigator: Christian Schwabe, MD Auckland Clinical Studies (ACS) Ltd.
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Responsible Party: Arcturus Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04416126    
Other Study ID Numbers: ARCT- 810-01
First Posted: June 4, 2020    Key Record Dates
Last Update Posted: December 24, 2020
Last Verified: December 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Ornithine Carbamoyltransferase Deficiency Disease
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
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases