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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/NCT05712538




Registration number
NCT05712538
Ethics application status
Date submitted
26/01/2023
Date registered
3/02/2023
Date last updated
4/12/2023

Titles & IDs
Public title
Safety, Tolerability, and Pharmacokinetics of ARCT-032 in Healthy Adult Subjects and Adults With Cystic Fibrosis.
Scientific title
A Study in Two Parts: (Phase 1) A Randomized, Double-blinded, Placebo--controlled, Single-ascending-dose Study in Healthy Adult Subjects and (Phase 1b) an Open-label, Nested, Divided-dose Study in Adults With Cystic Fibrosis to Assess the Safety, Tolerability, and Pharmacokinetics of ARCT-032
Secondary ID [1] 0 0
ARCT-032-01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ARCT-032
Other interventions - Placebo

Experimental: ARCT-032, Healthy Adults - Ascending single doses of ARCT-032 administered to healthy adults via nebulizer

Placebo Comparator: Placebo, Healthy Adults - Single doses of 0.9% Saline administered to healthy adults via nebulizer

Experimental: ARCT-032,. Adults with Cystic Fibrosis - Two doses of ARCT-032 administered to adults with Cystic Fibrosis via nebulizer


Treatment: Drugs: ARCT-032
ARCT-032 is messenger RNA (mRNA) coding for cystic fibrosis transmembrane conductance regulator (CFTR) protein, formulated in a lipid nanoparticle (LNP).

Other interventions: Placebo
Normal saline
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 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
Change in plasma area under the curve after single dose of ARCT-032
Timepoint [1] 0 0
Up to 2 Weeks
Secondary outcome [2] 0 0
Maximum observed plasma concentration (Cmax) after single dose of ARCT-032
Timepoint [2] 0 0
Up to 2 Weeks
Secondary outcome [3] 0 0
Time at which Cmax occurred after single dose of ARCT-032
Timepoint [3] 0 0
Up to 2 Weeks
Secondary outcome [4] 0 0
AUC0-inf after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032
Timepoint [4] 0 0
Up to 2 Weeks
Secondary outcome [5] 0 0
T1/2 after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032
Timepoint [5] 0 0
Up to 2 Weeks
Secondary outcome [6] 0 0
CL after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032
Timepoint [6] 0 0
Up to 2 Weeks
Secondary outcome [7] 0 0
Vss after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032
Timepoint [7] 0 0
Up to 2 Weeks

Eligibility
Key inclusion criteria
Key

1. Phase 1: Healthy males or females aged 18 to 65 years at the time of informed consent.

Phase 1b: Males or females aged 18 to 65 years with confirmed diagnosis of CF
documented in subject's medical record

2. Body weight between 40-100Kg and body mass index between 16-35 kg/m2

3. Phase 1: Forced expiratory volume (FEV1) at screening >85% of predicted value for age,
sex, and height.

Phase 1b: FEV1 at screening between 50% and 100% of predicted value

4. Surgically sterile or 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.

5. Phase 1b only: Subjects with CF on CFTR modulator therapy must be on a stable regimen
for at least 2 months prior to screening.

Key
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. History of illness or condition that might pose an additional risk or may confound
study results.

2. Pregnant or lactating (breast feeding)

3. History of severe allergic reaction to a liposomal product

4. Clinically significant abnormalities in Screening laboratory results

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

6. Treatment with another investigational drug, biological agent, or device within 30
days of screening, or 5 half-lives of investigational drug, whichever is longer

7. Drug or alcohol abuse within the past year

8. History of moderate to heavy smoking or vaping (>10 cigarettes/sessions per day)
within 6 months prior to the dose of study drug. Participants must be willing to
refrain from smoking or vaping within 1 week of dosing through Day 15

9. Systemic or inhaled corticosteroids within 3 months prior to screening (Phase 1 only).

10. 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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
15/02/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/05/2024
Actual
Sample size
Target
38
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Christchurch

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Arcturus Therapeutics, Inc.
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Novotech CRO
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-032 in
healthy adult subjects (Phase 1) and of two doses in Adults with Cystic Fibrosis (Phase 1b).
Trial website
https://clinicaltrials.gov/ct2/show/NCT05712538
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Program Director
Address 0 0
Arcturus Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Trial Disclosure Manager
Address 0 0
Country 0 0
Phone 0 0
(858) 900-2660
Fax 0 0
Email 0 0
clinicaltrials@arcturusrx.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05712538