Cystic FibrosisPhase 1NCT05712538
Safety, Tolerability, and Pharmacokinetics of ARCT-032 in Healthy Adult Subjects and Adults With Cystic Fibrosis.
A Phase 1 study testing ARCT-032 in people with Cystic Fibrosis, run by Arcturus Therapeutics, Inc. at 1 site in New Zealand.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- ARCT-032
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 39 participants
- Where
- 1 siteNew Zealand
- Study dates
- 15 Feb 2023to 29 Jul 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
- Body weight between 40-100Kg and body mass index between 16-35 kg/m2
- 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
- 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.
- Phase 1b only: Subjects with CF on CFTR modulator therapy must be on a stable regimen for at least 2 months prior to screening.
Who cannot join
- History of illness or condition that might pose an additional risk or may confound study results.
- Pregnant or lactating (breast feeding)
- History of severe allergic reaction to a liposomal product
- Clinically significant abnormalities in Screening laboratory results
- Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B
- Treatment with another investigational drug, biological agent, or device within 30 days of screening, or 5 half-lives of investigational drug, whichever is longer
Show all 10Show fewer
- Drug or alcohol abuse within the past year
- 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
- Systemic or inhaled corticosteroids within 3 months prior to screening (Phase 1 only).
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official 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
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).
Read the detailed description
Phase 1 of this study is a single ascending dose, first-in-human study to determine the safety, tolerability, and pharmacokinetics (PK) of ARCT-032. After screening, healthy adult participants will be randomized 3:1 to inhale a single dose of nebulized ARCT-032 or placebo. There are 4 planned sequential dose cohorts. After dosing, participants will have follow-up assessments over a 4-week period.
Phase 1b in adults with cystic fibrosis will enroll after Phase 1 is completed and safety data are reviewed. Phase 1b is an open-label, two-dose study in adults with cystic fibrosis to assess the safety, tolerability, and pharmacokinetics of ARCT-032. After completion of screening, each participants will inhale two doses of nebulized ARCT-032 two days apart. Participants will have follow-up assessments over a 4-week period.
- Conditions
- Cystic Fibrosis
- Being tested
- ARCT-032, Placebo
- Keywords
- Cystic Fibrosis, CF
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARCT-032Drug
ARCT-032 is messenger RNA (mRNA) coding for cystic fibrosis transmembrane conductance regulator (CFTR) protein, formulated in a lipid nanoparticle (LNP).
PlaceboOther
Normal saline
| Study group | Role | What they receive |
|---|---|---|
| ARCT-032, Healthy Adults | Experimental | Ascending single doses of ARCT-032 administered to healthy adults via nebulizer Receives: Drug: ARCT-032 |
| Placebo, Healthy Adults | Placebo comparator | Single doses of 0.9% Saline administered to healthy adults via nebulizer Receives: Other: Placebo |
| ARCT-032,. Adults with Cystic Fibrosis | Experimental | Two doses of ARCT-032 administered to adults with Cystic Fibrosis via nebulizer Receives: Drug: ARCT-032 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, severity and dose-relationship of AEs
Safety and tolerability of ARCT-032 assessed by determining the incidence, severity and dose-relationship of AEs by dose
Measured: 4 weeks
Secondary outcomes (7)
Change in plasma area under the curve after single dose of ARCT-032
Area under the plasma concentration versus time curve (AUC) from time zero to the last quantifiable time point
Measured: Up to 2 Weeks
Maximum observed plasma concentration (Cmax) after single dose of ARCT-032
The maximum observed plasma concentration (Cmax)
Measured: Up to 2 Weeks
Time at which Cmax occurred after single dose of ARCT-032
The time at which Cmax occurred (Tmax)
Measured: Up to 2 Weeks
AUC0-inf after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032
AUC from time zero extrapolated to infinity
Measured: Up to 2 Weeks
T1/2 after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032
Terminal half-life
Measured: Up to 2 Weeks
CL after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032
Total body clearance, calculated as dose divided by AUC0-inf
Measured: Up to 2 Weeks
Vss after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032
Volume of distribution
Measured: Up to 2 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2023
- Study start15 Feb 2023
- Primary completion29 Jul 2024
- Study completion29 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Double-blind, placebo controlled (Phase 1 only)
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arcturus Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Novotech CRO
- Responsible party
- Sponsor
- Investigators
- Clinical Program Director, Study director, Arcturus Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05712538
- Sponsor's protocol ID
- ARCT-032-01
- Status last verified
- Nov 2024
- First submitted
- 26 Jan 2023
- Last updated
- 13 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT05712538
- Acronym / sponsor ID
- ARCT-032-01
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, CF
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Arcturus Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Novotech CRO
- Study start
- 15 Feb 2023 (actual)
- Primary completion
- 29 Jul 2024 (actual)
- Study completion
- 29 Jul 2024 (actual)
- First posted
- 3 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
Phase 1 of this study is a single ascending dose, first-in-human study to determine the safety, tolerability, and pharmacokinetics (PK) of ARCT-032. After screening, healthy adult participants will be randomized 3:1 to inhale a single dose of nebulized ARCT-032 or placebo. There are 4 planned sequential dose cohorts. After dosing, participants will have follow-up assessments over a 4-week period.
Phase 1b in adults with cystic fibrosis will enroll after Phase 1 is completed and safety data are reviewed. Phase 1b is an open-label, two-dose study in adults with cystic fibrosis to assess the safety, tolerability, and pharmacokinetics of ARCT-032. After completion of screening, each participants will inhale two doses of nebulized ARCT-032 two days apart. Participants will have follow-up assessments over a 4-week period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
- Model description
- Double-blind, placebo controlled (Phase 1 only)
| Arm | Type | Receives |
|---|---|---|
| ARCT-032, Healthy Adults | Experimental | Drug: ARCT-032Ascending single doses of ARCT-032 administered to healthy adults via nebulizer |
| Placebo, Healthy Adults | Placebo comparator | Other: PlaceboSingle doses of 0.9% Saline administered to healthy adults via nebulizer |
| ARCT-032,. Adults with Cystic Fibrosis | Experimental | Drug: ARCT-032Two doses of ARCT-032 administered to adults with Cystic Fibrosis via nebulizer |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
- Body weight between 40-100Kg and body mass index between 16-35 kg/m2
- 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
Key exclusion criteria (10 listed)
- History of illness or condition that might pose an additional risk or may confound study results.
- Pregnant or lactating (breast feeding)
- History of severe allergic reaction to a liposomal product
- Clinically significant abnormalities in Screening laboratory results
- Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ARCT-032ARCT-032 is messenger RNA (mRNA) coding for cystic fibrosis transmembrane conductance regulator (CFTR) protein, formulated in a lipid nanoparticle (LNP). | Drug | ARCT-032, Healthy Adults, ARCT-032,. Adults with Cystic Fibrosis |
| PlaceboNormal saline | Other | Placebo, Healthy Adults |
Primary outcome
Incidence, severity and dose-relationship of AEs
Time frame: 4 weeks
Safety and tolerability of ARCT-032 assessed by determining the incidence, severity and dose-relationship of AEs by dose
7 secondary outcomes
Change in plasma area under the curve after single dose of ARCT-032
Time frame: Up to 2 Weeks
Maximum observed plasma concentration (Cmax) after single dose of ARCT-032
Time frame: Up to 2 Weeks
Time at which Cmax occurred after single dose of ARCT-032
Time frame: Up to 2 Weeks
AUC0-inf after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032
Time frame: Up to 2 Weeks
T1/2 after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032
Time frame: Up to 2 Weeks
CL after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032
Time frame: Up to 2 Weeks
Vss after single dose (Phase 1) or two doses (Phase 1b) of ARCT-032
Time frame: Up to 2 Weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Nov 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Program Director study director
Arcturus Therapeutics
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ARCT-032 in people with Cystic Fibrosis, run by Arcturus Therapeutics, Inc. at 1 site in New Zealand.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence, severity and dose-relationship of AEs (time frame: 4 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Nov 2024.