Cystic FibrosisPhase 2LunairCFNCT06747858
Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis
A Phase 2 study testing ARCT-032 in people with Cystic Fibrosis and CFTR Gene Mutation, run by Arcturus Therapeutics, Inc. at 13 sites in United States.
Trial summary
ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy or are not taking CFTR modulators due to drug intolerance, poor response, or lack of access to modulators. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis, CFTR Gene Mutation
- What is being tested?
- ARCT-032
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 13 sitesUnited States
- Study dates
- 12 Dec 2024to Jun 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed diagnosis of Cystic Fibrosis
- Not eligible for CFTR modulator therapy or not taking CFTR modulators for at least 60 days prior to dosing (e.g. due to intolerance, poor response, or lack of access to modulators)
- FEV1 between 40% to 100% (cohorts 1-3) and 45% to 90% (cohort 4, inclusive) of predicted value for age, sex and height
Who cannot join
- History of illness or medical condition that might pose an additional risk or may confound study results
- Recent moderate or severe hemoptysis
- Recent major surgery
- Solid organ or hematologic transplant
- Requirement of supplemental oxygen while awake or > 2L per minute while sleeping.
- Chronic maintenance systemic corticosteroids exceeding equivalent of daily 15 mg oral prednisone or 30 mg every other day
- Adequate liver and kidney function as determined by lab tests
Trial locations and contacts
13 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Clinical Trial Disclosure Manager Central Email Box contact
13 sites in 1 country
United States13 sites, 12 recruiting
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
- Recruiting
Medical University of South Carolina
Charleston, South Carolina, 29425
- Recruiting
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
- Recruiting
Joe DiMaggio Children's Hospital
Hollywood, Florida, 33021
- Recruiting
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205
- Recruiting
Children's Hospital of Los Angeles
Los Angeles, California, 90027
- Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
- Active, not recruiting
The Cystic Fibrosis Institute
Northfield, Illinois, 60093
- Recruiting
UT Health San Antonio
San Antonio, Texas, 78229
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
- Recruiting
Washington University School of Medicine
St Louis, Missouri, 63144
- Recruiting
University of Arizona
Tucson, Arizona, 85724
- Not yet recruiting
Children's National Hospital
Washington D.C., District of Columbia, 20010
About this study
Official title: A Phase 2, Open-label, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability and Efficacy of ARCT-032 in People With Cystic Fibrosis
ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy or are not taking CFTR modulators due to drug intolerance, poor response, or lack of access to modulators.
Read the detailed description
This is an open-label, multiple-ascending dose study of ARCT-032 in adults with CF who are not on CFTR modulator therapy. After successful screening, eligible participants enrolled in cohorts 1-3 will receive nebulized ARCT-032 daily for 4 weeks and then will be followed for safety for a total of 12 weeks. In cohort 4, eligible participants will receive nebulized ARCT-032 for 12 weeks and will then be followed for safety for an additional 12 weeks.
- Conditions
- Cystic Fibrosis, CFTR Gene Mutation
- Being tested
- ARCT-032
- Keywords
- CFTR, mRNA
- 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-032Biological
CFTR mRNA formulated in lipid nanoparticles
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Dose Level A of ARCT-032, inhaled daily for 28 days Receives: Biological: ARCT-032 |
| Cohort 2 | Experimental | Dose Level B of ARCT-032, inhaled daily for 28 days Receives: Biological: ARCT-032 |
| Cohort 3 | Experimental | Dose Level C of ARCT-032, inhaled daily for 28 days Receives: Biological: ARCT-032 |
| Cohort 4 | Experimental | Dose level B or C of ARCT-032, inhaled once daily for 12 weeks Receives: Biological: 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 adverse events
Safety and tolerability of ARCT-032 assess by determining incidence, severity and dose-relationship of AEs by dose level
Measured: Cohorts 1-3: 16 weeks and Cohort 4: 24 weeks
Secondary outcomes (5)
Pharmacokinetics - Plasma Concentration
Plasma concentration of ARCT-032 mRNA and lipid components
Measured: Up to 6 weeks
Pharmacodynamics--Lung Function
Change from baseline in FEV1
Measured: 4 weeks
Pharmacodynamics--Cystic Fibrosis Quality of Life Questionnaire-Revised (CFQ-R)
Change from baseline in CFQ-R RSS (Respiratory Symptoms Scale) Score. Overall CFQ-R score ranges from 0-100, with higher scores indicating better health.
Measured: 4 weeks
High Resolution Computed Chromatography
Changes from baseline to end of treatment in chest-HRCT-scan scores (e.g., airway wall thickness, air trapping, and mucus plugging scores)
Measured: 12 weeks
EQ-5D-5L (Cohort 4 only)
Change from baseline to end of treatment (Day 84) in EQ-5D-5L questionnaire scores
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2024
- Study start12 Dec 2024
- Primary completionJun 2027 (estimated)
- Study completionJun 2027 (estimated)
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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Multiple ascending dose
- Masking
- None (open label)
- Enrolment
- 33 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Arcturus Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06747858
- Sponsor's protocol ID
- ARCT-032-02
- Acronym
- LunairCF
- Status last verified
- May 2026
- First submitted
- 17 Dec 2024
- Last updated
- 8 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-label, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability and Efficacy of ARCT-032 in People With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT06747858
- Acronym / sponsor ID
- LunairCF / ARCT-032-02
- Conditions
- Cystic Fibrosis, CFTR Gene Mutation
- Keywords
- CFTR, mRNA
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 33 participants (estimated)
- Lead sponsor
- Arcturus Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 12 Dec 2024 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 24 Dec 2024 (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.
ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy or are not taking CFTR modulators due to drug intolerance, poor response, or lack of access to modulators.
Detailed description
This is an open-label, multiple-ascending dose study of ARCT-032 in adults with CF who are not on CFTR modulator therapy. After successful screening, eligible participants enrolled in cohorts 1-3 will receive nebulized ARCT-032 daily for 4 weeks and then will be followed for safety for a total of 12 weeks. In cohort 4, eligible participants will receive nebulized ARCT-032 for 12 weeks and will then be followed for safety for an additional 12 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- Multiple ascending dose
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: ARCT-032Dose Level A of ARCT-032, inhaled daily for 28 days |
| Cohort 2 | Experimental | Biological: ARCT-032Dose Level B of ARCT-032, inhaled daily for 28 days |
| Cohort 3 | Experimental | Biological: ARCT-032Dose Level C of ARCT-032, inhaled daily for 28 days |
| Cohort 4 | Experimental | Biological: ARCT-032Dose level B or C of ARCT-032, inhaled once daily for 12 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Confirmed diagnosis of Cystic Fibrosis
- Not eligible for CFTR modulator therapy or not taking CFTR modulators for at least 60 days prior to dosing (e.g. due to intolerance, poor response, or lack of access to modulators)
- FEV1 between 40% to 100% (cohorts 1-3) and 45% to 90% (cohort 4, inclusive) of predicted value for age, sex and height
Key exclusion criteria (7 listed)
- History of illness or medical condition that might pose an additional risk or may confound study results
- Recent moderate or severe hemoptysis
- Recent major surgery
- Solid organ or hematologic transplant
- Requirement of supplemental oxygen while awake or > 2L per minute while sleeping.
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-032CFTR mRNA formulated in lipid nanoparticles | Biological | Cohort 1, Cohort 2, Cohort 3, Cohort 4 |
Primary outcome
Incidence, severity and dose relationship of adverse events
Time frame: Cohorts 1-3: 16 weeks and Cohort 4: 24 weeks
Safety and tolerability of ARCT-032 assess by determining incidence, severity and dose-relationship of AEs by dose level
5 secondary outcomes
Pharmacokinetics - Plasma Concentration
Time frame: Up to 6 weeks
Pharmacodynamics--Lung Function
Time frame: 4 weeks
Pharmacodynamics--Cystic Fibrosis Quality of Life Questionnaire-Revised (CFQ-R)
Time frame: 4 weeks
High Resolution Computed Chromatography
Time frame: 12 weeks
EQ-5D-5L (Cohort 4 only)
Time frame: 12 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2026.
- 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.
Clinical Trial Disclosure Manager Central Email Box contact
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ARCT-032 in people with Cystic Fibrosis and CFTR Gene Mutation, run by Arcturus Therapeutics, Inc. at 13 sites in United States.
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 3 inclusion and 7 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: looking for participants now, as of the sponsor's last update on 8 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trial Disclosure Manager Central Email Box (858-900-2660, CFclinicaltrials@arcturusrx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence, severity and dose relationship of adverse events (time frame: Cohorts 1-3: 16 weeks and Cohort 4: 24 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 8 May 2026.