New mRNA drug hopes to help cystic fibrosis patients who Can't use current therapies
NCT ID NCT06747858
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests an experimental mRNA medicine called ARCT-032 in 33 adults with cystic fibrosis who cannot take or do not benefit from current CFTR modulators. The goal is to see if the drug is safe and can improve lung function and quality of life. Participants will receive multiple doses, and researchers will monitor side effects and measure changes in breathing tests.
Why investors are watching
Arcturus Therapeutics is testing ARCT-032 in 33 adults with cystic fibrosis who cannot use standard CFTR modulator drugs. For a small company, this Phase 2 readout matters because a positive safety and efficacy signal could validate its drug candidate and support further development.
If it works: If ARCT-032 shows good safety and improves lung function in these patients, Arcturus could advance the drug to a larger trial and strengthen its pipeline value.
If it fails: Phase 2 trials often fail to meet their goals, and a weak result or delay could set back the program and hurt the company's prospects.
AI-written from the trial record. Speculative, and not investment advice.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 33 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Dec 2024
- Expected to finish
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Jun 2027
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Key Inclusion Criteria: 1. Confirmed diagnosis of Cystic Fibrosis 2. 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) 3. FEV1 between 40% to 100% (cohorts 1-3) and 45% to 90% (cohort 4, inclusive) of predicted value for age, sex and height Exclusion Criteria: 1. History of illness or medical condition that might pose an additional risk or may confound study results 2. Recent moderate or severe hemoptysis 3. Recent major surgery 4. Solid organ or hematologic transplant 5. Requirement of supplemental oxygen while awake or \> 2L per minute while sleeping. 6. Chronic maintenance systemic corticosteroids exceeding equivalent of daily 15 mg oral prednisone or 30 mg every other day 7. Adequate liver and kidney function as determined by lab tests
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
12 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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Children's Hospital of Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
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Children's National Hospital
NOT_YET_RECRUITINGWashington D.C., District of Columbia, 20010, United States
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Cleveland Clinic Foundation
RECRUITINGCleveland, Ohio, 44195, United States
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Joe DiMaggio Children's Hospital
RECRUITINGHollywood, Florida, 33021, United States
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Medical University of South Carolina
RECRUITINGCharleston, South Carolina, 29425, United States
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Seattle Children's Hospital
RECRUITINGSeattle, Washington, 98105, United States
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The Cystic Fibrosis Institute
ACTIVE_NOT_RECRUITINGNorthfield, Illinois, 60093, United States
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UT Health San Antonio
RECRUITINGSan Antonio, Texas, 78229, United States
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University of Arizona
RECRUITINGTucson, Arizona, 85724, United States
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University of Arkansas for Medical Sciences
RECRUITINGLittle Rock, Arkansas, 72205, United States
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Vanderbilt University Medical Center
RECRUITINGNashville, Tennessee, 37232, United States
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Washington University School of Medicine
RECRUITINGSt Louis, Missouri, 63144, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can Device-Preserved donor lungs boost transplant success?
- Can a simple questionnaire reveal why some cystic fibrosis patients skip physiotherapy?
- Cystic fibrosis diabetes: do gut hormones and genes hold the key?
- Can a special lung scan catch cystic fibrosis damage earlier than standard tests?
- Can a pill replace the liquid? testing a new form of cystic fibrosis drug
- Triple therapy under the microscope: does it transform cystic fibrosis care?