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New mRNA drug hopes to help cystic fibrosis patients who Can't use current therapies

NCT ID NCT06747858

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 33 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2024

Expected to finish

Jun 2027

An estimate. End dates often move.

Lead sponsor

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

18 years and older

Sex

Anyone

Healthy volunteers

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

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.

  1. The places running it

    12 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Children's Hospital of Los Angeles

    RECRUITING

    Los Angeles, California, 90027, United States

  • Children's National Hospital

    NOT_YET_RECRUITING

    Washington D.C., District of Columbia, 20010, United States

  • Cleveland Clinic Foundation

    RECRUITING

    Cleveland, Ohio, 44195, United States

  • Joe DiMaggio Children's Hospital

    RECRUITING

    Hollywood, Florida, 33021, United States

  • Medical University of South Carolina

    RECRUITING

    Charleston, South Carolina, 29425, United States

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105, United States

  • The Cystic Fibrosis Institute

    ACTIVE_NOT_RECRUITING

    Northfield, Illinois, 60093, United States

  • UT Health San Antonio

    RECRUITING

    San Antonio, Texas, 78229, United States

  • University of Arizona

    RECRUITING

    Tucson, Arizona, 85724, United States

  • University of Arkansas for Medical Sciences

    RECRUITING

    Little Rock, Arkansas, 72205, United States

  • Vanderbilt University Medical Center

    RECRUITING

    Nashville, Tennessee, 37232, United States

  • Washington University School of Medicine

    RECRUITING

    St Louis, Missouri, 63144, United States

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Other studies related to the condition(s) this trial covers.