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New inhaled therapy for cystic fibrosis shows early promise

NCT ID NCT03375047

Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tested an inhaled drug called MRT5005 in 42 adults with cystic fibrosis. The goal was to check safety and see if it could improve lung function. Participants received either the drug or a placebo by nebulizer. The study is completed, and results will help decide if larger trials are warranted.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
MRT5005 (a drug given by inhalation)
What this could lead to
If successful, this could point toward a new inhaled treatment to improve lung function in people with cystic fibrosis.
What could go wrong
This is an early phase 1/2 trial with only 42 participants, so results may not confirm effectiveness or safety for wider use. The drug may cause side effects or fail to show meaningful benefit.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

42 people

The number who actually took part.

Started

May 2018

Finished

Mar 2022

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.

Inclusion Criteria: * Confirmed diagnosis of CF as defined by both of the following: * Two CF disease-causing cystic fibrosis transmembrane conductance regulator (CFTR) mutations in Class I or II (genotype confirmed at the screening visit). * Chronic sinopulmonary disease and/or gastrointestinal/nutritional abnormalities consistent with CF disease. * Clinically stable CF disease, as judged by the investigator. * Forced expiratory volume in 1 second (FEV1) ≥50% and ≤90% of the predicted normal for age, gender, and height at screening. * Resting oxygen saturation ≥92% on room air (pulse oximetry). Exclusion Criteria: * An acute upper or lower respiratory infection, pulmonary exacerbation, or clinically significant episode of hemoptysis or change in chronic respiratory medications (including antibiotics) for CF lung disease within 28 days prior to dosing with investigational product on Day 1. * Participants were receiving treatment with ivacaftor monotherapy (KALYDECO). * Parts A and B only: Were receiving treatment with triple combination therapy (TRIKAFTA). * Participants with a Class III, IV, or V CFTR gene mutation in at least 1 allele. * Infection with highly virulent bacteria associated with accelerated decline in pulmonary function and/or decreased survival (e.g., Burkholderia cenocepacia, Burkholderia dolosa, Mycobacterium abscessus). Treatment with ORKAMBI or SYMDEKO was not an exclusion for this study.

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Conditions

The condition(s) this trial relates to.

cystic fibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Cystic fibrosis

Contacts and locations

Locations

  • Johns Hopkins University

    Baltimore, Maryland, 21287, United States

  • Maine Medical Center

    Portland, Maine, 04102, United States

  • National Jewish Health

    Denver, Colorado, 80206, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • New Orleans Center for Clinical Research

    Knoxville, Tennessee, 37920, United States

  • Northwestern University

    Chicago, Illinois, 60611, United States

  • Oregon Health and Sciences University

    Portland, Oregon, 97239, United States

  • University Hospitals

    Cleveland, Ohio, 44106, United States

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35233, United States

  • University of Cincinnati

    Cincinnati, Ohio, 45267, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Indiana

    Indianapolis, Indiana, 46202, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Utah

    Salt Lake City, Utah, 84132, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

More trials for these conditions

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