Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis

Completed · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

This Phase 1/2, first-in-human study evaluated the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization to the respiratory tract of adult subjects with cystic fibrosis (CF).

Key facts

Study ID

NCT03375047

Run by

Sanofi

People needed

42

Starts

2018-05-10

Expected to finish

2022-03-15

Last updated by the study team

2026-02-09

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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).

You may not qualify if…

• 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.

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• National Jewish Health — Denver, Colorado, United States
• University of Florida — Gainesville, Florida, United States
• Northwestern University — Chicago, Illinois, United States
• University of Indiana — Indianapolis, Indiana, United States
• Maine Medical Center — Portland, Maine, United States
• Johns Hopkins University — Baltimore, Maryland, United States
• University of Michigan — Ann Arbor, Michigan, United States
• University of Cincinnati — Cincinnati, Ohio, United States
• University Hospitals — Cleveland, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Oregon Health and Sciences University — Portland, Oregon, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States
• New Orleans Center for Clinical Research — Knoxville, Tennessee, United States
• University of Utah — Salt Lake City, Utah, United States
• Virginia Commonwealth University — Richmond, Virginia, United States

Full record on ClinicalTrials.gov

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