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Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis | Clinical Trials | Clareo Health

COMPLETEDPHASE1/PHASE2

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

A Phase 1/2, Randomized, Double-Blinded, Placebo-Controlled, Combined Single and Multiple Ascending Dose Study Evaluating the Safety, Tolerability, and Biological Activity of MRT5005 Administered by Nebulization to Adult Subjects With Cystic Fibrosis

NCT03375047•Sanofi

View on ClinicalTrials.gov

Summary

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).

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

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.

Locations (16)

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

Key Dates

Start Date

May 10, 2018

Primary Completion Date

March 15, 2022

Completion Date

March 15, 2022

Last Updated

February 9, 2026

Enrollment

ACTUAL participants

Conditions

Cystic Fibrosis

Interventions

MRT5005

DRUG

Normal saline

DRUG

Natural History of Noncirrhotic Portal Hypertension

NCT02417740

Cystic FibrosisImmunologic Deficiency Syndrome+3 more

View study

RECRUITINGNA

Personalized Mobile Health Platform to Promote Physical Activity in Adolescents and Young Adults With Cystic Fibrosis

NCT06616857

Cystic Fibrosis (CF)

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 9, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

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