Purpose

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

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Confirmed diagnosis of CF as defined by both of the following: - Two CF disease-causing 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. - 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, 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. - Receiving treatment with ivacaftor monotherapy (KALYDECO) - For all groups except Daily dosing: Receiving treatment with triple combination therapy (TRIKAFTA). - Subjects 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 is not an exclusion for this study.

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Low Dose
8 mg MRT5005
  • Drug: MRT5005
    Nebulization of MRT5005
Experimental
Low/Mid Dose
12 mg MRT5005
  • Drug: MRT5005
    Nebulization of MRT5005
Experimental
Mid Dose
16 mg MRT5005
  • Drug: MRT5005
    Nebulization of MRT5005
Experimental
Mid/High Dose
20 mg MRT5005
  • Drug: MRT5005
    Nebulization of MRT5005
Experimental
High Dose
24 mg MRT5005
  • Drug: MRT5005
    Nebulization of MRT5005
Placebo Comparator
Placebo Comparator
Normal Saline 0.9% USP
  • Drug: Normal saline
    Normal Saline for Inhalation
Experimental
Daily Dose
20 mg MRT5005 delivered in 5 consecutive daily doses of 4mg
  • Drug: MRT5005
    Nebulization of MRT5005

Recruiting Locations

University of Alabama at Birmingham
Birmingham, Alabama 35233

More Details

Status
Recruiting
Sponsor
Translate Bio, Inc.

Study Contact

Elizabeth Brown
781-386-7261
ebrown@translate.bio

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.