Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis
- Registration Number
- NCT03375047
- Lead Sponsor
- Translate Bio, Inc.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 40
-
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).
- 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
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Comparator Normal saline Normal Saline 0.9% USP Low Dose MRT5005 8 mg MRT5005 Low/Mid Dose MRT5005 12 mg MRT5005 Mid/High Dose MRT5005 20 mg MRT5005 Mid Dose MRT5005 16 mg MRT5005 High Dose MRT5005 24 mg MRT5005 Daily Dose MRT5005 20 mg MRT5005 delivered in 5 consecutive daily doses of 4mg
- Primary Outcome Measures
Name Time Method Types, frequency and severity of treatment-emergent Adverse Events 12 months after last dose Safety and tolerability of nebulized MRT5005 will be assessed through the types, frequency and severity of treatment-emergent Adverse Events experienced by participants on the trial
- Secondary Outcome Measures
Name Time Method Biological activity of nebulized MRT5005 4 weeks after last dose Changes from baseline in ppFEV1
Trial Locations
- Locations (18)
Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
Central Florida Pulmonary Group
🇺🇸Orlando, Florida, United States
Northwestern University
🇺🇸Chicago, Illinois, United States
University of Indiana
🇺🇸Indianapolis, Indiana, United States
University of Cincinnati
🇺🇸Cincinnati, Ohio, United States
University Hospitals
🇺🇸Cleveland, Ohio, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Oregon Health and Sciences University
🇺🇸Portland, Oregon, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
University of Florida
🇺🇸Gainesville, Florida, United States
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
Maine Medical Center
🇺🇸Portland, Maine, United States
New Orleans Center for Clinical Research
🇺🇸Knoxville, Tennessee, United States