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).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
42
Nebulization of MRT5005
Normal Saline for Inhalation
University of Alabama at Birmingham
Birmingham, Alabama, United States
National Jewish Health
Denver, Colorado, United States
Parts A, B and D: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily had a causal relationship with the study treatment. A serious adverse event (SAE) was any untoward medical occurrence (whether considered to be related to study treatment or not) that at any dose: resulted in death; or was life-threatening; or required inpatient hospitalization or prolongation of existing hospitalization; or resulted in persistent or significant disability/incapacity; or was a congenital abnormality/birth defect; or was an important medical event. The TEAEs were defined as events that were newly reported or reported to worsen in severity after the start of study treatment up to 48 weeks (end of follow-up period) after the last dose of study treatment administration.
Time frame: From the first dose of study treatment administration (Day 1) up to 48 weeks (end of the follow-up period) after the last dose of study treatment administration (Part A: Day 337, Part B: Day 365 and Part D: Day 341)
Parts A, B and D: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Spirometry was performed according to the guidelines published by the American Thoracic Society for standardization of spirometry. The ppFEV1 was assessed during the spirometry testing. For Parts A and B, the baseline value was defined as the average of the results from testing on Day -1 and at pre-dose on Day 1. For Part D, the baseline value was defined as the result from testing pre-dose on Day 1. Here, Baseline= BL, Day= D, pre-dose= PrD, hours post dose= hPD.
Time frame: Part A:BL, D1(8hPD), D2(24hPD), D3, D8, D15 and D29; Part B:BL, D1(6hPD), D2, D8(PrD and 6hPD), D9, D15(PrD and 6hPD), D16, D22(PrD and 6hPD), D23, D29(PrD and 6hPD), D30, D36, D43 and D57; Part D:BL, D1(2hPD), PrD on D2, D3, D4 and D5, D11, D18 and D32
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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
...and 6 more locations