The purpose of this study is to look at pulmonary exacerbations in people with cystic fibrosis (CF) that need to be treated with antibiotics given through a tube inserted into a vein (intravenous or IV). A pulmonary exacerbation is a worsening of respiratory symptoms in people with CF that needs medical intervention. Both doctors and CF patients are trying to understand the best way to treat pulmonary exacerbations. This study is trying to answer the following questions about treating a pulmonary exacerbation: * Do participants have the same improvement in lung function and symptoms if they are treated with one type of antibiotic (called beta-lactams or β-lactams) versus taking two different types of antibiotics (tobramycin and β-lactams)? * Is taking one type of antibiotic just as good as taking two types?
Cystic Fibrosis Foundation (CFF) treatment guidelines for the management of pulmonary exacerbations (PEx) identified evidence gaps in current clinical best practices. The STOP program offers a platform for the conduct of controlled trials to develop the evidence base in order to define clinical best practices. The interventional Aminoglycoside Study (AG Study) will be a prospective, multi-center, parallel group, randomized (1:1 ratio), open-label, superiority study of intravenous aminoglycoside and β-lactams versus intravenous β-lactams only. Randomization will occur at Visit 1. The primary objective of this platform trial is to evaluate the efficacy and safety of differing treatments in CF PEx during a planned 14 day course of IV antimicrobials. Primary efficacy will be evaluated as the difference in mean Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) changes from Visit 1 to Visit 2 (Day 28 ± 2 days) between intervention arms.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
730
Intravenous (IV) β-lactam will be selected by the treating physician following standard of care. Treatment will last for 14 days (± 2 days).
Intravenous (IV) aminoglycoside will be selected by the treating physician following standard of care. Treatment will last for 14 days (± 2 days).
The Children's Hospital Alabama, University of Alabama at Birmingham
Birmingham, Alabama, United States
RECRUITINGTucson Cystic Fibrosis Center
Tucson, Arizona, United States
RECRUITINGUniversity of California San Diego
La Jolla, California, United States
RECRUITINGLong Beach Memorial Medical Center
Long Beach, California, United States
Absolute Change in FEV1 % Predicted at Week 4 in Aminoglycoside (AG) Study
Difference between aminoglycoside (AG) study intervention arms (AG - Non-AG) in the absolute change in FEV1 % predicted from Week 0 (Day 0) to Week 4 (Day 28 ± 2 days)
Time frame: Four weeks
Incidence of Adverse Events (AEs) in Aminoglycoside (AG) Study Intervention Arms
Difference between aminoglycoside (AG) study intervention arms (AG - Non-AG) in the proportion of participants with at least one AE from Week 0 (Day 0) to Week 6 (Day 44 ± 2 days).
Time frame: Six Weeks
Absolute Change in CFRSD-CRISS Score at Week 4 in Aminoglycoside (AG) Study
Difference between aminoglycoside (AG) study intervention arms (AG - Non-AG) in the absolute change in respiratory symptoms, as measured by the CF Respiratory Symptoms Diary-Chronic Respiratory Infection Symptom Score (CFRSD-CRISS) from Week 0 (Day 0) to Week 4 (Day 28 ± 2 days). The CFRSD-CRISS is derived from a set of questions asking a participant to state the extent of their 8 respiratory symptoms: difficulty breathing, feverishness, tiredness, chills or sweats, coughing, coughing up mucus, tightness in the chest and wheezing. Each respiratory symptom is assigned a score from 0-4 based on the response, with zero corresponding to the absence of the symptom and four corresponding to symptom being present 'a great deal' or 'extremely'. A summed score (range from 0-24) is calculated for each participant and converted to a final score with a range of 0 to 100, where the lowest scores indicate improvement of symptoms.
Time frame: Four weeks
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CHOC Children's Hospital
Orange, California, United States
RECRUITINGUniversity of California at Davis Medical Center
Sacramento, California, United States
RECRUITINGChildren's National Medical Center
Washington D.C., District of Columbia, United States
NOT_YET_RECRUITINGUniversity of Florida
Gainesville, Florida, United States
RECRUITINGJoe DiMaggio Children's Hospital
Hollywood, Florida, United States
RECRUITINGNemours Children's Clinic
Jacksonville, Florida, United States
RECRUITING...and 50 more locations