Clinical trial NCT05548283: Standardizing Treatments for Pulmonary Exacerbations - Aminoglycoside Study
Official title: Standardizing Treatments for Pulmonary Exacerbations: A Platform for Evaluating Treatment Decisions to Improve Outcomes (STOP360) Aminoglycoside Study (AG Study)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Cystic Fibrosis
Cystic Fibrosis Pulmonary Exacerbation
Keywords: Cystic Fibrosis
CF
Cystic Fibrosis Pulmonary Exacerbation
aminoglycoside
beta-lactam
β-lactam
STOP
STOP360
Lead sponsor: Seattle Children's Hospital
Sponsor class: OTHER
Collaborators: name: University of Washington
name: Medical University of South Carolina
name: Cystic Fibrosis Foundation
First posted: 2022-09-21
Enrollment: 730
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: The aminoglycoside study will compare treatment of an IV β-lactam and aminoglycoside (AG) versus an IV β-lactam only (non-AG). Individuals will be randomly assigned in a 1:1 fashion to receive either the AG or non-AG intervention for a planned 14 day course of IV antibiotics. IV antibiotic treatments for each intervention arm will be selected by the treating physician following standard of care.
Primary purpose: TREATMENT

Brief summary: 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?

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* All genders ≥ 6 years of age at Visit 1
* Documentation of a CF diagnosis
* Clinician intent to treat index CF PEx with a planned 14-day course of IV antimicrobials
* At least one documented Pa positive culture within two years prior to Visit 1

Exclusion Criteria:

* Participant is not pregnant
* No known renal impairment or history of solid organ transplantation
* No IV antimicrobial treatment, ICU admission, pneumothorax, or hemoptysis within 6 weeks prior to Visit 1
* No use of investigational therapies, new CF transmembrane conductance regulator (CFTR) modulators, or treatment for Nontuberculous mycobacteria (NTM) within 4 weeks prior to Visit 1
* No history of hypersensitivity, vestibular, or auditory toxicity with aminoglycosides
* No more than one day of IV aminoglycosides administered for the current PEx treatment prior to Visit 1

Primary outcomes:
measure: Absolute Change in FEV1 % Predicted at Week 4 in Aminoglycoside (AG) Study
timeFrame: Four weeks
measure: Incidence of Adverse Events (AEs) in Aminoglycoside (AG) Study Intervention Arms
timeFrame: Six Weeks
Secondary outcomes:
measure: Absolute Change in CFRSD-CRISS Score at Week 4 in Aminoglycoside (AG) Study
timeFrame: Four weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: The Children's Hospital Alabama, University of Alabama at Birmingham
state: Alabama
city: Tucson
country: United States
facility: Tucson Cystic Fibrosis Center
state: Arizona
city: La Jolla
country: United States
facility: University of California San Diego
state: California
city: Long Beach
country: United States
facility: Long Beach Memorial Medical Center
state: California
city: Orange
country: United States
facility: CHOC Children's Hospital
state: California
city: Sacramento
country: United States
facility: University of California at Davis Medical Center
state: California
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Hollywood
country: United States
facility: Joe DiMaggio Children's Hospital
state: Florida
city: Jacksonville
country: United States
facility: Nemours Children's Clinic
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Saint Petersburg
country: United States
facility: All Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Boise
country: United States
facility: Saint Luke's Cystic Fibrosis Center of Idaho
state: Idaho
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Peoria
country: United States
facility: OSF Saint Francis Medical Center
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University Medical Center
state: Indiana
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Louisville
country: United States
facility: University of Louisville
state: Kentucky
city: Portland
country: United States
facility: Maine Medical Center
state: Maine
city: Baltimore
country: United States
facility: John Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital, Brigham & Women's Hospital
state: Massachusetts
city: Worcester
country: United States
facility: University of Massachusetts Memorial Health Care
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan, Michigan Medicine
state: Michigan
city: Grand Rapids
country: United States
facility: Helen DeVos Children's Hospital
state: Michigan
city: Saint Louis
country: United States
facility: SSM Health Cardinal Glennon Children's Hospital
state: Missouri
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Billings
country: United States
facility: Billings Clinic
state: Montana
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: Morristown
country: United States
facility: Morristown Medical Center
state: New Jersey
city: New Brunswick
country: United States
facility: Rutgers - Robert Wood Johnson Medical School
state: New Jersey
city: New York
country: United States
facility: Lenox Hill Hospital Cystic Fibrosis Center
state: New York
city: New York
country: United States
facility: Children's Hospital of New York
state: New York
city: Valhalla
country: United States
facility: New York Medical College at Westchester Medical Center
state: New York
city: Akron
country: United States
facility: Children's Hospital Medical Center of Akron
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Cincinnati
country: United States
facility: University of Cincinnati Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Dayton
country: United States
facility: Dayton Children's Hospital
state: Ohio
city: Toledo
country: United States
facility: Toledo Children's Hospital
state: Ohio
city: Oklahoma City
country: United States
facility: Oklahoma Cystic Fibrosis Center
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health Sciences University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt Children's Hospital
state: Tennessee
city: Dallas
country: United States
facility: University of Texas Southwestern / Children's Health
state: Texas
city: Dallas
country: United States
facility: University of Texas Southwestern
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Seattle
country: United States
facility: University of Washington Medical Center
state: Washington
city: Spokane
country: United States
facility: Providence Medical Group, Cystic Fibrosis Clinic
state: Washington
city: Morgantown
country: United States
facility: West Virginia University - Morgantown
state: West Virginia
city: Madison
country: United States
facility: University of Wisconsin
state: Wisconsin
city: Calgary
country: Canada
facility: University of Calgary Adult Cystic Fibrosis Clinic (Calgary, AB)
state: Alberta
Study officials:
affiliation: Medical University of South Carolina
name: Patrick Flume, MD
role: PRINCIPAL_INVESTIGATOR
References: