Standardizing Treatments for Pulmonary Exacerbations - Aminoglycoside Study

Purpose

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?

Conditions

  • Cystic Fibrosis
  • Cystic Fibrosis Pulmonary Exacerbation

Eligibility

Eligible Ages
Over 6 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 4
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
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
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Other
β-lactam Only (Non-AG) 		Participants randomized to this arm will be prescribed a standard of care intravenous (IV) β-lactam as selected by their treating physician. Treatment must not include an IV aminoglycoside.
  • Drug: Beta-lactam antibiotic
    Intravenous (IV) β-lactam will be selected by the treating physician following standard of care. Treatment will last for 14 days (± 2 days).
    Other names:
    • β-lactam
Other
β-lactam and Aminoglycoside (AG) 		Participants randomized to this arm will be prescribed a standard of care intravenous (IV) β-lactam and aminoglycoside selected by their treating physician.
  • Drug: Beta-lactam antibiotic
    Intravenous (IV) β-lactam will be selected by the treating physician following standard of care. Treatment will last for 14 days (± 2 days).
    Other names:
    • β-lactam
  • Drug: Aminoglycoside
    Intravenous (IV) aminoglycoside will be selected by the treating physician following standard of care. Treatment will last for 14 days (± 2 days).
    Other names:
    • AG

Recruiting Locations

The Children's Hospital Alabama, University of Alabama at Birmingham
Birmingham, Alabama 35233
Contact:
Heather Hathorne
hyhathorne@uabmc.edu

More Details

Status
Recruiting
Sponsor
Chris Goss

Study Contact

Rachael Buckingham, BS
206-884-7517
Rachael.buckingham@seattlechildrens.org

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.