Standardizing Treatments for Pulmonary Exacerbations - Aminoglycoside Study

NCT05548283 | Recruiting Phase 4 DMC

• 1 other identifier: STOP360-IP-22 AG
• Study Type: interventional
• Target: 730
• Locations: 2 countries
• Sites: 60

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?

Conditions

Cystic Fibrosis; Cystic Fibrosis Pulmonary Exacerbation

Keywords

Cystic Fibrosis; CF; Cystic Fibrosis Pulmonary Exacerbation; aminoglycoside; beta-lactam; β-lactam; STOP; STOP360

Outcome Measures

Primary Outcomes (2)

• 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)

  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).

  Six Weeks

Secondary Outcomes (1)

• Absolute Change in CFRSD-CRISS Score at Week 4 in Aminoglycoside (AG) Study

  Four weeks

Study Arms (2)

β-lactam Only (Non-AG)

OTHER

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

β-lactam and Aminoglycoside (AG)

OTHER

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; Drug: Aminoglycoside

Interventions

Beta-lactam antibiotic DRUG

Intravenous (IV) β-lactam will be selected by the treating physician following standard of care. Treatment will last for 14 days (± 2 days).

Also known as: β-lactam

β-lactam Only (Non-AG); β-lactam and Aminoglycoside (AG)

Aminoglycoside DRUG

Intravenous (IV) aminoglycoside will be selected by the treating physician following standard of care. Treatment will last for 14 days (± 2 days).

Also known as: AG

β-lactam and Aminoglycoside (AG)

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Chris Goss: lead
• University of Washington: collaborator
• Medical University of South Carolina: collaborator
• Cystic Fibrosis Foundation: collaborator

Study Sites (60)

The Children's Hospital Alabama, University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

RECRUITING

Tucson Cystic Fibrosis Center

Tucson, Arizona, 85724, United States

RECRUITING

University of California San Diego

La Jolla, California, 92093, United States

RECRUITING

Long Beach Memorial Medical Center

Long Beach, California, 90806, United States

RECRUITING

CHOC Children's Hospital

Orange, California, 92868, United States

RECRUITING

University of California at Davis Medical Center

Sacramento, California, 95817, United States

RECRUITING

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

NOT YET RECRUITING

University of Florida

Gainesville, Florida, 32610, United States

RECRUITING

Joe DiMaggio Children's Hospital

Hollywood, Florida, 33021, United States

RECRUITING

Nemours Children's Clinic

Jacksonville, Florida, 32207, United States

RECRUITING

University of Miami

Miami, Florida, 33136, United States

RECRUITING

All Children's Hospital

St. Petersburg, Florida, 33701, United States

RECRUITING

Emory University

Atlanta, Georgia, 30327, United States

RECRUITING

Saint Luke's Cystic Fibrosis Center of Idaho

Boise, Idaho, 83702, United States

RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

RECRUITING

Northwestern University

Chicago, Illinois, 60611, United States

RECRUITING

OSF Saint Francis Medical Center

Peoria, Illinois, 61637, United States

RECRUITING

Indiana University Medical Center

Indianapolis, Indiana, 46202, United States

RECRUITING

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

RECRUITING

University of Iowa

Iowa City, Iowa, 52242, United States

RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

RECRUITING

University of Louisville

Louisville, Kentucky, 40202, United States

RECRUITING

Maine Medical Center

Portland, Maine, 04102, United States

RECRUITING

John Hopkins Hospital

Baltimore, Maryland, 21205, United States

RECRUITING

Boston Children's Hospital, Brigham & Women's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

University of Massachusetts Memorial Health Care

Worcester, Massachusetts, 01655, United States

RECRUITING

University of Michigan, Michigan Medicine

Ann Arbor, Michigan, 48109, United States

RECRUITING

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49546, United States

RECRUITING

SSM Health Cardinal Glennon Children's Hospital

St Louis, Missouri, 63104, United States

RECRUITING

Washington University School of Medicine

St Louis, Missouri, 63110, United States

RECRUITING

Billings Clinic

Billings, Montana, 59101, United States

RECRUITING

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

RECRUITING

Morristown Medical Center

Morristown, New Jersey, 07960, United States

RECRUITING

Rutgers - Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08903, United States

RECRUITING

Lenox Hill Hospital Cystic Fibrosis Center

New York, New York, 10003, United States

RECRUITING

Children's Hospital of New York

New York, New York, 10032, United States

RECRUITING

New York Medical College at Westchester Medical Center

Valhalla, New York, 10595, United States

RECRUITING

Children's Hospital Medical Center of Akron

Akron, Ohio, 44308, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

University of Cincinnati Medical Center

Cincinnati, Ohio, 45267, United States

RECRUITING

Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44146, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

RECRUITING

Dayton Children's Hospital

Dayton, Ohio, 45404, United States

RECRUITING

Toledo Children's Hospital

Toledo, Ohio, 43606, United States

RECRUITING

Oklahoma Cystic Fibrosis Center

Oklahoma City, Oklahoma, 73104, United States

RECRUITING

Oregon Health Sciences University

Portland, Oregon, 97239, United States

RECRUITING

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

RECRUITING

Vanderbilt Children's Hospital

Nashville, Tennessee, 37232, United States

RECRUITING

University of Texas Southwestern / Children's Health

Dallas, Texas, 75207, United States

RECRUITING

University of Texas Southwestern

Dallas, Texas, 75390, United States

RECRUITING

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

RECRUITING

Virginia Commonwealth University

Richmond, Virginia, 23219, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

University of Washington Medical Center

Seattle, Washington, 98195, United States

RECRUITING

Providence Medical Group, Cystic Fibrosis Clinic

Spokane, Washington, 99204, United States

RECRUITING

West Virginia University - Morgantown

Morgantown, West Virginia, 26507, United States

RECRUITING

University of Wisconsin

Madison, Wisconsin, 53792, United States

RECRUITING

University of Calgary Adult Cystic Fibrosis Clinic (Calgary, AB)

Calgary, Alberta, T2N4N1, Canada

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Monobactams; Aminoglycosides

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

beta-Lactams; Lactams; Amides; Organic Chemicals; Sulfur Compounds; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Glycosides; Carbohydrates

Study Officials

• Patrick Flume, MD

  Medical University of South Carolina

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Rachael Buckingham, BS

CONTACT

206-884-7517; Rachael.buckingham@seattlechildrens.org

Barbra Fogarty

CONTACT

206-884-7592; Barbra.fogarty@seattlechildrens.org

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Associate Director, UW Adult CF Center

Model Details: 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.

Study Record Dates

• First Submitted: September 16, 2022
• First Posted: September 21, 2022
• Study Start: April 23, 2023
• Primary Completion (Estimated): November 15, 2026
• Study Completion (Estimated): November 15, 2026
• Last Updated: August 26, 2024

Record last verified: 2024-08

Locations

CA (1); US (59)