Pilot Observational Study to Determine Feasibility of a Standardized Treatment of Pulmonary Exacerb. in Patients With CF

NCT ID: NCT02109822

Last Updated: 2016-10-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

220 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-01-31

Study Completion Date

2015-06-30

Brief Summary

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The goal of this research study is to better understand current treatment practices for pulmonary exacerbations (lung infections) and whether the Cystic Fibrosis National Patient Registry (CFFNPR)can be used for this type of study.

Detailed Description

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Cystic fibrosis (CF), a life-shortening genetic disease, is marked by acute episodes during which symptoms of lung infection increase and lung function decreases. These pulmonary exacerbations (PEs) are treated with varying antibiotics for varying time periods based on needs determined by individual patients, their families, and the health care providers. Cystic fibrosis pulmonary guidelines for the treatment of PE published by the Cystic Fibrosis Foundation (CFF) in 2009 provided recommendations for treatment and also identified key questions for which additional studies were needed.

Standard treatment for PE involves many facets including selection of antibiotics, duration of use, and outcomes that define treatment success. Understanding current treatment practices and measures of treatment success are needed before a study can be designed to define optimal treatment strategies.

This is a multi-center, prospective, observational study designed to prospectively follow patients with CF that are initially admitted to the hospital for treatment of a pulmonary exacerbation.

Conditions

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Cystic Fibrosis Lung Infection Cystic Fibrosis Pulmonary Exacerbation

Keywords

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Cystic Fibrosis Pulmonary Exacerbation Lung Infection Cystic Fibrosis Foundation Cystic Fibrosis Foundation National Patient Registry

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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CF patients with pulmonary exacerbations

Patients with CF who are hospitalized with a pulmonary exacerbation treated with intravenous antibiotics.

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* Male or female ≥12 years of age at Visit 1
* Enrolled in the CFFNPR (Patients may enroll in the Registry at Visit 1 if not previously enrolled.)
* Current hospitalization for treatment of a pulmonary exacerbation
* Planned hospital admission of at least 5 days with intravenous (IV) antibiotics at Visit 1
* Able to perform spirometry at admission and willing to perform spirometry on subsequent treatment and visit days
* Willing and able to complete symptom score daily
* Willing to return for a follow up visit at end of treatment (if necessary) and 28 days after start of IV antibiotic therapy
* Written informed consent (and assent when applicable) obtained from the participant or participant's legal representative

Exclusion Criteria

* Previous enrollment in this study
* Treatment with IV antibiotics in the 6 weeks prior to Visit 1
* Admission to the intensive care unit for current pulmonary exacerbation
* Pneumothorax on admission
* Current hospitalization for scheduled pulmonary clean out
* Current hospitalization for sinusitis as the primary diagnosis
* Massive hemoptysis defined as \> 250 cc in a 24 hour period, or 100 cc/day over 4 consecutive days occurring within one week of Visit 1
* Current pulmonary exacerbation thought to be due to allergic bronchopulmonary aspergillosis (ABPA)
* Ongoing treatment with prednisone equivalent \>10 mg/day for greater than 2 weeks initiated prior to Visit 1
* History of solid organ transplantation Currently receiving antimicrobial therapy to treat non-tuberculous mycobacterium (e.g., M. abscessus, M. avium complex)
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Medical University of South Carolina

OTHER

Sponsor Role collaborator

Cystic Fibrosis Foundation

OTHER

Sponsor Role collaborator

University of Washington

OTHER

Sponsor Role lead

Responsible Party

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Chris Goss

Professor of Medicine

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Christopher H. Goss, MD MSc

Role: PRINCIPAL_INVESTIGATOR

University of Washington

Patrick Flume, MD

Role: PRINCIPAL_INVESTIGATOR

Medical University of South Carolina

Locations

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CFF Adult Program University of Alabama

Birmingham, Alabama, United States

Site Status

CFF Care Center Arizona Health Science Center

Tucson, Arizona, United States

Site Status

National Jewish Health

Denver, Colorado, United States

Site Status

Johns Hopkins

Baltimore, Maryland, United States

Site Status

CFF Care Center & Pediatric Program Rainbow Babies and Children's Hospita

Cleveland, Ohio, United States

Site Status

Rainbow Babies and Children's Hospital & University Hospitals Case Medical Center

Cleveland, Ohio, United States

Site Status

CFF Care Center & Pediatric Program Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

CFF Care Center & Pediatric Program The University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, United States

Site Status

CFF Care Center & Pediatric Program Seattle Children's Hospital

Seattle, Washington, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

CFF Care Center & Pediatric Program University of Wisconsin

Madison, Wisconsin, United States

Site Status

Countries

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United States

References

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VanDevanter DR, Heltshe SL, Sanders DB, West NE, Skalland M, Flume PA, Goss CH; STOP-OB Study. Changes in symptom scores as a potential clinical endpoint for studies of cystic fibrosis pulmonary exacerbation treatment. J Cyst Fibros. 2021 Jan;20(1):36-38. doi: 10.1016/j.jcf.2020.08.006. Epub 2020 Aug 13.

Reference Type DERIVED
PMID: 32800708 (View on PubMed)

Stuart Elborn J, Geller DE, Conrad D, Aaron SD, Smyth AR, Fischer R, Kerem E, Bell SC, Loutit JS, Dudley MN, Morgan EE, VanDevanter DR, Flume PA. A phase 3, open-label, randomized trial to evaluate the safety and efficacy of levofloxacin inhalation solution (APT-1026) versus tobramycin inhalation solution in stable cystic fibrosis patients. J Cyst Fibros. 2015 Jul;14(4):507-14. doi: 10.1016/j.jcf.2014.12.013. Epub 2015 Jan 13.

Reference Type DERIVED
PMID: 25592656 (View on PubMed)

Other Identifiers

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46256-EA

Identifier Type: -

Identifier Source: org_study_id