Clinical trial NCT02109822: Pilot Observational Study to Determine Feasibility of a Standardized Treatment of Pulmonary Exacerb. in Patients With CF
Official title: Pilot Observational Study to Determine Feasibility of a Standardized Treatment of Pulmonary Exacerbations in Patients With Cystic Fibrosis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis
Lung Infection
Cystic Fibrosis Pulmonary Exacerbation
Keywords: Cystic Fibrosis
Pulmonary Exacerbation
Lung Infection
Cystic Fibrosis Foundation
Cystic Fibrosis Foundation National Patient Registry
Lead sponsor: University of Washington
Sponsor class: OTHER
Collaborators: name: Medical University of South Carolina
name: Cystic Fibrosis Foundation
First posted: 2014-04-10
Enrollment: 220
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with Cystic Fibrosis who are hospitalized with a pulmonary exacerbation treated with intravenous antibiotics.

Brief summary: 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: 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.

Eligibility criteria: 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)

Primary outcomes:
measure: Feasibility of using the CFF National Patient Registry
timeFrame: 28 days from time of start of IV antibiotic therapy.
Secondary outcomes:
measure: Physician and patient level clinical outcomes for their use in comparative studies of CF pulmonary exacerbations to determine the optimal treatment endpoints
timeFrame: During hospitalization and during a period following discharge of 28 days from time of start of IV antibiotic therapy.
Other outcomes:
measure: Variability of practicing clinicians' treatment objectives, approaches, and assessment of outcomes related to CF pulmonary exacerbation
timeFrame: during hospitalization and during a period following discharge of 28 days from time of start of IV antibiotic therapy.
measure: Inform the design of future pragmatic research of CF pulmonary exacerbation
timeFrame: During hospitalization and during a period following discharge of 28 days from time of start of IV antibiotic therapy.
Locations:
city: Birmingham
country: United States
facility: CFF Adult Program University of Alabama
state: Alabama
city: Tucson
country: United States
facility: CFF Care Center Arizona Health Science Center
state: Arizona
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
city: Baltimore
country: United States
facility: Johns Hopkins
state: Maryland
city: Cleveland
country: United States
facility: CFF Care Center & Pediatric Program Rainbow Babies and Children's Hospita
state: Ohio
city: Cleveland
country: United States
facility: Rainbow Babies and Children's Hospital & University Hospitals Case Medical Center
state: Ohio
city: Pittsburgh
country: United States
facility: CFF Care Center & Pediatric Program Children's Hospital of Pittsburgh
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Dallas
country: United States
facility: CFF Care Center & Pediatric Program The University of Texas Southwestern Medical Center at Dallas
state: Texas
city: Seattle
country: United States
facility: CFF Care Center & Pediatric Program Seattle Children's Hospital
state: Washington
city: Seattle
country: United States
facility: University of Washington
state: Washington
city: Madison
country: United States
facility: CFF Care Center & Pediatric Program University of Wisconsin
state: Wisconsin
Study officials:
affiliation: University of Washington
name: Christopher H. Goss, MD MSc
role: PRINCIPAL_INVESTIGATOR
affiliation: Medical University of South Carolina
name: Patrick Flume, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.