STOP-OB-13: Pilot Observational Study to Determine Feasibility of a Standardized Treatment of Pulmonary Exacerb. in Patients With CF

Sponsor

University of Washington (Other)

Overall Status

Completed

CT.gov ID

NCT02109822

Collaborator

Medical University of South Carolina (Other), Cystic Fibrosis Foundation (Other)

220

Enrollment

Locations

Duration (Months)

18.3

Patients Per Site

1.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis Lung Infection Cystic Fibrosis Pulmonary Exacerbation

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.

Study Design

Study Type:

Observational

Actual Enrollment :

220 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Pilot Observational Study to Determine Feasibility of a Standardized Treatment of Pulmonary Exacerbations in Patients With Cystic Fibrosis

Study Start Date :

Jan 1, 2014

Actual Primary Completion Date :

Jun 1, 2015

Actual Study Completion Date :

Jun 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
CF patients with pulmonary exacerbations Patients with CF who are hospitalized with a pulmonary exacerbation treated with intravenous antibiotics.

Outcome Measures

Primary Outcome Measures

Feasibility of using the CFF National Patient Registry [28 days from time of start of IV antibiotic therapy.]
Assess feasibility using the CFF National Patient Registry as measured by the accuracy of data entry

Secondary Outcome Measures

Physician and patient level clinical outcomes for their use in comparative studies of CF pulmonary exacerbations to determine the optimal treatment endpoints [During hospitalization and during a period following discharge of 28 days from time of start of IV antibiotic therapy.]
Evaluate physician assessment of treatment response as measured by the physician treatment assessment questionnaire. Evaluate change in lung function and patient reported respiratory symptoms and quality of life in response to treatment of an acute pulmonary exacerbation.

Other Outcome Measures

Variability of practicing clinicians' treatment objectives, approaches, and assessment of outcomes related to CF pulmonary exacerbation [during hospitalization and during a period following discharge of 28 days from time of start of IV antibiotic therapy.]
Describe the variability of practicing clinicians' treatment objectives as measured by the physician assessment questionnaire, treatment approaches as measured by the choices of medications (specifically antibiotics), and assessment of patient level outcomes related to CF pulmonary exacerbation (change in lung function as measured by spirometry, respiratory symptoms as measure by the CFRSD-CRISS questionnaire) and health related quality of life (as measured by the EQ-5D questionnaire).
Inform the design of future pragmatic research of CF pulmonary exacerbation [During hospitalization and during a period following discharge of 28 days from time of start of IV antibiotic therapy.]
Identify the appropriate target patient population for future clinical trials by clarifying required stratification factors, and estimating target treatment effects and variability of treatment response.

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	CFF Adult Program University of Alabama	Birmingham	Alabama	United States	35233
2	CFF Care Center Arizona Health Science Center	Tucson	Arizona	United States	85721
3	National Jewish Health	Denver	Colorado	United States	80206
4	Johns Hopkins	Baltimore	Maryland	United States	21205
5	CFF Care Center & Pediatric Program Rainbow Babies and Children's Hospita	Cleveland	Ohio	United States	44106
6	Rainbow Babies and Children's Hospital & University Hospitals Case Medical Center	Cleveland	Ohio	United States	44106
7	CFF Care Center & Pediatric Program Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania	United States	15224
8	Medical University of South Carolina	Charleston	South Carolina	United States	29425
9	CFF Care Center & Pediatric Program The University of Texas Southwestern Medical Center at Dallas	Dallas	Texas	United States	75235
10	CFF Care Center & Pediatric Program Seattle Children's Hospital	Seattle	Washington	United States	98105
11	University of Washington	Seattle	Washington	United States	98195
12	CFF Care Center & Pediatric Program University of Wisconsin	Madison	Wisconsin	United States	53706