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

Cystic Fibrosis Clinical Trial

— STOP-OB-13  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02109822
• Other study ID #: 46256-EA
• Status: Completed
• Phase: N/A
• First received: March 6, 2014
• Last updated: October 24, 2016
• Start date: January 2014
• Est. completion date: June 2015

Study information

• Verified date: October 2016
• Source: University of Washington
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 220
• Est. completion date: June 2015
• Est. primary completion date: June 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years and older

Eligibility

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)

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Cystic Fibrosis
• Cystic Fibrosis Pulmonary Exacerbation
• Fibrosis
• Lung Infection
• Pulmonary Fibrosis

Locations

Country	Name	City	State
United States	Johns Hopkins	Baltimore	Maryland
United States	CFF Adult Program University of Alabama	Birmingham	Alabama
United States	Medical University of South Carolina	Charleston	South Carolina
United States	CFF Care Center & Pediatric Program Rainbow Babies and Children's Hospita	Cleveland	Ohio
United States	Rainbow Babies and Children's Hospital & University Hospitals Case Medical Center	Cleveland	Ohio
United States	CFF Care Center & Pediatric Program The University of Texas Southwestern Medical Center at Dallas	Dallas	Texas
United States	National Jewish Health	Denver	Colorado
United States	CFF Care Center & Pediatric Program University of Wisconsin	Madison	Wisconsin
United States	CFF Care Center & Pediatric Program Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	CFF Care Center & Pediatric Program Seattle Children's Hospital	Seattle	Washington
United States	University of Washington	Seattle	Washington
United States	CFF Care Center Arizona Health Science Center	Tucson	Arizona

Sponsors (3)

Lead Sponsor	Collaborator
University of Washington	Cystic Fibrosis Foundation Therapeutics, Medical University of South Carolina

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Variability of practicing clinicians' treatment objectives, approaches, and assessment of outcomes related to CF pulmonary exacerbation	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).	during hospitalization and during a period following discharge of 28 days from time of start of IV antibiotic therapy.	No
Other	Inform the design of future pragmatic research of CF pulmonary exacerbation	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.	During hospitalization and during a period following discharge of 28 days from time of start of IV antibiotic therapy.	No
Primary	Feasibility of using the CFF National Patient Registry	Assess feasibility using the CFF National Patient Registry as measured by the accuracy of data entry	28 days from time of start of IV antibiotic therapy.	No
Secondary	Physician and patient level clinical outcomes for their use in comparative studies of CF pulmonary exacerbations to determine the optimal treatment endpoints	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.	During hospitalization and during a period following discharge of 28 days from time of start of IV antibiotic therapy.	No