Biomarkers of Iron Homeostasis and Responses to Cystic Fibrosis Pulmonary Exacerbation (CFPE) Treatment

Completed

• Conditions: Cystic Fibrosis
• Interventions: Other: Adults - CFPE Treatment

• Registration Number: NCT02188758
• Lead Sponsor: Dartmouth-Hitchcock Medical Center

Brief Summary

The goal of this study is to identify chemical compounds in the blood and sputum (i.e., biomarkers) that are associated with objective measurements of health status in patients with cystic fibrosis (CF). This study builds upon observations that blood levels of hepcidin-25, a protein that regulates how the body uses and stores iron, vary during CF pulmonary exacerbation (CFPE).

Detailed Description

Not available

Study Timeline

• Study Start: 2014-07
• Study First Submitted: 2014-07-09
• Study First Submitted QC: 2014-07-10
• Study First Posted: 2014-07-14
• Primary Completion: 2017-12
• Study Completion: 2018-01
• Status Verified: 2018-03
• Last Update Submitted: 2018-03-09
• Last Update Posted: 2018-03-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

Not provided

Exclusion Criteria

• Women who are pregnant or lactating;
• Subject does not meet Inclusion criteria;
• Recent and/or persistent visible blood in sputum (hemoptysis);
• Rescue use of oral antibiotics within the previous 3 weeks, defined as antibiotic use for health deterioration rather than chronic suppression

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Adults - CFPE Treatment	Adults - CFPE Treatment	Other: CF Pulmonary Exacerbation (CFPE) Treatment

Primary Outcome Measures

Name	Time	Method
Change in Serum Hepcidin-25 Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment	Duration of hospitalization, an expected average of 12 days	The primary endpoint of this study is the characterization of 3 groups (i.e., "low," "intermediate," and "high") of serum hepcidin-25 responders to CFPE treatment. Response will be defined as the ratio of post- to pre-treatment serum hepcidin-25 concentration for each subject.

Secondary Outcome Measures

Name	Time	Method
Change in Serum Iron After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment	Duration of hospitalization, an expected average of 12 days	Post- to pre-treatment ratio for serum iron concentration for each subject.
Change in Serum Interleukin-6 (IL-6) Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment	Duration of hospitalization, an expected average of 12 days	Post- to pre-treatment ratio for serum interleukin-6 (IL-6) concentration for each subject.
Change in Sputum Iron Content After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment	Duration of hospitalization, an expected average of 12 days	Post- to pre-treatment ratio for sputum iron content for each subject.
Change in Percent-Predicted Forced Expiratory Volume in One Second (FEV1%) After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment	Duration of hospitalization, an expected average of 12 days	Post- to pre-treatment ratio for FEV1% for each subject.
Change in Body Mass Index (BMI) After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment	Duration of hospitalization, an expected average of 12 days	Post- to pre-treatment ratio for BMI for each subject.
Change in Hemoglobin Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment	Duration of hospitalization, an expected average of 12 days	Post- to pre-treatment ratio for serum hemoglobin concentration for each subject.
Change in CFRSD-CRISS Score After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment	Duration of hospitalization, an expected average of 12 days	CFRSD-CRISS is a patient-reported outcome (PRO) instrument developed by the Seattle Quality of Life Group at the University of Washington and used herein under license to evaluate the severity of symptoms of CF in adults and adolescents (≥12 years) with a chronic respiratory infection. Symptoms assessed in the CFRSD-CRISS are: difficulty breathing, cough, cough up mucus, chest tightness, wheeze, feeling feverish, tired, and chills/sweats. The 8 items quantify symptom severity for the previous 24 hours to capture the magnitude of symptoms in stable CF, during medically treated CF exacerbations, and during recovery from an exacerbation. We will determine the within-subject differences in CFRSD-CRISS score associated with CFPE treatment.
Change in Serum EPO Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment	Duration of hospitalization, an expected average of 12 days	Post- to pre-treatment ratio for serum erythropoietin (EPO) for each subject.
Change in Transferrin Saturation After Hospitalization for CF Pulmonary Exacerbation Treatment	Duration of hospitalization, an expected average of 12 days	Post- to pre-treatment ratio for transferrin saturation for each subject.
Change in Serum TREM-1 Concentration After Hospitalization for CF Pulmonary Exacerbation Treatment	Duration of hospitalization, an expected average of 12 days	Post- to pre-treatment ratio for serum triggering receptor expressed on myeloid cells-1 (TREM-1) for each subject.
Change in Serum sIL-6R Concentration After Hospitalization for CF Pulmonary Exacerbation Treatment	Duration of hospitalization, an expected average of 12 days	Post- to pre-treatment ratio for serum soluble IL-6 receptor (sIL-6R) concentration for each subject.

Trial Locations

Locations (2)

Maine Medical Center; 🇺🇸 South Portland, Maine, United States

Dartmouth-Hitchcock Medical Center; 🇺🇸 Lebanon, New Hampshire, United States