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

March 9, 2018 updated by: Alex H. Gifford, Dartmouth-Hitchcock Medical Center

Do Changes in Serum Hepcidin-25 Concentration Predict Cystic Fibrosis Pulmonary Exacerbation (CFPE) Treatment Responses?

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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

20

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Maine
      • South Portland, Maine, United States, 04106
        • Maine Medical Center
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Dartmouth-Hitchcock Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients greater than

Description

Inclusion Criteria (required at screening visit):

  • Diagnosis of CF confirmed by history of positive chloride sweat test and/or CFTR mutation analysis;
  • History of consistent sputum production on most occasions;
  • FEV1% greater than or equal to 75% of best measurement in previous 6 months;
  • 1 or more hospitalizations for CFPE treatment with intravenous antibiotics within the previous year;
  • Absence of CFPE (i.e., Akron Pulmonary Exacerbation Score <5);
  • Not admitted to hospital within the previous 3 weeks;
  • Body weight greater than or equal to 75% of best measurement in previous 6 months;
  • Provision of signed informed-consent to study protocol;
  • 18<Age>65

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Adults - CFPE Treatment
Other: CF Pulmonary Exacerbation (CFPE) Treatment
Other: Adults - CFPE Treatment
Hospitalization for comprehensive treatment of CF pulmonary exacerbation, including intravenous (IV) antibiotics, nutritional assessment and support, airway clearance of mucus, use of inhaled mucolytic agents and bronchodilators, glycemic control with insulin, and psychosocial support.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Serum Hepcidin-25 Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time Frame: 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.
Duration of hospitalization, an expected average of 12 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Percent-Predicted Forced Expiratory Volume in One Second (FEV1%) After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time Frame: Duration of hospitalization, an expected average of 12 days
Post- to pre-treatment ratio for FEV1% for each subject.
Duration of hospitalization, an expected average of 12 days
Change in Body Mass Index (BMI) After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time Frame: Duration of hospitalization, an expected average of 12 days
Post- to pre-treatment ratio for BMI for each subject.
Duration of hospitalization, an expected average of 12 days
Change in CFRSD-CRISS Score After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time Frame: 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.
Duration of hospitalization, an expected average of 12 days
Change in Serum Iron After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time Frame: Duration of hospitalization, an expected average of 12 days
Post- to pre-treatment ratio for serum iron concentration for each subject.
Duration of hospitalization, an expected average of 12 days
Change in Serum Interleukin-6 (IL-6) Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time Frame: Duration of hospitalization, an expected average of 12 days
Post- to pre-treatment ratio for serum interleukin-6 (IL-6) concentration for each subject.
Duration of hospitalization, an expected average of 12 days
Change in Sputum Iron Content After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time Frame: Duration of hospitalization, an expected average of 12 days
Post- to pre-treatment ratio for sputum iron content for each subject.
Duration of hospitalization, an expected average of 12 days
Change in Serum EPO Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time Frame: Duration of hospitalization, an expected average of 12 days
Post- to pre-treatment ratio for serum erythropoietin (EPO) for each subject.
Duration of hospitalization, an expected average of 12 days
Change in Transferrin Saturation After Hospitalization for CF Pulmonary Exacerbation Treatment
Time Frame: Duration of hospitalization, an expected average of 12 days
Post- to pre-treatment ratio for transferrin saturation for each subject.
Duration of hospitalization, an expected average of 12 days
Change in Serum TREM-1 Concentration After Hospitalization for CF Pulmonary Exacerbation Treatment
Time Frame: 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.
Duration of hospitalization, an expected average of 12 days
Change in Serum sIL-6R Concentration After Hospitalization for CF Pulmonary Exacerbation Treatment
Time Frame: 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.
Duration of hospitalization, an expected average of 12 days
Change in Hemoglobin Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time Frame: Duration of hospitalization, an expected average of 12 days
Post- to pre-treatment ratio for serum hemoglobin concentration for each subject.
Duration of hospitalization, an expected average of 12 days

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Sputum SDI After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time Frame: Duration of hospitalization, an expected average of 12 days
Post- to pre-treatment ratio for Simpson Diversity Index (SDI) for each subject.
Duration of hospitalization, an expected average of 12 days
Change in Sputum Pseudomonas aeruginosa Gene Expression After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time Frame: Duration of hospitalization, an expected average of 12 days
Post- to pre-treatment ratios for selected Pseudomonas aeruginosa mRNA transcript levels.
Duration of hospitalization, an expected average of 12 days
Change in Peripheral Blood Mononuclear Cell (PBMC) Gene Expression After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time Frame: Duration of hospitalization, an expected average of 12 days
Post- to pre-treatment ratios for selected mRNA transcript levels.
Duration of hospitalization, an expected average of 12 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Dartmouth-Hitchcock Medical Center

Collaborators

MaineHealth

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 1, 2014

Primary Completion (Actual)

December 1, 2017

Study Completion (Actual)

January 1, 2018

Study Registration Dates

First Submitted

July 9, 2014

First Submitted That Met QC Criteria

July 10, 2014

First Posted (Estimate)

July 14, 2014

Study Record Updates

Last Update Posted (Actual)

March 13, 2018

Last Update Submitted That Met QC Criteria

March 9, 2018

Last Verified

March 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • D14136
  • KL2TR001088 (U.S. NIH Grant/Contract)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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