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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Dartmouth-Hitchcock Medical Center

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 1, 2014

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2017

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 1, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 9, 2014

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 10, 2014

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 14, 2014

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 13, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 9, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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