Clinical trial NCT02188758: Biomarkers of Iron Homeostasis and Responses to Cystic Fibrosis Pulmonary Exacerbation (CFPE) Treatment
Official title: Do Changes in Serum Hepcidin-25 Concentration Predict Cystic Fibrosis Pulmonary Exacerbation (CFPE) Treatment Responses?
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis
Keywords: iron
hepcidin-25
pulmonary exacerbation
interleukin-6
Lead sponsor: Dartmouth-Hitchcock Medical Center
Sponsor class: OTHER
Collaborators: name: MaineHealth
First posted: 2014-07-14
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients greater than

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).


Eligibility criteria: 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

Primary outcomes:
measure: Change in Serum Hepcidin-25 Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
Secondary outcomes:
measure: Change in Percent-Predicted Forced Expiratory Volume in One Second (FEV1%) After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
measure: Change in Body Mass Index (BMI) After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
measure: Change in CFRSD-CRISS Score After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
measure: Change in Serum Iron After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
measure: Change in Serum Interleukin-6 (IL-6) Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
measure: Change in Sputum Iron Content After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
measure: Change in Serum EPO Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
measure: Change in Transferrin Saturation After Hospitalization for CF Pulmonary Exacerbation Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
measure: Change in Serum TREM-1 Concentration After Hospitalization for CF Pulmonary Exacerbation Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
measure: Change in Serum sIL-6R Concentration After Hospitalization for CF Pulmonary Exacerbation Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
measure: Change in Hemoglobin Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
Other outcomes:
measure: Change in Sputum SDI After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
measure: Change in Sputum Pseudomonas aeruginosa Gene Expression After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
measure: Change in Peripheral Blood Mononuclear Cell (PBMC) Gene Expression After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
timeFrame: Duration of hospitalization, an expected average of 12 days
Locations:
city: South Portland
country: United States
facility: Maine Medical Center
state: Maine
city: Lebanon
country: United States
facility: Dartmouth-Hitchcock Medical Center
state: New Hampshire
Study officials:
References:
citation: Gifford AH. What is hepcidin telling us about the natural history of cystic fibrosis? J Cyst Fibros. 2015 Jan;14(1):155-7. doi: 10.1016/j.jcf.2014.03.012. Epub 2014 Apr 30. No abstract available.
citation: Gifford AH, Alexandru DM, Li Z, Dorman DB, Moulton LA, Price KE, Hampton TH, Sogin ML, Zuckerman JB, Parker HW, Stanton BA, O'Toole GA. Iron supplementation does not worsen respiratory health or alter the sputum microbiome in cystic fibrosis. J Cyst Fibros. 2014 May;13(3):311-8. doi: 10.1016/j.jcf.2013.11.004. Epub 2013 Dec 13.
citation: Gifford AH. Hemoglobin </= 12.9 g/dl predicts risk of antibiotic treatment in cystic fibrosis. J Cyst Fibros. 2014 Jan;13(1):114-5. doi: 10.1016/j.jcf.2013.06.007. Epub 2013 Jul 16. No abstract available.
citation: Gifford AH, Moulton LA, Dorman DB, Olbina G, Westerman M, Parker HW, Stanton BA, O'Toole GA. Iron homeostasis during cystic fibrosis pulmonary exacerbation. Clin Transl Sci. 2012 Aug;5(4):368-73. doi: 10.1111/j.1752-8062.2012.00417.x. Epub 2012 Jun 1.
citation: Gifford AH, Miller SD, Jackson BP, Hampton TH, O'Toole GA, Stanton BA, Parker HW. Iron and CF-related anemia: expanding clinical and biochemical relationships. Pediatr Pulmonol. 2011 Feb;46(2):160-5. doi: 10.1002/ppul.21335. Epub 2010 Oct 20.