NCT02188758

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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jul 2014

Typical duration for all trials

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2014

Completed
8 days until next milestone

First Submitted

Initial submission to the registry

July 9, 2014

Completed
5 days until next milestone

First Posted

Study publicly available on registry

July 14, 2014

Completed
3.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2017

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2018

Completed
Last Updated

March 13, 2018

Status Verified

March 1, 2018

Enrollment Period

3.4 years

First QC Date

July 9, 2014

Last Update Submit

March 9, 2018

Conditions

Cystic Fibrosis

Keywords

ironhepcidin-25pulmonary exacerbationinterleukin-6

Outcome Measures

Primary Outcomes (1)

  • Change in Serum Hepcidin-25 Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment

    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 Outcomes (11)

  • 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

  • Change in Body Mass Index (BMI) After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment

    Duration of hospitalization, an expected average of 12 days

  • Change in CFRSD-CRISS Score After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment

    Duration of hospitalization, an expected average of 12 days

  • Change in Serum Iron After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment

    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

    Duration of hospitalization, an expected average of 12 days

  • +6 more secondary outcomes

Other Outcomes (3)

  • Change in Sputum SDI After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment

    Duration of hospitalization, an expected average of 12 days

  • Change in Sputum Pseudomonas aeruginosa Gene Expression After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment

    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

    Duration of hospitalization, an expected average of 12 days

Study Arms (1)

Adults - CFPE Treatment

Other: CF Pulmonary Exacerbation (CFPE) Treatment

Other: Adults - CFPE Treatment

Interventions

Adults - CFPE TreatmentOTHER

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.

Adults - CFPE Treatment

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients greater than

You may qualify if:

  • 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;
  • 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;
  • \<Age\>65

You may not qualify if:

  • Women who are pregnant or lactating;
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Maine Medical Center

South Portland, Maine, 04106, United States

Location

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

Location

Related Publications (5)

  • 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.

    PMID: 24795218BACKGROUND

  • 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.

    PMID: 24332997BACKGROUND

  • 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.

    PMID: 23867071BACKGROUND

  • 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.

    PMID: 22883617BACKGROUND

  • 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.

    PMID: 20963784BACKGROUND

Biospecimen

Retention: SAMPLES WITH DNA

Bacterial DNA and RNA will be isolated from sputum samples and stored for future analyses.

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Associate Professor of Medicine

Study Record Dates

First Submitted

July 9, 2014

First Posted

July 14, 2014

Study Start

July 1, 2014

Primary Completion

December 1, 2017

Study Completion

January 1, 2018

Last Updated

March 13, 2018

Record last verified: 2018-03

Locations

US(2)