Cystic FibrosisNCT02188758
Biomarkers of Iron Homeostasis and Responses to Cystic Fibrosis Pulmonary Exacerbation (CFPE) Treatment
An observational study of Cystic Fibrosis, run by Dartmouth-Hitchcock Medical Center at 2 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Adults - CFPE Treatment
- Type of treatment
- Injection or infusion
- How many people
- 20 participants
- Where
- 2 sitesUnited States
- Study dates
- Jul 2014to Jan 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients greater than
Who may be able to join
- 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;
Show all 9Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, 03756
Maine Medical Center
South Portland, Maine, 04106
About this study
Official title: 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).
- Conditions
- Cystic Fibrosis
- Being tested
- Adults - CFPE Treatment
- Keywords
- iron, hepcidin-25, pulmonary exacerbation, interleukin-6
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| Adults - CFPE Treatment | Other: CF Pulmonary Exacerbation (CFPE) Treatment Receives: Other: Adults - CFPE Treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Post- to pre-treatment ratio for FEV1% for each subject.
Measured: Duration of hospitalization, an expected average of 12 days
Change in Body Mass Index (BMI) After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Post- to pre-treatment ratio for BMI for each subject.
Measured: Duration of hospitalization, an expected average of 12 days
Change in CFRSD-CRISS Score After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
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.
Measured: Duration of hospitalization, an expected average of 12 days
Change in Serum Iron After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Post- to pre-treatment ratio for serum iron concentration for each subject.
Measured: 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
Post- to pre-treatment ratio for serum interleukin-6 (IL-6) concentration for each subject.
Measured: Duration of hospitalization, an expected average of 12 days
Change in Sputum Iron Content After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Post- to pre-treatment ratio for sputum iron content for each subject.
Measured: Duration of hospitalization, an expected average of 12 days
Change in Serum EPO Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Post- to pre-treatment ratio for serum erythropoietin (EPO) for each subject.
Measured: Duration of hospitalization, an expected average of 12 days
Change in Transferrin Saturation After Hospitalization for CF Pulmonary Exacerbation Treatment
Post- to pre-treatment ratio for transferrin saturation for each subject.
Measured: Duration of hospitalization, an expected average of 12 days
Change in Serum TREM-1 Concentration After Hospitalization for CF Pulmonary Exacerbation Treatment
Post- to pre-treatment ratio for serum triggering receptor expressed on myeloid cells-1 (TREM-1) for each subject.
Measured: Duration of hospitalization, an expected average of 12 days
Change in Serum sIL-6R Concentration After Hospitalization for CF Pulmonary Exacerbation Treatment
Post- to pre-treatment ratio for serum soluble IL-6 receptor (sIL-6R) concentration for each subject.
Measured: Duration of hospitalization, an expected average of 12 days
Change in Hemoglobin Concentration After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Post- to pre-treatment ratio for serum hemoglobin concentration for each subject.
Measured: Duration of hospitalization, an expected average of 12 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jul 2014 (estimated)
- Study startJul 2014
- Primary completionDec 2017
- Study completionJan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Dartmouth-Hitchcock Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dartmouth-Hitchcock Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- MaineHealth
- Responsible party
- Principal investigator, Alex H. Gifford, Dartmouth-Hitchcock Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02188758
- Sponsor's protocol ID
- D14136
- Other IDs
- KL2TR001088
- Status last verified
- Mar 2018
- First submitted
- 9 Jul 2014
- Last updated
- 13 Mar 2018
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 24795218Gifford 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.
PubMed 24332997Gifford 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.
PubMed 23867071Gifford 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.
PubMed 22883617Gifford 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.
PubMed 20963784
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Do Changes in Serum Hepcidin-25 Concentration Predict Cystic Fibrosis Pulmonary Exacerbation (CFPE) Treatment Responses?
- ClinicalTrials.gov ID
- NCT02188758
- Acronym / sponsor ID
- D14136
- Conditions
- Cystic Fibrosis
- Keywords
- iron, hepcidin-25, pulmonary exacerbation, interleukin-6
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Dartmouth-Hitchcock Medical CenterUniversity, hospital or other
- Collaborators
- MaineHealth
- Study start
- Jul 2014 (actual)
- Primary completion
- Dec 2017 (actual)
- Study completion
- Jan 2018 (actual)
- First posted
- 14 Jul 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Adults - CFPE Treatment | – | Other: Adults - CFPE TreatmentOther: CF Pulmonary Exacerbation (CFPE) Treatment |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients greater than
Key inclusion criteria (9 listed)
- 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);
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Adults - CFPE TreatmentHospitalization 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. | Other | Adults - CFPE Treatment |
Primary outcome
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.
11 secondary outcomes
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
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
Change in CFRSD-CRISS Score After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time frame: 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
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
Change in Sputum Iron Content After Hospitalization for CF Pulmonary Exacerbation (CFPE) Treatment
Time frame: 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
Change in Transferrin Saturation After Hospitalization for CF Pulmonary Exacerbation Treatment
Time frame: 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
Change in Serum sIL-6R Concentration After Hospitalization for CF Pulmonary Exacerbation Treatment
Time frame: 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2018.
- Background: PubMed 24795218Gifford 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.
- Background: PubMed 24332997Gifford 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.
- Background: PubMed 23867071Gifford 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.
- Background: PubMed 22883617Gifford 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.
- Background: PubMed 20963784Gifford 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Dartmouth-Hitchcock Medical Center at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02188758, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Mar 2018.