Cystic FibrosisNCT01805713
Biomarkers of Injury and Destruction in the Cystic Fibrosis Lung
An observational study of Cystic Fibrosis, run by University of Minnesota at 1 site in United States.
Trial summary
Cystic fibrosis (CF) is the most common autosomal recessive genetic disease in Caucasians. It results in lung disease that affects quality of life and causes early death. Lung damage from CF starts in infancy and continues over time. Lung damage can negatively affect how the lung functions. Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 150 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2012to Dec 2017
- 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 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Infants with Cystic Fibrosis Diagnosed by Newborn Screen. Subjects with Cystic Fibrosis => 8 Years of Age.
Who may be able to join
None listed.
Who cannot join
- Age over 6months
- Child Study Inclusion Criteria
- FEV1 < 35% predicted A
- Use of IV antibiotics for a pulmonary exacerbation or respiratory symptoms 12 months prior to enrollment.
- Hospitalization for a pulmonary exacerbation 12 months prior to enrollment.
- Use of oral antibiotic for respiratory symptoms 28 days prior to enrollment.
Show all 12Show fewer
- Any changes in medical regimen for treatment of CF (e.g. no addition of or elimination of therapies such as hypertonic saline, inhaled corticosteroids or mucolytic therapy) within 28 days of enrollment visit.
- FEV1 < 35% predicted A
- Use of IV antibiotics for a pulmonary exacerbation or respiratory symptoms 12 months prior to enrollment.
- Hospitalization for a pulmonary exacerbation 12 months prior to enrollment.
- Use of oral antibiotic for respiratory symptoms 28 days prior to enrollment.
- Any changes in medical regimen for treatment of CF (e.g. no addition of or elimination of therapies such as hypertonic saline, inhaled corticosteroids or mucolytic therapy) within 28 days of enrollment visit.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota
Minneapolis, Minnesota, 55455
About this study
Cystic fibrosis (CF) is the most common autosomal recessive genetic disease in Caucasians. It results in lung disease that affects quality of life and causes early death. Lung damage from CF starts in infancy and continues over time. Lung damage can negatively affect how the lung functions. It would be ideal to measure lung damage in CF patients in three instances: (1) During the first year of life after diagnosis by state newborn screening programs, (2) In children and adults over long periods of time (years), and (3) During times of illness (pulmonary exacerbation), to allow for better treatment and therapy to prevent loss of lung function. The lung is made of elastin, collagen and cartilage. When the lung is damaged by CF, these components break down into pieces that can be measured in urine, sputum and blood. These products may represent markers of lung injury. We believe that the levels of these markers will be increased over time in CF patients and even higher in patients who are sick with lung symptoms. The goal of my research is to measure the amount of lung breakdown products in urine, sputum and blood in infants, children and adults with CF during times when well and also during times of illness. I also hope to use new technologies involving the study of proteins and metabolites in samples like sputum, urine and blood to help provide new information regarding CF lung disease. These studies will help us to better treat CF lung disease.
Read the detailed description
This is a prospective, observational cohort study of two CF populations (and appropriate control populations):
Infants with CF Diagnosed by Newborn Screen: The purpose of this prospective cohort study is to measure concentrations of biomarkers of lung injury in urine, blood and bronchial alveolar lavage fluid (BALF) from CF infants. We plan to characterize these concentrations during the first two years of life with the goal of determining if their profiles alone or in combination are potentially useful biomarkers of early lung disease in CF.
Each CF infant enrolled has blood, urine and BALF samples, along with an oropharyngeal swab (throat culture) in conjunction with infant lung function testing at approximately 6 months of age and again at one year. Our CF Center performs infant Pulmonary Function Tests (PFT's), throat culture and a blood draw as part of routine care at 6 months and at one year of age, so the bronchoscopy and urine collection are obtained for research purposes only. Urine is collected quarterly during routine outpatient clinic visits to the CF Center (4 samples total/year for two years) which is also for research purposes only. During the second year of the study, urine, blood and throat swab sample collection plus lung function testing continue as per year one. In the event of a hospitalization for pulmonary exacerbation, blood and urine samples are obtained at three time points during hospitalization and BALF samples are obtained via bronchoscopy upon admission. An oropharyngeal culture is also obtained upon admission. Subsequent hospitalizations for pulmonary exacerbation are recorded with the same samples collected as described above. In the event of an outpatient exacerbation requiring antibiotic therapy, the patient will be asked to provide a urine sample at the onset of exacerbation. Normal CF treatment or care is not altered in any way by participation in this study.
We collect urine only from age-matched healthy infants as a control group for comparison.
Subjects with CF ≥ 8 Years of Age: The purpose of this prospective cohort study is to measure concentrations of biomarkers of lung injury in urine, blood and sputum from CF patients. This is an observational study that does not involve a specific treatment or intervention. We plan to characterize these concentrations during two years of both clinically stable time points as well during hospitalizations and outpatient treatments for pulmonary exacerbations. The study goal is determining if these profiles alone or in combination are potentially useful biomarkers of early lung disease in CF.
All study participants (CF Exacerbation Group and CF Non-Exacerbation Group) are asked to provide urine, blood and sputum samples, as well as pulmonary function tests, during their regularly scheduled quarterly CF Clinic visits over the study period. Quarterly lung function testing is standard of care for all CF patients. For those patients in the CF Exacerbation group, subjects are asked to provide three urine, blood and sputum samples as well as three sets of pulmonary function before during and after an inpatient hospitalization for a pulmonary exacerbation. We incorporate PFT's, and labs done as part of their hospital admission whenever possible, to avoid duplicate testing. All hospitalized patients will receive their routine, standard-of-care therapies including airway clearance, nutritional support, IV antibiotics and other pulmonary medications. Treatment or care is not be altered in any way by participation in this study. If a patient in the CF Non-Exacerbation Group experiences a pulmonary exacerbation during the study period, samples will be obtained as described for the CF Exacerbation Group.
For a control group comparison we collect sputum and urine only from volunteers > 8 years of age who do not have CF.
- Conditions
- Cystic Fibrosis
- Keywords
- Biomarkers
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| CF Infant Cohort | Infants with CF followed for the first two years of life. | |
| CF Child/Adult Cohort | CF patients > 8 years of age followed during hospitalization for pulmonary exacerbation and when well for two years. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Biomarkes of Lung Injury
Desmosine,Clara Cell Protein(CC10) and cathepsin B concentration in biological specimens.
Measured: Two Years
Secondary outcomes (2)
Pulmonary Function
Forced expiratory volume (time) (FEV1) and FEV0.5 decline over two years.
Measured: Two Years
Pulmonary Exacerbations
The number of CF pulmonary exacerbations.
Measured: Two years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2013 (estimated)
- Study startDec 2012
- Primary completionDec 2017
- Study completionDec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University of MinnesotaThe 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
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Minnesota
- Sponsor type
- University, hospital or other
- Collaborators
- National Institutes of Health (NIH), Minnesota Medical Foundation, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Responsible party
- Sponsor
- Investigators
- Theresa A Laguna, MD, Principal investigator, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01805713
- Sponsor's protocol ID
- 1210M22861
- Other IDs
- K12HD068322
- Status last verified
- Jan 2018
- First submitted
- 4 Mar 2013
- Last updated
- 12 Jan 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Biomarkers of Injury and Destruction in the Cystic Fibrosis Lung
- ClinicalTrials.gov ID
- NCT01805713
- Acronym / sponsor ID
- 1210M22861
- Conditions
- Cystic Fibrosis
- Keywords
- Biomarkers
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- University of MinnesotaUniversity, hospital or other
- Collaborators
- National Institutes of Health (NIH), Minnesota Medical Foundation, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Study start
- Dec 2012
- Primary completion
- Dec 2017 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 6 Mar 2013 (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.
Cystic fibrosis (CF) is the most common autosomal recessive genetic disease in Caucasians. It results in lung disease that affects quality of life and causes early death. Lung damage from CF starts in infancy and continues over time. Lung damage can negatively affect how the lung functions. It would be ideal to measure lung damage in CF patients in three instances: (1) During the first year of life after diagnosis by state newborn screening programs, (2) In children and adults over long periods of time (years), and (3) During times of illness (pulmonary exacerbation), to allow for better treatment and therapy to prevent loss of lung function. The lung is made of elastin, collagen and cartilage. When the lung is damaged by CF, these components break down into pieces that can be measured in urine, sputum and blood. These products may represent markers of lung injury. We believe that the levels of these markers will be increased over time in CF patients and even higher in patients who are sick with lung symptoms. The goal of my research is to measure the amount of lung breakdown products in urine, sputum and blood in infants, children and adults with CF during times when well and also during times of illness. I also hope to use new technologies involving the study of proteins and metabolites in samples like sputum, urine and blood to help provide new information regarding CF lung disease. These studies will help us to better treat CF lung disease.
Detailed description
This is a prospective, observational cohort study of two CF populations (and appropriate control populations):
Infants with CF Diagnosed by Newborn Screen: The purpose of this prospective cohort study is to measure concentrations of biomarkers of lung injury in urine, blood and bronchial alveolar lavage fluid (BALF) from CF infants. We plan to characterize these concentrations during the first two years of life with the goal of determining if their profiles alone or in combination are potentially useful biomarkers of early lung disease in CF.
Each CF infant enrolled has blood, urine and BALF samples, along with an oropharyngeal swab (throat culture) in conjunction with infant lung function testing at approximately 6 months of age and again at one year. Our CF Center performs infant Pulmonary Function Tests (PFT's), throat culture and a blood draw as part of routine care at 6 months and at one year of age, so the bronchoscopy and urine collection are obtained for research purposes only. Urine is collected quarterly during routine outpatient clinic visits to the CF Center (4 samples total/year for two years) which is also for research purposes only. During the second year of the study, urine, blood and throat swab sample collection plus lung function testing continue as per year one. In the event of a hospitalization for pulmonary exacerbation, blood and urine samples are obtained at three time points during hospitalization and BALF samples are obtained via bronchoscopy upon admission. An oropharyngeal culture is also obtained upon admission. Subsequent hospitalizations for pulmonary exacerbation are recorded with the same samples collected as described above. In the event of an outpatient exacerbation requiring antibiotic therapy, the patient will be asked to provide a urine sample at the onset of exacerbation. Normal CF treatment or care is not altered in any way by participation in this study.
We collect urine only from age-matched healthy infants as a control group for comparison.
Subjects with CF ≥ 8 Years of Age: The purpose of this prospective cohort study is to measure concentrations of biomarkers of lung injury in urine, blood and sputum from CF patients. This is an observational study that does not involve a specific treatment or intervention. We plan to characterize these concentrations during two years of both clinically stable time points as well during hospitalizations and outpatient treatments for pulmonary exacerbations. The study goal is determining if these profiles alone or in combination are potentially useful biomarkers of early lung disease in CF.
All study participants (CF Exacerbation Group and CF Non-Exacerbation Group) are asked to provide urine, blood and sputum samples, as well as pulmonary function tests, during their regularly scheduled quarterly CF Clinic visits over the study period. Quarterly lung function testing is standard of care for all CF patients. For those patients in the CF Exacerbation group, subjects are asked to provide three urine, blood and sputum samples as well as three sets of pulmonary function before during and after an inpatient hospitalization for a pulmonary exacerbation. We incorporate PFT's, and labs done as part of their hospital admission whenever possible, to avoid duplicate testing. All hospitalized patients will receive their routine, standard-of-care therapies including airway clearance, nutritional support, IV antibiotics and other pulmonary medications. Treatment or care is not be altered in any way by participation in this study. If a patient in the CF Non-Exacerbation Group experiences a pulmonary exacerbation during the study period, samples will be obtained as described for the CF Exacerbation Group.
For a control group comparison we collect sputum and urine only from volunteers > 8 years of age who do not have CF.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| CF Infant Cohort | – | –Infants with CF followed for the first two years of life. |
| CF Child/Adult Cohort | – | –CF patients > 8 years of age followed during hospitalization for pulmonary exacerbation and when well for two years. |
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Infants with Cystic Fibrosis Diagnosed by Newborn Screen. Subjects with Cystic Fibrosis => 8 Years of Age.
Key inclusion criteria (0 listed)
Key exclusion criteria (12 listed)
- Age over 6months
- Child Study Inclusion Criteria
- FEV1 < 35% predicted A
- Use of IV antibiotics for a pulmonary exacerbation or respiratory symptoms 12 months prior to enrollment.
- Hospitalization for a pulmonary exacerbation 12 months prior to enrollment.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Biomarkes of Lung Injury
Time frame: Two Years
Desmosine,Clara Cell Protein(CC10) and cathepsin B concentration in biological specimens.
2 secondary outcomes
Pulmonary Function
Time frame: Two Years
Pulmonary Exacerbations
Time frame: Two years
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 12 Jan 2018.
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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.
Theresa A Laguna, MD principal investigator
University of Minnesota
1 site 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 University of Minnesota at 1 site 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 0 inclusion and 12 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 12 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Biomarkes of Lung Injury (time frame: Two Years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 12 Jan 2018.