Cystic Fibrosis in ChildrenNCT03579173
Determinants of Early Cystic Fibrosis Lung Disease
An observational study of Cystic Fibrosis in Children, run by Indiana University at 1 site in United States.
Trial summary
The overall objective of this study is to determine the impact early nutritional and respiratory indices have on early CF lung disease. This knowledge will guide clinical management of infants with CF, who are now primarily diagnosed through newborn screening. Read more about this study
- Who is it for?
- Ages 1 month to 5 years
- Conditions the study is for
- Cystic Fibrosis in Children
- What is being tested?
- Infant PFT, Lung Clearance Index and Passive tidal breathing
- How many people
- 61 participants
- Where
- 1 siteUnited States
- Study dates
- 8 May 2018to 6 Feb 2019
- 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 1 month to 5 years
- All sexes
- No healthy volunteers
- Child
Study population: Infants and preschoolers with cystic fibrosis diagnosed by newborn screen.
Who may be able to join
- Informed consent by a parent or legal guardian
- A confirmed diagnosis of CF by newborn screening and either a documented sweat chloride of at least 60 mEq/L by quantitative pilocarpine iontophoresis or a genotype with two CF-causing mutations
- One month to five years of age.
Who cannot join
- Chronic lung disease not due to CF
- Contraindications to sedation, including structural abnormalities of the upper airway or chest wall and severe gastroesophageal reflux
- Gestational age < 36 weeks
Trial locations and contacts
1 site in 1 country.
United States1 site
Riley Hospital for Children
Indianapolis, Indiana, 46202
About this study
The overall objective of this study is to determine the impact early nutritional and respiratory indices have on early CF lung disease. This knowledge will guide clinical management of infants with CF, who are now primarily diagnosed through newborn screening.
Read the detailed description
The overall objective of this study is to determine the impact early nutritional and respiratory indices have on early CF lung disease. This knowledge will guide clinical management of infants with CF, who are now primarily diagnosed through newborn screening. We hypothesize that respiratory and nutritional indices during the first year of life are critical determinants of lung function in infants and preschoolers with CF.
Aim #1 - To examine the relationship between nutritional status (weight-for-age (WFA) and weight-for-length (WFL)) at 6 months of age and lung function at 1-2 years of age in infants with CF.
Hypothesis: Infants with CF with poor nutritional status at 6 months of age will have worse lung function, as assessed through the raised volume rapid thoracoabdominal compression technique and plethysmography, compared to those with better nutritional indices, defined as improved weight-for-age and weight-for-length.
Aim #2 - To examine the relationship between nutritional status (WFA and WFL) in infants with CF at 12 months of age and the lung clearance index (LCI) at 3-5 years of age.
Hypothesis: Infants with CF with poor nutritional indices at 12 months of age will have a higher (worse) LCI at 3-5 years of age compared to those with better nutritional status.
Aim #3 - To delineate the relationship between passive tidal breathing lung function testing in infants with CF at 4-8 weeks of age and subsequent lung function at 6-12 months of age.
Hypothesis: Abnormalities in passive tidal breathing lung function testing will be associated with abnormal infant pulmonary function testing obtained via the raised volume rapid thoracoabdominal compression technique and plethysmography at 6-12 months of age.
- Conditions
- Cystic Fibrosis in Children
- Being tested
- Infant PFT, Lung Clearance Index, Passive tidal breathing
- Keywords
- Nutritional Status, Infant Pulmonary Function Testing, Lung Clearance Index, Multiple Breath Washout
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Infant PFTDiagnostic test
Assessed through the raised volume rapid thoracoabdominal compression technique and plethysmography
Lung Clearance IndexDiagnostic test
The Lung Clearance Index is derived from the Multiple Breath Washout test. It is the cumulative exhaled volume (the volume of gas needed to wash out resident gas mixture out of the lungs) divided by the FRC. The FRC is the amount of air left in the lungs after normal exhalation.
Passive tidal breathingDiagnostic test
During quiet sleep, tidal breathing flow-volume curves are obtained. The tPEF/tE is calculated by taking the time it takes to reach peak expiratory flow divided by the total peak expiratory time.
| Study group | Role | What they receive |
|---|---|---|
| Nutrition and Infant PFT | To examine the relationship between nutritional status (weight-for-age (WFA) and weight-for-length (WFL)) at 6 months of age and lung function at 1-2 years of age in infants with CF. Receives: Diagnostic Test: Infant PFT | |
| Nutrition and Lung Clearance Index | To examine the relationship between nutritional status (WFA and WFL) in infants with CF at 12 months of age and the lung clearance index (LCI) at 3-5 years of age. Receives: Diagnostic Test: Lung Clearance Index | |
| Passive Tidal Breathing and Infant PFT | To delineate the relationship between passive tidal breathing lung function testing in infants with CF at 4-8 weeks of age and subsequent lung function at 6-12 months of age. Receives: Diagnostic Test: Infant PFT; Diagnostic Test: Passive tidal breathing |
What is being measured
The primary outcome is the main question the study is designed to answer.
Lung function
Forced expiratory volume in 0.5 seconds
Measured: 24 months
Secondary outcomes (2)
Lung function - FRC
Functional Residual Capacity
Measured: 24 months
Lung function - MBW
Multiple breath washout result
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jul 2018
- Study start8 May 2018
- Primary completion6 Feb 2019
- Study completion6 Feb 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis in ChildrenStudies of the same condition, open and under way.
- More trials run by Indiana UniversityThe 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
- 61 participants (actual)
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Heather Nicole Muston, Indiana University
- Investigators
- Heather Muston, MD, Principal investigator, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03579173
- Sponsor's protocol ID
- 1712581512
- Status last verified
- Jun 2022
- First submitted
- 25 Jun 2018
- Last updated
- 22 Jun 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Determinants of Early Cystic Fibrosis Lung Disease
- ClinicalTrials.gov ID
- NCT03579173
- Acronym / sponsor ID
- 1712581512
- Conditions
- Cystic Fibrosis in Children
- Keywords
- Nutritional Status, Infant Pulmonary Function Testing, Lung Clearance Index, Multiple Breath Washout
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 61 participants (actual)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Study start
- 8 May 2018 (actual)
- Primary completion
- 6 Feb 2019 (actual)
- Study completion
- 6 Feb 2019 (actual)
- First posted
- 6 Jul 2018 (actual)
- 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 overall objective of this study is to determine the impact early nutritional and respiratory indices have on early CF lung disease. This knowledge will guide clinical management of infants with CF, who are now primarily diagnosed through newborn screening.
Detailed description
The overall objective of this study is to determine the impact early nutritional and respiratory indices have on early CF lung disease. This knowledge will guide clinical management of infants with CF, who are now primarily diagnosed through newborn screening. We hypothesize that respiratory and nutritional indices during the first year of life are critical determinants of lung function in infants and preschoolers with CF.
Aim #1 - To examine the relationship between nutritional status (weight-for-age (WFA) and weight-for-length (WFL)) at 6 months of age and lung function at 1-2 years of age in infants with CF.
Hypothesis: Infants with CF with poor nutritional status at 6 months of age will have worse lung function, as assessed through the raised volume rapid thoracoabdominal compression technique and plethysmography, compared to those with better nutritional indices, defined as improved weight-for-age and weight-for-length.
Aim #2 - To examine the relationship between nutritional status (WFA and WFL) in infants with CF at 12 months of age and the lung clearance index (LCI) at 3-5 years of age.
Hypothesis: Infants with CF with poor nutritional indices at 12 months of age will have a higher (worse) LCI at 3-5 years of age compared to those with better nutritional status.
Aim #3 - To delineate the relationship between passive tidal breathing lung function testing in infants with CF at 4-8 weeks of age and subsequent lung function at 6-12 months of age.
Hypothesis: Abnormalities in passive tidal breathing lung function testing will be associated with abnormal infant pulmonary function testing obtained via the raised volume rapid thoracoabdominal compression technique and plethysmography at 6-12 months of age.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Nutrition and Infant PFT | – | Diagnostic Test: Infant PFTTo examine the relationship between nutritional status (weight-for-age (WFA) and weight-for-length (WFL)) at 6 months of age and lung function at 1-2 years of age in infants with CF. |
| Nutrition and Lung Clearance Index | – | Diagnostic Test: Lung Clearance IndexTo examine the relationship between nutritional status (WFA and WFL) in infants with CF at 12 months of age and the lung clearance index (LCI) at 3-5 years of age. |
| Passive Tidal Breathing and Infant PFT | – | Diagnostic Test: Infant PFT, Diagnostic Test: Passive tidal breathingTo delineate the relationship between passive tidal breathing lung function testing in infants with CF at 4-8 weeks of age and subsequent lung function at 6-12 months of age. |
Who may qualify
- Ages
- 1 month to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Infants and preschoolers with cystic fibrosis diagnosed by newborn screen.
Key inclusion criteria (3 listed)
- Informed consent by a parent or legal guardian
- A confirmed diagnosis of CF by newborn screening and either a documented sweat chloride of at least 60 mEq/L by quantitative pilocarpine iontophoresis or a genotype with two CF-causing mutations
- One month to five years of age.
Key exclusion criteria (3 listed)
- Chronic lung disease not due to CF
- Contraindications to sedation, including structural abnormalities of the upper airway or chest wall and severe gastroesophageal reflux
- Gestational age < 36 weeks
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 |
|---|---|---|
| Infant PFTAssessed through the raised volume rapid thoracoabdominal compression technique and plethysmography | Diagnostic test | Nutrition and Infant PFT, Passive Tidal Breathing and Infant PFT |
| Lung Clearance IndexThe Lung Clearance Index is derived from the Multiple Breath Washout test. It is the cumulative exhaled volume (the volume of gas needed to wash out resident gas mixture out of the lungs) divided by the FRC. The FRC is the amount of air left in the lungs after normal exhalation. | Diagnostic test | Nutrition and Lung Clearance Index |
| Passive tidal breathingDuring quiet sleep, tidal breathing flow-volume curves are obtained. The tPEF/tE is calculated by taking the time it takes to reach peak expiratory flow divided by the total peak expiratory time. | Diagnostic test | Passive Tidal Breathing and Infant PFT |
Primary outcome
Lung function
Time frame: 24 months
Forced expiratory volume in 0.5 seconds
2 secondary outcomes
Lung function - FRC
Time frame: 24 months
Lung function - MBW
Time frame: 24 months
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 22 Jun 2022.
- Individual participant dataSharing planned: No
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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.
Heather Muston, MD principal investigator
Indiana University
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 in Children, run by Indiana University 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 3 inclusion and 3 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 22 Jun 2022. 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?
Lung function (time frame: 24 months). 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 22 Jun 2022.