Cystic FibrosisPhase 1NCT02947126
Multilevel Models of Therapeutic Response in the Lungs
A Phase 1 study testing Hypertonic Saline, Isotonic Saline and Indium-DTPA in people with Cystic Fibrosis, run by Tim Corcoran at 1 site in United States.
Trial summary
When developing new medications for lung diseases like Cystic Fibrosis (CF), scientists perform lab experiments using cells from the airways, physiology studies of how the lungs change when a drug is given, and clinical studies to determine how drugs affect overall health. Read more about this study
- Who is it for?
- Ages 12 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Hypertonic Saline, Isotonic Saline and Indium-DTPA
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2017to Dec 2022
- Phase
- Phase 1
- 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 12 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Cystic Fibrosis Subjects: Inclusion Criteria
- Ages 12 or older
- Diagnosis of cystic fibrosis as determined by sweat test or genotype
- Clinically stable as determined by a physician co-investigator
Cystic Fibrosis Subjects: Exclusion Criteria
- Smokers or users of electronic cigarettes
- FEV1%p <30% of predicted
- Nursing, pregnant or unwilling to test for pregnancy
- Intolerant to hypertonic saline
- Unable or unwilling to discontinue hypertonic saline, Pulmozyme, and long acting bronchodilators for 24 hrs before testing and short acting bronchodilators on testing days.
CF parents: Inclusion Criteria
- Ages 18 and older
- Biological parent of a CF patient who is also enrolled in the study
CF parents: Exclusion Criteria
- Smokers or users of electronic cigarettes
- FEV1%p <30% of predicted
- Nursing, pregnant or unwilling to test for pregnancy
- Unwilling to discontinue long acting bronchodilators for 24 hrs before testing and short acting bronchodilators on testing days.
- Unwilling to perform CFTR genotyping.
Healthy controls: Inclusion Criteria
- Ages 18 and older
- No history of lung disease
Healthy Controls: Exclusion Criteria
- Smokers or users of electronic cigarettes
- FEV1%p <70% of predicted
- Nursing, pregnant or unwilling to test for pregnancy
- Carriers of known disease causing CFTR mutations
- Unwilling to perform CFTR genotyping.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
About this study
When developing new medications for lung diseases like Cystic Fibrosis (CF), scientists perform lab experiments using cells from the airways, physiology studies of how the lungs change when a drug is given, and clinical studies to determine how drugs affect overall health. The investigators of this study are seeking to develop computer models that will predict how patients will respond to drugs by just doing lab studies on cell samples from their noses. Such models would allow for medications to be developed more rapidly for all patients and allow treatments to be personalized as well. In order to develop these computer models a series of tests will be performed on patients who have CF. Tests will include sampling cells from the nose and measuring lung physiology using a combination of different imaging, breathing, and other studies performed both before and after participants take a therapy. Similar tests will be performed on people who do not have CF, and on the parents of the CF participants who carry a single CF gene because this will provide information on how specific genes might affect CF lung disease.
Read the detailed description
The goal of this research is to develop a series of interconnected models of therapeutic response in the diseased lung, focused primarily on Cystic Fibrosis (CF), that will ultimately provide a means for predicting in vivo response based on patient-specific in vitro testing, allowing for the optimization and personalization of therapies. Investigators use both human bronchial epithelial (HBE) and more recently human nasal epithelial (HNE) cell cultures to study CF pathophysiology. The investigators performing this study have also developed functional imaging biomarkers in the lung that provide organ level quantification of CF lung physiology (mucociliary clearance and airway liquid absorption), and, more recently, in silico systems models of lung physiology at both the cell and organ level. The in silico models provide a framework of differential equations that describe how basic physiological processes interact and contribute to experimental outcomes. Their use allows these mechanisms to be more specifically differentiated. Here the investigators propose to link in vitro and in vivo response by sampling and culturing HNE cell cultures from both non-CF and CF subjects who will also perform a series of physiological assessments, including functional imaging scans. The in silico models will facilitate linking therapeutic studies in cells to therapeutic outcomes in patients.
- CF PATIENTS will perform 2 study days. Study day 1 will include:
- nasal potential difference measurements
- pulmonary function testing
- inert gas washout testing
- urine pregnancy testing
- nasal cell sampling
- nuclear MCC/ABS scan (to include inhalation of isotonic or hypertonic saline - randomized order)
- blood draw for CFTR genotyping if not already available. Study day 2 will include
- pulmonary function testing
- urine pregnancy testing
- nuclear MCC/ABS scan (to include inhalation of isotonic or hypertonic saline - randomized order)
- PARENTS OF ENROLLED CF patients who choose to participate will perform 1 study day which will include:
- nasal potential difference measurements
- pulmonary function testing
- inert gas washout testing
- urine pregnancy testing
- nasal cell sampling
- nuclear MCC/ABS scan (to include inhalation of isotonic saline)
- a single blood sample drawn for CFTR genotyping.
- HEALTHY CONTROLS will perform 1 screening and 1 study day which will include:
- pulmonary function testing
- inert gas washout testing
- urine pregnancy testing
- nasal cell sampling
- nuclear MCC/ABS scan (to include inhalation of isotonic saline)
- a single blood sample drawn for CFTR genotyping (at screening).
- Conditions
- Cystic Fibrosis
- Being tested
- Hypertonic Saline, Isotonic Saline, Indium-DTPA, Technetium Sulfur Colloid
- Keywords
- cystic fibrosis, mucociliary clearance, CFTR
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hypertonic SalineDrug
Inhaled 7% Hypertonic saline
Isotonic SalineDrug
Inhaled 0.9% Isotonic saline
Indium-DTPADrug
Inhaled Indium 111 DTPA
Technetium Sulfur ColloidDrug
Inhaled Technetium99m sulfur colloid
| Study group | Role | What they receive |
|---|---|---|
| Cystic Fibrosis (HS, IS) | Experimental | CF subjects: ages 12 or older with a diagnosis of cystic fibrosis as determined by sweat test or genotype and clinical symptoms who are clinically stable as determined by a physician co-investigator. Subjects receive HS dose on first imaging day and IS dose on the second imaging day,
Indium-DTPA (1.5 mCi), Technetium sulfur colloid (8mCi), Inhaled isotonic saline (4ml), Inhaled Hypertonic Saline (4ml) Receives: Drug: Hypertonic Saline; Drug: Isotonic Saline; Drug: Indium-DTPA; Drug: Technetium Sulfur Colloid |
| Cystic Fibrosis (IS, HS) | Experimental | CF subjects: ages 12 or older with a diagnosis of cystic fibrosis as determined by sweat test or genotype and clinical symptoms who are clinically stable as determined by a physician co-investigator. Subjects receive IS dose on first imaging day and HS dose on the second imaging day.
Indium-DTPA (1.5 mCi), Technetium sulfur colloid (8mCi), Inhaled isotonic saline (4ml), Inhaled Hypertonic Saline (4ml) Receives: Drug: Hypertonic Saline; Drug: Isotonic Saline; Drug: Indium-DTPA; Drug: Technetium Sulfur Colloid |
| Parents of CF subjects | Experimental | Ages 18 and older, biological parent of a CF patient who is also enrolled in the study
Indium-DTPA (1.5 mCi), Technetium sulfur colloid (8mCi), Inhaled isotonic saline (4ml) Receives: Drug: Isotonic Saline; Drug: Indium-DTPA; Drug: Technetium Sulfur Colloid |
| non CF controls | Experimental | Ages 18 and older with no history of lung disease
Indium-DTPA (1.5 mCi), Technetium sulfur colloid (8mCi), Inhaled isotonic saline (4ml). Receives: Drug: Isotonic Saline; Drug: Indium-DTPA; Drug: Technetium Sulfur Colloid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mucociliary Clearance
Clearance rate of Technetium sulfur colloid from the lungs
Measured: 80 minutes
DTPA absorption rate
DTPA absorption rate from the lungs (difference between total In-DTPA clearance rate and mucociliary clearance rate)
Measured: 80 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2016 (estimated)
- Study startJan 2017
- Primary completionFeb 2020
- Study completionDec 2022
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.
- 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tim Corcoran
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor-investigator, Tim Corcoran, University of Pittsburgh
- Investigators
- Tim Corcoran, Ph.D., Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02947126
- Sponsor's protocol ID
- PRO15070376
- Other IDs
- 1U01HL131046-01
- Status last verified
- Jan 2023
- First submitted
- 25 Oct 2016
- Last updated
- 13 Jan 2023
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multilevel Models of Therapeutic Response in the Lungs
- ClinicalTrials.gov ID
- NCT02947126
- Acronym / sponsor ID
- PRO15070376
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, mucociliary clearance, CFTR
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 56 participants (actual)
- Lead sponsor
- Tim CorcoranUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Jan 2017 (actual)
- Primary completion
- Feb 2020 (actual)
- Study completion
- Dec 2022 (actual)
- First posted
- 27 Oct 2016 (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.
When developing new medications for lung diseases like Cystic Fibrosis (CF), scientists perform lab experiments using cells from the airways, physiology studies of how the lungs change when a drug is given, and clinical studies to determine how drugs affect overall health. The investigators of this study are seeking to develop computer models that will predict how patients will respond to drugs by just doing lab studies on cell samples from their noses. Such models would allow for medications to be developed more rapidly for all patients and allow treatments to be personalized as well. In order to develop these computer models a series of tests will be performed on patients who have CF. Tests will include sampling cells from the nose and measuring lung physiology using a combination of different imaging, breathing, and other studies performed both before and after participants take a therapy. Similar tests will be performed on people who do not have CF, and on the parents of the CF participants who carry a single CF gene because this will provide information on how specific genes might affect CF lung disease.
Detailed description
The goal of this research is to develop a series of interconnected models of therapeutic response in the diseased lung, focused primarily on Cystic Fibrosis (CF), that will ultimately provide a means for predicting in vivo response based on patient-specific in vitro testing, allowing for the optimization and personalization of therapies. Investigators use both human bronchial epithelial (HBE) and more recently human nasal epithelial (HNE) cell cultures to study CF pathophysiology. The investigators performing this study have also developed functional imaging biomarkers in the lung that provide organ level quantification of CF lung physiology (mucociliary clearance and airway liquid absorption), and, more recently, in silico systems models of lung physiology at both the cell and organ level. The in silico models provide a framework of differential equations that describe how basic physiological processes interact and contribute to experimental outcomes. Their use allows these mechanisms to be more specifically differentiated. Here the investigators propose to link in vitro and in vivo response by sampling and culturing HNE cell cultures from both non-CF and CF subjects who will also perform a series of physiological assessments, including functional imaging scans. The in silico models will facilitate linking therapeutic studies in cells to therapeutic outcomes in patients.
- CF PATIENTS will perform 2 study days. Study day 1 will include:
- nasal potential difference measurements
- pulmonary function testing
- inert gas washout testing
- urine pregnancy testing
- nasal cell sampling
- nuclear MCC/ABS scan (to include inhalation of isotonic or hypertonic saline - randomized order)
- blood draw for CFTR genotyping if not already available. Study day 2 will include
- pulmonary function testing
- urine pregnancy testing
- nuclear MCC/ABS scan (to include inhalation of isotonic or hypertonic saline - randomized order)
- PARENTS OF ENROLLED CF patients who choose to participate will perform 1 study day which will include:
- nasal potential difference measurements
- pulmonary function testing
- inert gas washout testing
- urine pregnancy testing
- nasal cell sampling
- nuclear MCC/ABS scan (to include inhalation of isotonic saline)
- a single blood sample drawn for CFTR genotyping.
- HEALTHY CONTROLS will perform 1 screening and 1 study day which will include:
- pulmonary function testing
- inert gas washout testing
- urine pregnancy testing
- nasal cell sampling
- nuclear MCC/ABS scan (to include inhalation of isotonic saline)
- a single blood sample drawn for CFTR genotyping (at screening).
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cystic Fibrosis (HS, IS) | Experimental | Drug: Hypertonic Saline, Drug: Isotonic Saline, Drug: Indium-DTPA, Drug: Technetium Sulfur ColloidCF subjects: ages 12 or older with a diagnosis of cystic fibrosis as determined by sweat test or genotype and clinical symptoms who are clinically stable as determined by a physician co-investigator. Subjects receive HS dose on first imaging day and IS dose on the second imaging day, Indium-DTPA (1.5 mCi), Technetium sulfur colloid (8mCi), Inhaled isotonic saline (4ml), Inhaled Hypertonic Saline (4ml) |
| Cystic Fibrosis (IS, HS) | Experimental | Drug: Hypertonic Saline, Drug: Isotonic Saline, Drug: Indium-DTPA, Drug: Technetium Sulfur ColloidCF subjects: ages 12 or older with a diagnosis of cystic fibrosis as determined by sweat test or genotype and clinical symptoms who are clinically stable as determined by a physician co-investigator. Subjects receive IS dose on first imaging day and HS dose on the second imaging day. Indium-DTPA (1.5 mCi), Technetium sulfur colloid (8mCi), Inhaled isotonic saline (4ml), Inhaled Hypertonic Saline (4ml) |
| Parents of CF subjects | Experimental | Drug: Isotonic Saline, Drug: Indium-DTPA, Drug: Technetium Sulfur ColloidAges 18 and older, biological parent of a CF patient who is also enrolled in the study Indium-DTPA (1.5 mCi), Technetium sulfur colloid (8mCi), Inhaled isotonic saline (4ml) |
| non CF controls | Experimental | Drug: Isotonic Saline, Drug: Indium-DTPA, Drug: Technetium Sulfur ColloidAges 18 and older with no history of lung disease Indium-DTPA (1.5 mCi), Technetium sulfur colloid (8mCi), Inhaled isotonic saline (4ml). |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| Hypertonic SalineInhaled 7% Hypertonic saline | Drug | Cystic Fibrosis (HS, IS), Cystic Fibrosis (IS, HS) |
| Isotonic SalineInhaled 0.9% Isotonic saline | Drug | Cystic Fibrosis (HS, IS), Cystic Fibrosis (IS, HS), Parents of CF subjects, non CF controls |
| Indium-DTPAInhaled Indium 111 DTPA | Drug | Cystic Fibrosis (HS, IS), Cystic Fibrosis (IS, HS), Parents of CF subjects, non CF controls |
| Technetium Sulfur ColloidInhaled Technetium99m sulfur colloid | Drug | Cystic Fibrosis (HS, IS), Cystic Fibrosis (IS, HS), Parents of CF subjects, non CF controls |
Primary outcome
Mucociliary Clearance
Time frame: 80 minutes
Clearance rate of Technetium sulfur colloid from the lungs
DTPA absorption rate
Time frame: 80 minutes
DTPA absorption rate from the lungs (difference between total In-DTPA clearance rate and mucociliary clearance rate)
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 Jan 2023.
- 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.
Tim Corcoran, Ph.D. principal investigator
University of Pittsburgh
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Hypertonic Saline, Isotonic Saline and Indium-DTPA in people with Cystic Fibrosis, run by Tim Corcoran 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?
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 Jan 2023. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Mucociliary Clearance (time frame: 80 minutes). The study lists 2 primary outcomes in all. 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 13 Jan 2023.