Cystic FibrosisPhase 1NCT01887197
Repeatability and Response Study of Absorptive Clearance Scans
A Phase 1 study testing Absorptive clearance scan, inhaled hypertonic saline (7%) and mannitol inhalation powder in people with Cystic Fibrosis, run by Tim Corcoran at 1 site in United States.
Trial summary
The investigators are developing a new nuclear medicine imaging technique for measuring liquid absorption in the airways that can be applied to screen new medications being developed to treat cystic fibrosis (CF). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Absorptive clearance scan, inhaled hypertonic saline (7%) and mannitol inhalation powder
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2013to May 2018
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- subjects 18 years old 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
Who cannot join
- one second forced expiratory volume (FEV1) <50% of predicted
- nursing mother
- positive urine pregnancy test or unwilling to test
- cigarette smoker
- unwilling to stop hypertonic saline therapy for 72 hours prior to each test day
- are intolerant to hypertonic saline (response only)
Show all 10Show fewer
- are intolerant to any inhaled therapies (response only)
- fail mannitol tolerance testing (response only)
- have a history of excessive (uncontrollable) coughing after an osmotic stimulus (response only)
- have a history of hemoptysis (response only)
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
About this study
The investigators are developing a new nuclear medicine imaging technique for measuring liquid absorption in the airways that can be applied to screen new medications being developed to treat cystic fibrosis (CF). The investigators believe that the absorption of the small molecule radiopharmaceutical Indium 111 diethylene triamine pentaacetic acid (In-DTPA) will indicate changes in liquid absorption in the airways and demonstrate whether new CF medications will be effective. In this study the investigators will determine whether the imaging technique will demonstrate similar results when it is repeated on different days. They will also determine how their results change when subjects utilize several common CF medications.
Read the detailed description
Cystic Fibrosis (CF) is an autosomal recessive genetic disease, caused by mutations of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) that impairs ion transport at epithelial surfaces. This results in the accumulation of dehydrated secretions in the airways and chronic infection and inflammation in the lungs, leading to significant morbidity and mortality. The investigators understanding of CF pathogenesis has increased substantially and many new targeted therapies are being developed to treat this disease, however, the measurements of clinical efficacy used to evaluate these therapies require long trials to demonstrate an effect. New translational techniques are needed to assess changes in the most basic aspects of the disease and allow for the rapid screening of disease-altering therapies. The investigators have recently developed a novel aerosol-based imaging technique to measure liquid absorption in the airways - a central pathophysiological process related to CFTR dysfunction. The investigators propose that airway liquid hyper-absorption is a key link between cellular defects in ion and fluid transport and progressive airway dysfunction in CF. Thus The investigators technique may provide a measure of disease severity and rapid indication of therapeutic correction in advance of currently available outcome measures.
- Conditions
- Cystic Fibrosis
- Being tested
- Absorptive clearance scan, inhaled hypertonic saline (7%), mannitol inhalation powder
- Keywords
- cystic fibrosis, mucociliary clearance, airway surface liquid, nuclear medicine, mannitol
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Absorptive clearance scanOther
Subjects inhale a nebulized mixture of the radiopharmaceuticals Indium 111-DTPA and Technetium 99m sulfur colloid.
inhaled hypertonic saline (7%)Drug
Also called Hypersal
nebulized hypertonic saline (7%)
mannitol inhalation powderDrug
Also called Bronchitol
mannitol inhalation powder
| Study group | Role | What they receive |
|---|---|---|
| Repeatability | Experimental | Subjects perform two sequential absorptive clearance scans (within 30 days) to determine the repeatability of the technique. Subjects also perform a third scan two years later so that longitudinal change can be measured. Receives: Other: Absorptive clearance scan |
| Response | Experimental | Subjects perform three different absorptive clearance scans. One is a baseline measurement while the other two measure absorptive clearance after an intervention (inhaled hypertonic saline, mannitol inhalation powder). Receives: Other: Absorptive clearance scan; Drug: inhaled hypertonic saline (7%); Drug: mannitol inhalation powder |
What is being measured
The primary outcome is the main question the study is designed to answer.
absorptive clearance variability
The difference in absorptive clearance measured on two different study days in the same subjects as an indicator of measurement variability
Measured: 30 days
absorptive clearance response
The change in absorptive clearance demonstrated in response to therapy when compared to baseline
Measured: 14 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2013 (estimated)
- Study startJun 2013
- Primary completionDec 2017
- Study completionMay 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- 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
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tim Corcoran
- Sponsor type
- University, hospital or other
- 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
- NCT01887197
- Sponsor's protocol ID
- 1RO1 HL108929-01 (B)
- Other IDs
- 1R01HL108929-01
- Status last verified
- Jun 2018
- First submitted
- 24 Jun 2013
- Last updated
- 6 Nov 2018
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Corcoran TE, Thomas KM, Brown S, Myerburg MM, Locke LW, Pilewski JM. Liquid hyper-absorption as a cause of increased DTPA clearance in the cystic fibrosis airway. EJNMMI Res. 2013 Feb 27;3(1):14. doi: 10.1186/2191-219X-3-14.
PubMed 23446051Corcoran TE, Thomas KM, Myerburg MM, Muthukrishnan A, Weber L, Frizzell R, Pilewski JM. Absorptive clearance of DTPA as an aerosol-based biomarker in the cystic fibrosis airway. Eur Respir J. 2010 Apr;35(4):781-6. doi: 10.1183/09031936.00059009. Epub 2009 Aug 28.
PubMed 19717485Locke LW, Myerburg MM, Weiner DJ, Markovetz MR, Parker RS, Muthukrishnan A, Weber L, Czachowski MR, Lacy RT, Pilewski JM, Corcoran TE. Pseudomonas infection and mucociliary and absorptive clearance in the cystic fibrosis lung. Eur Respir J. 2016 May;47(5):1392-401. doi: 10.1183/13993003.01880-2015. Epub 2016 Mar 23.
PubMed 27009167
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Repeatability and Response Study of Absorptive Clearance Scans
- ClinicalTrials.gov ID
- NCT01887197
- Acronym / sponsor ID
- 1RO1 HL108929-01 (B)
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, mucociliary clearance, airway surface liquid, nuclear medicine, mannitol
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Tim CorcoranUniversity, hospital or other
- Study start
- Jun 2013 (actual)
- Primary completion
- Dec 2017 (actual)
- Study completion
- May 2018 (actual)
- First posted
- 26 Jun 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.
The investigators are developing a new nuclear medicine imaging technique for measuring liquid absorption in the airways that can be applied to screen new medications being developed to treat cystic fibrosis (CF). The investigators believe that the absorption of the small molecule radiopharmaceutical Indium 111 diethylene triamine pentaacetic acid (In-DTPA) will indicate changes in liquid absorption in the airways and demonstrate whether new CF medications will be effective. In this study the investigators will determine whether the imaging technique will demonstrate similar results when it is repeated on different days. They will also determine how their results change when subjects utilize several common CF medications.
Detailed description
Cystic Fibrosis (CF) is an autosomal recessive genetic disease, caused by mutations of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) that impairs ion transport at epithelial surfaces. This results in the accumulation of dehydrated secretions in the airways and chronic infection and inflammation in the lungs, leading to significant morbidity and mortality. The investigators understanding of CF pathogenesis has increased substantially and many new targeted therapies are being developed to treat this disease, however, the measurements of clinical efficacy used to evaluate these therapies require long trials to demonstrate an effect. New translational techniques are needed to assess changes in the most basic aspects of the disease and allow for the rapid screening of disease-altering therapies. The investigators have recently developed a novel aerosol-based imaging technique to measure liquid absorption in the airways - a central pathophysiological process related to CFTR dysfunction. The investigators propose that airway liquid hyper-absorption is a key link between cellular defects in ion and fluid transport and progressive airway dysfunction in CF. Thus The investigators technique may provide a measure of disease severity and rapid indication of therapeutic correction in advance of currently available outcome measures.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Repeatability | Experimental | Other: Absorptive clearance scanSubjects perform two sequential absorptive clearance scans (within 30 days) to determine the repeatability of the technique. Subjects also perform a third scan two years later so that longitudinal change can be measured. |
| Response | Experimental | Other: Absorptive clearance scan, Drug: inhaled hypertonic saline (7%), Drug: mannitol inhalation powderSubjects perform three different absorptive clearance scans. One is a baseline measurement while the other two measure absorptive clearance after an intervention (inhaled hypertonic saline, mannitol inhalation powder). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- subjects 18 years old 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
Key exclusion criteria (10 listed)
- one second forced expiratory volume (FEV1) <50% of predicted
- nursing mother
- positive urine pregnancy test or unwilling to test
- cigarette smoker
- unwilling to stop hypertonic saline therapy for 72 hours prior to each test day
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 |
|---|---|---|
| Absorptive clearance scanSubjects inhale a nebulized mixture of the radiopharmaceuticals Indium 111-DTPA and Technetium 99m sulfur colloid. | Other | Repeatability, Response |
| inhaled hypertonic saline (7%)Also: Hypersalnebulized hypertonic saline (7%) | Drug | Response |
| mannitol inhalation powderAlso: Bronchitolmannitol inhalation powder | Drug | Response |
Primary outcome
absorptive clearance variability
Time frame: 30 days
The difference in absorptive clearance measured on two different study days in the same subjects as an indicator of measurement variability
absorptive clearance response
Time frame: 14 day
The change in absorptive clearance demonstrated in response to therapy when compared to baseline
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 6 Nov 2018.
- Background: PubMed 23446051Corcoran TE, Thomas KM, Brown S, Myerburg MM, Locke LW, Pilewski JM. Liquid hyper-absorption as a cause of increased DTPA clearance in the cystic fibrosis airway. EJNMMI Res. 2013 Feb 27;3(1):14. doi: 10.1186/2191-219X-3-14.
- Background: PubMed 19717485Corcoran TE, Thomas KM, Myerburg MM, Muthukrishnan A, Weber L, Frizzell R, Pilewski JM. Absorptive clearance of DTPA as an aerosol-based biomarker in the cystic fibrosis airway. Eur Respir J. 2010 Apr;35(4):781-6. doi: 10.1183/09031936.00059009. Epub 2009 Aug 28.
- Derived from this study: PubMed 27009167Locke LW, Myerburg MM, Weiner DJ, Markovetz MR, Parker RS, Muthukrishnan A, Weber L, Czachowski MR, Lacy RT, Pilewski JM, Corcoran TE. Pseudomonas infection and mucociliary and absorptive clearance in the cystic fibrosis lung. Eur Respir J. 2016 May;47(5):1392-401. doi: 10.1183/13993003.01880-2015. Epub 2016 Mar 23.
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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 Absorptive clearance scan, inhaled hypertonic saline (7%) and mannitol inhalation powder 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?
The sponsor lists 1 inclusion and 10 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 6 Nov 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.
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?
absorptive clearance variability (time frame: 30 days). 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?
On the ClinicalTrials.gov record for NCT01887197, which also lists 3 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 6 Nov 2018.