Cystic FibrosisNCT01031706
Effect of Hypertonic Saline on Mucus Clearance in Children Ages 5-12 With Cystic Fibrosis
A study testing Hypertonic Saline in people with Cystic Fibrosis, run by University of North Carolina, Chapel Hill at 1 site in United States.
Trial summary
Previous work demonstrated that inhaled hypertonic saline (HS) reduces exacerbation frequency and improves lung function in adults with cystic fibrosis (CF). It is unclear, however, whether HS will benefit young patients suffering from CF. Read more about this study
- Who is it for?
- Ages 5 to 12 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Hypertonic Saline
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2009to Jul 2012
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 5 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Gender: Females or Males. If the subject is female and of childbearing potential (first menses has occurred), she must have a documented negative pregnancy test at screening and prior to each mucociliary clearance study. Those of childbearing potential must be abstinent or using an acceptable method of birth control (i.e. an Intrauterine Contraceptive Device with a failure rate of <1%, hormonal contraceptives or a barrier method).
- Age: 5-12 years, inclusive
- Diagnosis: Cystic fibrosis documented by a compatible clinical presentation and sweat chloride > 60 mEq/l or 2 disease causing CFTR mutations.
- Severity of the Disease: Suitable patients will have mild lung disease, as defined by:
- Pulmonary Function: Each patient must have an FEV1 of greater than or equal to 60% of predicted at the screening visit.
- Hemoglobin saturation: Patients must have an oxygen saturation of >92% on room air as determined by pulse oximetry at the screening visit.
- Informed consent - The patient and a parent or legally authorized guardian must agree to the subject's participation in the study by signing and dating the informed consent/assent forms after the nature of the study has been fully explained and all questions have been satisfactorily answered.
Who cannot join
- Unstable or asthmatic lung disease: As defined by a change in medical regimen during the preceding 2 weeks; an FEV1 15% below recent (within 6 months) clinical measurements. Patients with a history of co-existent asthma, as manifested by wheezing and significant bronchoreactivity (>15% increase in FEV1 with bronchodilator), will also be excluded.
- Other medication usage: Patients unable or unwilling to be withdrawn from hypertonic saline therapy for two weeks prior to Visit 1 (baseline MCC visit). Patients using Pulmozyme will be permitted to participate in this trial. Patients on chronic, cycling antibiotics will be required to have completed at least 2 full cycles of the prescribed antibiotic prior to enrollment and should not cycle on or off this therapy during the treatment period of the study.
- Spirometry Performance: Those subjects who are unable to perform acceptable, reproducible spirometry will be excluded from this study.
- Drug allergy: A history of allergy or intolerance to any of the study medications, including albuterol or hypertonic saline.
- Have received an investigational drug or therapy during the preceding 30 days.
- Have had radiation exposure within the past year that would cause them to exceed Federal Regulations by participating in this study.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of North Carolina
Chapel Hill, North Carolina, 27599
About this study
Official title: Sustained Impact of Hypertonic Saline on Mucociliary Clearance in Young Children With Cystic Fibrosis
Previous work demonstrated that inhaled hypertonic saline (HS) reduces exacerbation frequency and improves lung function in adults with cystic fibrosis (CF). It is unclear, however, whether HS will benefit young patients suffering from CF. The investigators propose to further support the concept that HS can benefit children with mild CF lung disease by performing a relatively short, placebo controlled study of HS in 5-12 year olds, using lung function and mucociliary clearance as key outcome measures.
Read the detailed description
Our current understanding of the pathogenesis of CF lung disease stems from data that demonstrate the presence of airway surface liquid (ASL) dehydration in CF. ASL dehydration in CF is caused by defective chloride secretion through the cystic fibrosis transmembrane regulator (CFTR) and increased sodium reabsorption through the epithelial sodium channel (ENaC). ASL dehydration, in turn, interferes with the mucociliary clearance apparatus, causing a breach in a critical line of lung host defense. A number of novel therapeutics are now being developed to address this basic defect of disease, including the use of inhaled hypertonic saline.
Previous work demonstrated that inhaled hypertonic saline (HS) reduces exacerbation frequency and improves lung function in patients with clinically apparent lung disease. A number of issues revolving around the use of HS in CF remain unresolved. First, the typical patients enrolled in previous studies were adults (mean age = 26 yrs) with established lung disease (mean FEV1=78%). Despite our hypothesis that HS should positively affect MCC in preserved/normal airways, a common view of HS is that it benefits CF patients by inducing cough and transiently promoting the clearance of thick CF secretions. It has been questioned, therefore, whether HS will benefit patients who are younger and have mild (or undetectable) lung disease and potentially normal (though unmeasured) rates of MCC. Second, it is unclear whether the substantial beneficial effects of HS in CF were achieved because of a long (>4 hours) duration of action or in spite of an extremely short (~45 minutes) duration of action (the traditional view based upon experiments in normal epithelia). This issue is important, as it relates to the development and dosing of hydrator therapies that may have different pharmacodynamic profiles. Certainly, if twice daily dosing of a short acting compound is sufficient to provide significant clinical benefit, it would reduce the challenge of drug discovery for CF and ease the treatment burden imposed upon patients. The study of HS in CF provides us an opportunity to address this issue.
The hypothesis being tested is that HS will rehydrate CF airway secretions, producing a sustained acceleration in MCC in young children with CF, regardless of whether a measurable mucus clearance defect exists at this relatively early stage of disease. We predict a substantial acceleration of MCC will reduce the exacerbation rate in young children with CF. In addition, with the growing number of treatment modalities that are prescribed to patients with CF, adherence to complex and time consuming medical regimens becomes increasingly problematic and important. We therefore, wish to test an improved drug delivery platform for HS- the eFlow (Pari Pharma) vibrating mesh nebulizer, which has the potential to reduce treatment times, improve compliance, and increase treatment efficacy.
- Conditions
- Cystic Fibrosis
- Being tested
- Hypertonic Saline, Placebo
- Keywords
- Hypertonic Saline, Pediatrics, Cystic Fibrosis, Mucociliary Clearance, Lung Clearance Index
- 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
Also called HS
inhaled HS (6% NaCl, 4mL) three times a day for 28 days
PlaceboDrug
Also called .12% NaCl
4 ml 0.12% NaCl inhaled three times a day x 28 days
| Study group | Role | What they receive |
|---|---|---|
| Hypertonic saline | Experimental | 6% NaCl, 4 ml TID via eFlow Receives: Drug: Hypertonic Saline |
| Placebo | Placebo comparator | 0.12% x 4ml via eFlow nebulizer Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Mucociliary Clearance Rate
Average radio tracer clearance through 90 minutes (MCC90) is primary index of mucociliary clearance at each study.
Primary study outcome: is absolute change in MCC90 between baseline and at end of treatment (where MCC measured 8-12 hours after final dose of study drug) - reflects sustained impact on MCC
Measured: Baseline versus after completion of 4 week treatment period
Secondary outcomes (1)
FEV1 (Spirometry) Change
Absolute change in % predicted FEV1 between baseline and after 4 weeks of treatment calculated
Measured: Baseline and after 4 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2009 (estimated)
- Study startSep 2009
- Primary completionJul 2012
- Study completionJul 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (8)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 University of North Carolina, Chapel HillThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of North Carolina, Chapel Hill
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Principal investigator, Scott Donaldson, MD, University of North Carolina, Chapel Hill
- Investigators
- Scott H Donaldson, MD, Principal investigator, University of North Carolina, Chapel Hill
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01031706
- Sponsor's protocol ID
- 09-1258
- Other IDs
- 1P50HL084934
- Status last verified
- Mar 2014
- First submitted
- 11 Dec 2009
- Last updated
- 3 Mar 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Elkins MR, Robinson M, Rose BR, Harbour C, Moriarty CP, Marks GB, Belousova EG, Xuan W, Bye PT; National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N Engl J Med. 2006 Jan 19;354(3):229-40. doi: 10.1056/NEJMoa043900.
PubMed 16421364Donaldson SH, Bennett WD, Zeman KL, Knowles MR, Tarran R, Boucher RC. Mucus clearance and lung function in cystic fibrosis with hypertonic saline. N Engl J Med. 2006 Jan 19;354(3):241-50. doi: 10.1056/NEJMoa043891.
PubMed 16421365Bennett WD, Olivier KN, Zeman KL, Hohneker KW, Boucher RC, Knowles MR. Effect of uridine 5'-triphosphate plus amiloride on mucociliary clearance in adult cystic fibrosis. Am J Respir Crit Care Med. 1996 Jun;153(6 Pt 1):1796-801. doi: 10.1164/ajrccm.153.6.8665037.
PubMed 8665037Matsui H, Grubb BR, Tarran R, Randell SH, Gatzy JT, Davis CW, Boucher RC. Evidence for periciliary liquid layer depletion, not abnormal ion composition, in the pathogenesis of cystic fibrosis airways disease. Cell. 1998 Dec 23;95(7):1005-15. doi: 10.1016/s0092-8674(00)81724-9.
PubMed 9875854Tarran R. Regulation of airway surface liquid volume and mucus transport by active ion transport. Proc Am Thorac Soc. 2004;1(1):42-6. doi: 10.1513/pats.2306014.
PubMed 16113411Elkins MR, Bye PT. Inhaled hypertonic saline as a therapy for cystic fibrosis. Curr Opin Pulm Med. 2006 Nov;12(6):445-52. doi: 10.1097/01.mcp.0000245714.89632.b2.
PubMed 17053496Modi AC, Quittner AL. Validation of a disease-specific measure of health-related quality of life for children with cystic fibrosis. J Pediatr Psychol. 2003 Dec;28(8):535-45. doi: 10.1093/jpepsy/jsg044.
PubMed 14602844Donaldson SH, Danielle Samulski T, LaFave C, Zeman K, Wu J, Trimble A, Ceppe A, Bennett WD, Davis SD. A four week trial of hypertonic saline in children with mild cystic fibrosis lung disease: Effect on mucociliary clearance and clinical outcomes. J Cyst Fibros. 2020 Nov;19(6):942-948. doi: 10.1016/j.jcf.2020.07.009. Epub 2020 Jul 12.
PubMed 32669217
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sustained Impact of Hypertonic Saline on Mucociliary Clearance in Young Children With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT01031706
- Acronym / sponsor ID
- 09-1258
- Conditions
- Cystic Fibrosis
- Keywords
- Hypertonic Saline, Pediatrics, Cystic Fibrosis, Mucociliary Clearance, Lung Clearance Index
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- University of North Carolina, Chapel HillUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Sep 2009
- Primary completion
- Jul 2012 (actual)
- Study completion
- Jul 2012 (actual)
- First posted
- 15 Dec 2009 (estimated)
- Results
- Posted 30 Jun 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Previous work demonstrated that inhaled hypertonic saline (HS) reduces exacerbation frequency and improves lung function in adults with cystic fibrosis (CF). It is unclear, however, whether HS will benefit young patients suffering from CF. The investigators propose to further support the concept that HS can benefit children with mild CF lung disease by performing a relatively short, placebo controlled study of HS in 5-12 year olds, using lung function and mucociliary clearance as key outcome measures.
Detailed description
Our current understanding of the pathogenesis of CF lung disease stems from data that demonstrate the presence of airway surface liquid (ASL) dehydration in CF. ASL dehydration in CF is caused by defective chloride secretion through the cystic fibrosis transmembrane regulator (CFTR) and increased sodium reabsorption through the epithelial sodium channel (ENaC). ASL dehydration, in turn, interferes with the mucociliary clearance apparatus, causing a breach in a critical line of lung host defense. A number of novel therapeutics are now being developed to address this basic defect of disease, including the use of inhaled hypertonic saline.
Previous work demonstrated that inhaled hypertonic saline (HS) reduces exacerbation frequency and improves lung function in patients with clinically apparent lung disease. A number of issues revolving around the use of HS in CF remain unresolved. First, the typical patients enrolled in previous studies were adults (mean age = 26 yrs) with established lung disease (mean FEV1=78%). Despite our hypothesis that HS should positively affect MCC in preserved/normal airways, a common view of HS is that it benefits CF patients by inducing cough and transiently promoting the clearance of thick CF secretions. It has been questioned, therefore, whether HS will benefit patients who are younger and have mild (or undetectable) lung disease and potentially normal (though unmeasured) rates of MCC. Second, it is unclear whether the substantial beneficial effects of HS in CF were achieved because of a long (>4 hours) duration of action or in spite of an extremely short (~45 minutes) duration of action (the traditional view based upon experiments in normal epithelia). This issue is important, as it relates to the development and dosing of hydrator therapies that may have different pharmacodynamic profiles. Certainly, if twice daily dosing of a short acting compound is sufficient to provide significant clinical benefit, it would reduce the challenge of drug discovery for CF and ease the treatment burden imposed upon patients. The study of HS in CF provides us an opportunity to address this issue.
The hypothesis being tested is that HS will rehydrate CF airway secretions, producing a sustained acceleration in MCC in young children with CF, regardless of whether a measurable mucus clearance defect exists at this relatively early stage of disease. We predict a substantial acceleration of MCC will reduce the exacerbation rate in young children with CF. In addition, with the growing number of treatment modalities that are prescribed to patients with CF, adherence to complex and time consuming medical regimens becomes increasingly problematic and important. We therefore, wish to test an improved drug delivery platform for HS- the eFlow (Pari Pharma) vibrating mesh nebulizer, which has the potential to reduce treatment times, improve compliance, and increase treatment efficacy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hypertonic saline | Experimental | Drug: Hypertonic Saline6% NaCl, 4 ml TID via eFlow |
| Placebo | Placebo comparator | Drug: Placebo0.12% x 4ml via eFlow nebulizer |
Who may qualify
- Ages
- 5 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Gender: Females or Males. If the subject is female and of childbearing potential (first menses has occurred), she must have a documented negative pregnancy test at screening and prior to each mucociliary clearance study. Those of childbearing potential must be abstinent or using an acceptable method of birth control (i.e. an Intrauterine Contraceptive Device with a failure rate of <1%, hormonal contraceptives or a barrier method).
- Age: 5-12 years, inclusive
- Diagnosis: Cystic fibrosis documented by a compatible clinical presentation and sweat chloride > 60 mEq/l or 2 disease causing CFTR mutations.
- Severity of the Disease: Suitable patients will have mild lung disease, as defined by:
- Informed consent - The patient and a parent or legally authorized guardian must agree to the subject's participation in the study by signing and dating the informed consent/assent forms after the nature of the study has been fully explained and all questions have been satisfactorily answered.
Key exclusion criteria (6 listed)
- Unstable or asthmatic lung disease: As defined by a change in medical regimen during the preceding 2 weeks; an FEV1 15% below recent (within 6 months) clinical measurements. Patients with a history of co-existent asthma, as manifested by wheezing and significant bronchoreactivity (>15% increase in FEV1 with bronchodilator), will also be excluded.
- Other medication usage: Patients unable or unwilling to be withdrawn from hypertonic saline therapy for two weeks prior to Visit 1 (baseline MCC visit). Patients using Pulmozyme will be permitted to participate in this trial. Patients on chronic, cycling antibiotics will be required to have completed at least 2 full cycles of the prescribed antibiotic prior to enrollment and should not cycle on or off this therapy during the treatment period of the study.
- Spirometry Performance: Those subjects who are unable to perform acceptable, reproducible spirometry will be excluded from this study.
- Drug allergy: A history of allergy or intolerance to any of the study medications, including albuterol or hypertonic saline.
- Have received an investigational drug or therapy during the preceding 30 days.
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 SalineAlso: HSinhaled HS (6% NaCl, 4mL) three times a day for 28 days | Drug | Hypertonic saline |
| PlaceboAlso: .12% NaCl4 ml 0.12% NaCl inhaled three times a day x 28 days | Drug | Placebo |
Primary outcome
Change in Mucociliary Clearance Rate
Time frame: Baseline versus after completion of 4 week treatment period
Average radio tracer clearance through 90 minutes (MCC90) is primary index of mucociliary clearance at each study. Primary study outcome: is absolute change in MCC90 between baseline and at end of treatment (where MCC measured 8-12 hours after final dose of study drug) - reflects sustained impact on MCC
1 secondary outcome
FEV1 (Spirometry) Change
Time frame: Baseline and after 4 weeks of treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 16421364Elkins MR, Robinson M, Rose BR, Harbour C, Moriarty CP, Marks GB, Belousova EG, Xuan W, Bye PT; National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N Engl J Med. 2006 Jan 19;354(3):229-40. doi: 10.1056/NEJMoa043900.
- Background: PubMed 16421365Donaldson SH, Bennett WD, Zeman KL, Knowles MR, Tarran R, Boucher RC. Mucus clearance and lung function in cystic fibrosis with hypertonic saline. N Engl J Med. 2006 Jan 19;354(3):241-50. doi: 10.1056/NEJMoa043891.
- Background: PubMed 8665037Bennett WD, Olivier KN, Zeman KL, Hohneker KW, Boucher RC, Knowles MR. Effect of uridine 5'-triphosphate plus amiloride on mucociliary clearance in adult cystic fibrosis. Am J Respir Crit Care Med. 1996 Jun;153(6 Pt 1):1796-801. doi: 10.1164/ajrccm.153.6.8665037.
- Background: PubMed 9875854Matsui H, Grubb BR, Tarran R, Randell SH, Gatzy JT, Davis CW, Boucher RC. Evidence for periciliary liquid layer depletion, not abnormal ion composition, in the pathogenesis of cystic fibrosis airways disease. Cell. 1998 Dec 23;95(7):1005-15. doi: 10.1016/s0092-8674(00)81724-9.
- Background: PubMed 16113411Tarran R. Regulation of airway surface liquid volume and mucus transport by active ion transport. Proc Am Thorac Soc. 2004;1(1):42-6. doi: 10.1513/pats.2306014.
- Background: PubMed 17053496Elkins MR, Bye PT. Inhaled hypertonic saline as a therapy for cystic fibrosis. Curr Opin Pulm Med. 2006 Nov;12(6):445-52. doi: 10.1097/01.mcp.0000245714.89632.b2.
- Background: PubMed 14602844Modi AC, Quittner AL. Validation of a disease-specific measure of health-related quality of life for children with cystic fibrosis. J Pediatr Psychol. 2003 Dec;28(8):535-45. doi: 10.1093/jpepsy/jsg044.
- Derived from this study: PubMed 32669217Donaldson SH, Danielle Samulski T, LaFave C, Zeman K, Wu J, Trimble A, Ceppe A, Bennett WD, Davis SD. A four week trial of hypertonic saline in children with mild cystic fibrosis lung disease: Effect on mucociliary clearance and clinical outcomes. J Cyst Fibros. 2020 Nov;19(6):942-948. doi: 10.1016/j.jcf.2020.07.009. Epub 2020 Jul 12.
- Individual participant dataSharing planned: No
Related trials for Cystic Fibrosis
RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete
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.
Scott H Donaldson, MD principal investigator
University of North Carolina, Chapel Hill
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Hypertonic Saline in people with Cystic Fibrosis, run by University of North Carolina, Chapel Hill 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 7 inclusion and 6 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 3 Mar 2017. 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 30 Jun 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Mucociliary Clearance Rate (time frame: Baseline versus after completion of 4 week treatment period). 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 NCT01031706, which also lists 8 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 3 Mar 2017.