Cystic FibrosisPhase 2NCT01293084
Hypertonic Saline and Mucociliary Clearance in Children
A Phase 2 study testing 0.12% saline and 7% saline in people with Cystic Fibrosis, run by Johns Hopkins University.
Trial summary
Previously, the investigators and others have shown that mucociliary clearance (MCC) is defective in patients with cystic fibrosis (CF) and it is now thought that alterations in airway mucus rheology figure prominently in the impairment. Read more about this study
- Who is it for?
- Ages 7 to 12 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- 0.12% saline and 7% saline
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 17 participants
- Where
- No sites listed yet
- Study dates
- Jul 2007to Jun 2009
- Phase
- Phase 2
- 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 7 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Males and females
- Age 7-12 years old
- Diagnosis of cystic fibrosis by sweat chloride > 60 meq/L, or presence of two CFTR mutations known to cause CF
- Routinely treated with the short-acting bronchodilator albuterol
- FEV1 > 90% of predicted values
Who cannot join
- FEV1 < 90% of predicted values
- Routine use of hypertonic saline, mannitol, or amiloride
- Allergic bronchopulmonary aspergillosis (ABPA)
- Sputum colonization with Burkholderia cepacia or multiple antibiotic resistant organisms
- Evidence of a pulmonary exacerbation within past two weeks
- Treated with intravenous or oral antibiotics in the past two weeks for a pulmonary exacerbation
Show all 12Show fewer
- Presence of an acute respiratory illness characterized by:
- Coughing above baseline values
- Wheezing
- Respiratory distress
- Hemoptysis
- Cannot perform the inhalation maneuvers that are required for drug inhalation or radioaerosol administration
About this study
Official title: Acute Inhalation of Hypertonic Saline Does Not Improve Mucociliary Clearance in All Children With Cystic Fibrosis
Previously, the investigators and others have shown that mucociliary clearance (MCC) is defective in patients with cystic fibrosis (CF) and it is now thought that alterations in airway mucus rheology figure prominently in the impairment. Mucociliary clearance works by trapping toxic particles, bacteria and viruses in the lung mucus and then quickly removing the mucus out of the lungs. Defects in MCC typically lead to the accumulation of mucus in the airways, and this in turn is associated with acute infections, chronic bacterial colonization and chronic inflammation. One treatment strategy that is gaining acceptance as an important therapy for improving MCC in adults with CF is the inhalation of the osmotic stimulus, hypertonic saline (HS). A number of studies have shown that acute inhalation of HS (7% saline) significantly improves MCC in adults with CF and results from a recent study indicate that two weeks of inhaling HS leads to a significant increase in MCC that is sustained for 8 hours post inhalation and is associated with significant improvements in FEV1, FVC and FEF25-75 values. Since MCC in patients with CF appears to be impaired by adulthood, any drug that disrupts or slows the impairment in childhood could prove enormously beneficial in the long-term prognosis of the disease. Nevertheless, no studies have been conducted to determine if HS treatment improves MCC in children with CF. This is most problematic for physicians who care for children with CF who have normal FEV1 and FVC values, since it is unclear if they should treat these children with HS or not. This research study is designed to begin to answer this question. The investigators hypothesize that acute inhalation of hypertonic saline (7%) will improve MCC in CF children with normal pulmonary function. Our hypothesis will be tested in a one-year clinical trial that will be randomized and placebo-controlled. Twelve children with CF who are 7-12 years old and have normal FEV1 and FVC values will participate. Our goal will be to compare MCC in these children on two study visits after acute inhalations of placebo (0.12% saline) or hypertonic saline (HS) (7% saline) aerosol. The investigators predict that MCC values after acute inhalation of 7% HS aerosol will be statistically significantly greater than after placebo inhalation.
Read the detailed description
Several studies report that mucociliary clearance (MCC) is impaired in adults with CF. Because MCC is an important airway defense mechanism, drugs that slow impairment of MCC in children could prove beneficial in the long-term prognosis of the disease. A few studies have shown that inhalation of hypertonic saline (HS) significantly improves MCC in adults with CF and improvement is associated with increases in pulmonary function and decreases in pulmonary exacerbations. Nevertheless, no studies have examined if HS improves MCC in CF children. This is problematic for physicians who care for CF children with normal pulmonary function, since it is unclear if they should treat with HS or not. This study was designed to begin to answer this question. Twelve children with CF (7-12 yrs; 5 males) and normal pulmonary function (FEV1 and FVC > 90% of predicted values) participated in a screening visit and two study visits. On the screening visit, children underwent an induced sputum test. On the two study visits, they inhaled 0.12% saline (placebo), or HS, in a double-blind, randomized, cross-over study. Following inhalation of placebo or HS, patients inhaled the radioisotope 99mtechnetium and underwent sequential imaging of their lungs with a gamma camera for 90 min and approximately 24 hrs later. Mucociliary clearance was quantified at 60 min (MCC60), 90 min (MCC90) and 24 hrs (MCC24hrs) after inhalation of the radioisotope. Between the 60 min and 90 min measurements, children coughed 30 times.
- Conditions
- Cystic Fibrosis
- Being tested
- 0.12% saline, 7% saline
- Keywords
- mucociliary clearance, children, cystic fibrosis, hypertonic saline
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
0.12% salineDrug
5mL of 0.12% saline inhaled once over 20 minutes
7% salineDrug
Also called hypertonic saline
5mL 7% saline inhaled once over 20 minutes
| Study group | Role | What they receive |
|---|---|---|
| 7% saline | Experimental | 5 mL of 7% saline was inhaled once over a 20 minute period. Receives: Drug: 7% saline |
| 0.12% saline | Placebo comparator | 5mL 0.12% saline inhaled once during 20 minutes Receives: Drug: 0.12% saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Mucociliary Clearance at 60 Minutes
Measured: 60 minutes
Secondary outcomes (1)
Percent Mucociliary Clearance at 90 Minutes
Measured: 90 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2011 (estimated)
- Study startJul 2007
- Primary completionJun 2009
- Study completionJun 2009
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 (1)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 Johns Hopkins 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johns Hopkins University
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Principal investigator, Beth L. Laube, Johns Hopkins University
- Investigators
- Beth L Laube, PhD, Principal investigator, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01293084
- Sponsor's protocol ID
- CFF Account #LAUBE06A0
- Status last verified
- Sep 2015
- First submitted
- 9 Feb 2011
- Last updated
- 22 Sep 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Laube BL, Sharpless G, Carson KA, Kelly A, Mogayzel PJ Jr. Acute inhalation of hypertonic saline does not improve mucociliary clearance in all children with cystic fibrosis. BMC Pulm Med. 2011 Sep 6;11:45. doi: 10.1186/1471-2466-11-45.
PubMed 21896198
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Acute Inhalation of Hypertonic Saline Does Not Improve Mucociliary Clearance in All Children With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT01293084
- Acronym / sponsor ID
- CFF Account #LAUBE06A0
- Conditions
- Cystic Fibrosis
- Keywords
- mucociliary clearance, children, cystic fibrosis, hypertonic saline
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Johns Hopkins UniversityUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- Jul 2007
- Primary completion
- Jun 2009 (actual)
- Study completion
- Jun 2009 (actual)
- First posted
- 10 Feb 2011 (estimated)
- Results
- Posted 22 Sep 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Previously, the investigators and others have shown that mucociliary clearance (MCC) is defective in patients with cystic fibrosis (CF) and it is now thought that alterations in airway mucus rheology figure prominently in the impairment. Mucociliary clearance works by trapping toxic particles, bacteria and viruses in the lung mucus and then quickly removing the mucus out of the lungs. Defects in MCC typically lead to the accumulation of mucus in the airways, and this in turn is associated with acute infections, chronic bacterial colonization and chronic inflammation. One treatment strategy that is gaining acceptance as an important therapy for improving MCC in adults with CF is the inhalation of the osmotic stimulus, hypertonic saline (HS). A number of studies have shown that acute inhalation of HS (7% saline) significantly improves MCC in adults with CF and results from a recent study indicate that two weeks of inhaling HS leads to a significant increase in MCC that is sustained for 8 hours post inhalation and is associated with significant improvements in FEV1, FVC and FEF25-75 values. Since MCC in patients with CF appears to be impaired by adulthood, any drug that disrupts or slows the impairment in childhood could prove enormously beneficial in the long-term prognosis of the disease. Nevertheless, no studies have been conducted to determine if HS treatment improves MCC in children with CF. This is most problematic for physicians who care for children with CF who have normal FEV1 and FVC values, since it is unclear if they should treat these children with HS or not. This research study is designed to begin to answer this question. The investigators hypothesize that acute inhalation of hypertonic saline (7%) will improve MCC in CF children with normal pulmonary function. Our hypothesis will be tested in a one-year clinical trial that will be randomized and placebo-controlled. Twelve children with CF who are 7-12 years old and have normal FEV1 and FVC values will participate. Our goal will be to compare MCC in these children on two study visits after acute inhalations of placebo (0.12% saline) or hypertonic saline (HS) (7% saline) aerosol. The investigators predict that MCC values after acute inhalation of 7% HS aerosol will be statistically significantly greater than after placebo inhalation.
Detailed description
Several studies report that mucociliary clearance (MCC) is impaired in adults with CF. Because MCC is an important airway defense mechanism, drugs that slow impairment of MCC in children could prove beneficial in the long-term prognosis of the disease. A few studies have shown that inhalation of hypertonic saline (HS) significantly improves MCC in adults with CF and improvement is associated with increases in pulmonary function and decreases in pulmonary exacerbations. Nevertheless, no studies have examined if HS improves MCC in CF children. This is problematic for physicians who care for CF children with normal pulmonary function, since it is unclear if they should treat with HS or not. This study was designed to begin to answer this question. Twelve children with CF (7-12 yrs; 5 males) and normal pulmonary function (FEV1 and FVC > 90% of predicted values) participated in a screening visit and two study visits. On the screening visit, children underwent an induced sputum test. On the two study visits, they inhaled 0.12% saline (placebo), or HS, in a double-blind, randomized, cross-over study. Following inhalation of placebo or HS, patients inhaled the radioisotope 99mtechnetium and underwent sequential imaging of their lungs with a gamma camera for 90 min and approximately 24 hrs later. Mucociliary clearance was quantified at 60 min (MCC60), 90 min (MCC90) and 24 hrs (MCC24hrs) after inhalation of the radioisotope. Between the 60 min and 90 min measurements, children coughed 30 times.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 7% saline | Experimental | Drug: 7% saline5 mL of 7% saline was inhaled once over a 20 minute period. |
| 0.12% saline | Placebo comparator | Drug: 0.12% saline5mL 0.12% saline inhaled once during 20 minutes |
Who may qualify
- Ages
- 7 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Males and females
- Age 7-12 years old
- Diagnosis of cystic fibrosis by sweat chloride > 60 meq/L, or presence of two CFTR mutations known to cause CF
- Routinely treated with the short-acting bronchodilator albuterol
- FEV1 > 90% of predicted values
Key exclusion criteria (12 listed)
- FEV1 < 90% of predicted values
- Routine use of hypertonic saline, mannitol, or amiloride
- Allergic bronchopulmonary aspergillosis (ABPA)
- Sputum colonization with Burkholderia cepacia or multiple antibiotic resistant organisms
- Evidence of a pulmonary exacerbation within past two 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 |
|---|---|---|
| 0.12% saline5mL of 0.12% saline inhaled once over 20 minutes | Drug | 0.12% saline |
| 7% salineAlso: hypertonic saline5mL 7% saline inhaled once over 20 minutes | Drug | 7% saline |
Primary outcome
Percent Mucociliary Clearance at 60 Minutes
Time frame: 60 minutes
1 secondary outcome
Percent Mucociliary Clearance at 90 Minutes
Time frame: 90 minutes
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 21896198Laube BL, Sharpless G, Carson KA, Kelly A, Mogayzel PJ Jr. Acute inhalation of hypertonic saline does not improve mucociliary clearance in all children with cystic fibrosis. BMC Pulm Med. 2011 Sep 6;11:45. doi: 10.1186/1471-2466-11-45.
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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.
Beth L Laube, PhD principal investigator
Johns Hopkins University
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 0.12% saline and 7% saline in people with Cystic Fibrosis, run by Johns Hopkins University.
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 5 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 22 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Sep 2015). 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?
Percent Mucociliary Clearance at 60 Minutes (time frame: 60 minutes). 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 NCT01293084, which also lists 1 linked publication.
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 Sep 2015.