Cystic FibrosisPhase 3NCT00635141
The Effect of Hypertonic Saline on the Lung Clearance Index in Patients With Cystic Fibrosis
A Phase 3 study testing hypertonic saline (7 %) and isotonic saline (0.9%) in people with Cystic Fibrosis, run by The Hospital for Sick Children at 1 site in Canada.
Trial summary
This study is examining the effect of hypertonic saline compared to placebo on the Lung Clearance Index in Cystic Fibrosis patients. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- hypertonic saline (7 %) and isotonic saline (0.9%)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteCanada
- Study dates
- Mar 2008to Dec 2008
- Phase
- Phase 3
- 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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of CF as defined by two or more clinical features of CF and a documented sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis test or a genotype showing two well characterized disease causing mutations
- Informed consent and verbal assent (as appropriate) provided by the subject's parent or legal guardian and the subject
- 6-18 years of age at enrolment and able to perform reproducible spirometry
- Clinically stable at enrolment
- FEV1 % predicted ≥ 80 % as calculated by the Wang reference equations
- Ability to comply with medication use, study visits and study procedures
Who cannot join
- Respiratory culture positive for NTM or B. cepacia complex within past year or screening
- Use of intravenous antibiotics or oral quinolones within 14 days of screening
- Investigational drug use within 30 days of screening
- Physical findings at screening that would compromise the safety of the participant or the quality of the study data
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Effect of Inhaled Hypertonic Saline (7%) Versus Normal Saline (0.9%) on the Lung Clearance Index in Patients With Cystic Fibrosis
This study is examining the effect of hypertonic saline compared to placebo on the Lung Clearance Index in Cystic Fibrosis patients.
Read the detailed description
The life expectancy of patients with Cystic Fibrosis (CF) has greatly increased over time due to improved clinical care. While this is certainly beneficial to CF patients, the overall stability in lung function has made it more difficult to assess the effect of therapeutic interventions. Currently, FEV1 (forced expiratory volume in 1 second) remains the primary outcome measure for most clinical trials, but many CF patients have normal pulmonary function and the annual rate of decline is now less than 2% in large specialized centres. Therefore, additional parameters are needed that are more sensitive parameters to define abnormalities in CF patients and be used in therapeutic trials.
One such promising parameter is the Lung Clearance Index (LCI). The LCI is a test to quantify ventilation inhomogeneity by assessing the washout of an inhaled inert gas. Abnormalities in gas clearance from the lung are largely due to retention of inhaled gases due to mucous obstruction in the lower airways.
Interventions that improve mucus accumulation are expected to improve the LCI. Hypertonic saline has been shown to increase airway surface liquid (ASL), mucociliary clearance and pulmonary function. Therefore, this study will examine the effect of hypertonic saline compared to placebo on the Lung Clearance Index in Cystic Fibrosis patients.
- Conditions
- Cystic Fibrosis
- Being tested
- hypertonic saline (7 %) and isotonic saline (0.9%)
- Keywords
- Cystic Fibrosis, Lung Clearance Index, Hypertonic Saline, Pediatrics
- 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 saline (7 %) and isotonic saline (0.9%)Drug
Patients in study arm 1 will receive hypertonic saline (7 %) during the first phase of the study and will crossover to placebo (0.9% isotonic saline) during the second phase. Both drugs will be administered via PARI LC® Star reusable nebulizer.
hypertonic saline (7 %) and isotonic saline (0.9%)Drug
Patients in study arm 2 will receive placebo (0.9% isotonic saline) during the first phase of the study and will crossover to hypertonic saline (7 %) during the second phase. Both drugs will be administered via PARI LC® Star reusable nebulizer.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Not described Receives: Drug: hypertonic saline (7 %) and isotonic saline (0.9%) |
| 2 | Experimental | Not described Receives: Drug: hypertonic saline (7 %) and isotonic saline (0.9%) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in LCI from baseline to end of treatment in hypertonic saline treated patients versus patients receiving placebo (isotonic saline)
Measured: Duration of patient's involvement in study
Secondary outcomes (3)
Change in FEV1 % predicted
Measured: Duration of patient's involvement in study
Change in FVC (forced vital capacity) % predicted
Measured: Duration of patient's involvement in study
Change in FEF25-75% (forced expiratory flow between 25 and 75 % of vital capacity) predicted
Measured: Duration of patient's involvement in study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2008 (estimated)
- Study startMar 2008
- Primary completionDec 2008
- Study completionDec 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)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 The Hospital for Sick ChildrenThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Hospital for Sick Children
- Sponsor type
- University, hospital or other
- Collaborators
- Canadian Cystic Fibrosis Foundation
- Responsible party
- Principal investigator, Felix Ratjen, The Hospital for Sick Children
- Investigators
- Felix Ratjen, MD, Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00635141
- Sponsor's protocol ID
- 1000011193
- Status last verified
- Aug 2013
- First submitted
- 4 Mar 2008
- Last updated
- 2 Sep 2013
- Results posted
- No
- Data monitoring committee
- No
Publications
Amin R, Subbarao P, Jabar A, Balkovec S, Jensen R, Kerrigan S, Gustafsson P, Ratjen F. Hypertonic saline improves the LCI in paediatric patients with CF with normal lung function. Thorax. 2010 May;65(5):379-83. doi: 10.1136/thx.2009.125831.
PubMed 20435858Wark P, McDonald VM, Smith S. Nebulised hypertonic saline for cystic fibrosis. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD001506. doi: 10.1002/14651858.CD001506.pub5.
PubMed 37319354
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Inhaled Hypertonic Saline (7%) Versus Normal Saline (0.9%) on the Lung Clearance Index in Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00635141
- Acronym / sponsor ID
- 1000011193
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Lung Clearance Index, Hypertonic Saline, Pediatrics
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- The Hospital for Sick ChildrenUniversity, hospital or other
- Collaborators
- Canadian Cystic Fibrosis Foundation
- Study start
- Mar 2008
- Primary completion
- Dec 2008 (actual)
- Study completion
- Dec 2008 (actual)
- First posted
- 13 Mar 2008 (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.
This study is examining the effect of hypertonic saline compared to placebo on the Lung Clearance Index in Cystic Fibrosis patients.
Detailed description
The life expectancy of patients with Cystic Fibrosis (CF) has greatly increased over time due to improved clinical care. While this is certainly beneficial to CF patients, the overall stability in lung function has made it more difficult to assess the effect of therapeutic interventions. Currently, FEV1 (forced expiratory volume in 1 second) remains the primary outcome measure for most clinical trials, but many CF patients have normal pulmonary function and the annual rate of decline is now less than 2% in large specialized centres. Therefore, additional parameters are needed that are more sensitive parameters to define abnormalities in CF patients and be used in therapeutic trials.
One such promising parameter is the Lung Clearance Index (LCI). The LCI is a test to quantify ventilation inhomogeneity by assessing the washout of an inhaled inert gas. Abnormalities in gas clearance from the lung are largely due to retention of inhaled gases due to mucous obstruction in the lower airways.
Interventions that improve mucus accumulation are expected to improve the LCI. Hypertonic saline has been shown to increase airway surface liquid (ASL), mucociliary clearance and pulmonary function. Therefore, this study will examine the effect of hypertonic saline compared to placebo on the Lung Clearance Index in Cystic Fibrosis patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: hypertonic saline (7 %) and isotonic saline (0.9%) |
| 2 | Experimental | Drug: hypertonic saline (7 %) and isotonic saline (0.9%) |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Diagnosis of CF as defined by two or more clinical features of CF and a documented sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis test or a genotype showing two well characterized disease causing mutations
- Informed consent and verbal assent (as appropriate) provided by the subject's parent or legal guardian and the subject
- 6-18 years of age at enrolment and able to perform reproducible spirometry
- Clinically stable at enrolment
- FEV1 % predicted ≥ 80 % as calculated by the Wang reference equations
Key exclusion criteria (4 listed)
- Respiratory culture positive for NTM or B. cepacia complex within past year or screening
- Use of intravenous antibiotics or oral quinolones within 14 days of screening
- Investigational drug use within 30 days of screening
- Physical findings at screening that would compromise the safety of the participant or the quality of the study data
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 saline (7 %) and isotonic saline (0.9%)Patients in study arm 1 will receive hypertonic saline (7 %) during the first phase of the study and will crossover to placebo (0.9% isotonic saline) during the second phase. Both drugs will be administered via PARI LC® Star reusable nebulizer. | Drug | 1 |
| hypertonic saline (7 %) and isotonic saline (0.9%)Patients in study arm 2 will receive placebo (0.9% isotonic saline) during the first phase of the study and will crossover to hypertonic saline (7 %) during the second phase. Both drugs will be administered via PARI LC® Star reusable nebulizer. | Drug | 2 |
Primary outcome
Change in LCI from baseline to end of treatment in hypertonic saline treated patients versus patients receiving placebo (isotonic saline)
Time frame: Duration of patient's involvement in study
3 secondary outcomes
Change in FEV1 % predicted
Time frame: Duration of patient's involvement in study
Change in FVC (forced vital capacity) % predicted
Time frame: Duration of patient's involvement in study
Change in FEF25-75% (forced expiratory flow between 25 and 75 % of vital capacity) predicted
Time frame: Duration of patient's involvement in study
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 2 Sep 2013.
- Results publication: PubMed 20435858Amin R, Subbarao P, Jabar A, Balkovec S, Jensen R, Kerrigan S, Gustafsson P, Ratjen F. Hypertonic saline improves the LCI in paediatric patients with CF with normal lung function. Thorax. 2010 May;65(5):379-83. doi: 10.1136/thx.2009.125831.
- Derived from this study: PubMed 37319354Wark P, McDonald VM, Smith S. Nebulised hypertonic saline for cystic fibrosis. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD001506. doi: 10.1002/14651858.CD001506.pub5.
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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.
Felix Ratjen, MD principal investigator
The Hospital for Sick Children
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing hypertonic saline (7 %) and isotonic saline (0.9%) in people with Cystic Fibrosis, run by The Hospital for Sick Children at 1 site in Canada.
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 6 inclusion and 4 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 2 Sep 2013. 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 Canada. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Change in LCI from baseline to end of treatment in hypertonic saline treated patients versus patients receiving placebo (isotonic saline) (time frame: Duration of patient's involvement in study). 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 NCT00635141, which also lists 2 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 2 Sep 2013.