Cystic FibrosisISIS PilotNCT00546663
Tolerability of Inhaled Hypertonic Saline in Infants With Cystic Fibrosis
A study testing inhaled 7% hypertonic saline (HS) in people with Cystic Fibrosis, run by CF Therapeutics Development Network Coordinating Center at 3 sites in 2 countries.
Trial summary
This is an open-label pilot study of the safety and tolerability of 7% hypertonic saline inhaled twice daily for 14 days in infants with CF, 12 to 30 months of age. Read more about this study
- Who is it for?
- Ages 12 to 30 months
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- inhaled 7% hypertonic saline (HS)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 3 sitesin 2 countries
- Study dates
- Sep 2007to Jul 2008
- Phase
- Interventional
- 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 to 30 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of CF as defined by one 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 by parent or legal guardian
- 12-30 months of age at enrollment
Who cannot join
- Wheezing at the baseline evaluation at the enrollment visit
- Oxygen saturation < 95 % at the baseline evaluation at the enrollment visit
- Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate, or new rhinorrhea, nasal congestion or rhinorrhea, with onset in the week preceding the enrollment visit
- Investigational drug use within 30 days prior to the enrollment visit
- Known intolerance of albuterol
- Current enrollment in a therapeutic clinical trial
- Condition or situation which, in the opinion of the investigator, would affect the ability of the patient or family to complete study procedures
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
University of North Carolina
Chapel Hill, North Carolina, 27599-7248
Children's Hospital and Regional Medical Center
Seattle, Washington, 98105
About this study
Official title: A Pilot Study to Evaluate the Tolerability of Inhaled 7% Hypertonic Saline in Infants With Cystic Fibrosis
This is an open-label pilot study of the safety and tolerability of 7% hypertonic saline inhaled twice daily for 14 days in infants with CF, 12 to 30 months of age.
Read the detailed description
The efficacy and safety of hypertonic saline (HS) in CF patients over 6 years of age has been demonstrated in clinical trials of 2 to 48 weeks' duration. Based on these results, a large randomized, placebo-controlled trial of the efficacy and safety of 7% HS administered twice daily for 48 weeks to infants with CF, 4 to 15 months of age at enrollment, is planned (the Infant Study of Inhaled Saline (ISIS) trial). It is anticipated that 150 infants at up to 16 sites will be enrolled in the ISIS trial.
To date, the only evaluations of the safety of HS in infants with CF have been small single-dose studies. There has been no evaluation of the tolerability of chronic HS administration. The goal of this study is to assess the safety and tolerability of exposure to 14 days of 7% HS administered twice daily in infants with CF, prior to enrolling subjects in the planned large, randomized, controlled trial. Conduct of this study will provide evidence for the tolerability of chronic HS administration in infants with CF and estimates of the proportion of infants who do not tolerate chronic HS treatment. The results will be used to establish the appropriate measures of tolerability at enrollment in the ISIS trial, and to refine sample size estimates to account for withdrawal due to intolerance of HS.
- Conditions
- Cystic Fibrosis
- Being tested
- inhaled 7% hypertonic saline (HS)
- Keywords
- 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.
inhaled 7% hypertonic saline (HS)Drug
Also called Hyper-Sal™, inhaled saline
7% hypertonic saline administered twice daily for 14 days by nebulization.
A Pari Sprint Junior nebulizer equipped with a Pari Baby face mask and a Pari Proneb compressor will be used to administer the HS (PARI Respiratory Equipment, Inc., Midlothian, VA).
To minimize the risk of cough and bronchospasm with HS inhalation, infants will be pre-treated prior to each dose of HS with albuterol by metered dose inhaler
| Study group | Role | What they receive |
|---|---|---|
| Open-label | Experimental | Not described Receives: Drug: inhaled 7% hypertonic saline (HS) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of infants who are intolerant of single and repeated doses of HS according to protocol-defined criteria
Measured: At the enrollment visit, during the period of home administration (Days 0 to 14), and at the final study visit.
Secondary outcomes (5)
New or increased cough, increased qualitative breathing rate, new or increased noisy breathing, or new or increased emesis at home as noted by parents on the daily symptom report
Measured: During the period of home administration (Days 0 to 14)
Change in respiratory rate, oxygen saturation, or cough frequency between the baseline measurement and measurements obtained after HS administration
Measured: At the enrollment visit
Change in respiratory rate, oxygen saturation, or cough frequency between the baseline value at the enrollment visit and the value at the final study visit
Measured: Over two weeks of study participation
Unanticipated adverse events
Measured: Over two weeks of study participation
Adherence, as measured by (1) the number of doses of HS administered per the home symptom report and (2) returned study drug vials
Measured: During the period of home administration (Days 0 to 14)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2007 (estimated)
- Study startSep 2007
- Primary completionMay 2008
- Study completionJul 2008
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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- CF Therapeutics Development Network Coordinating Center
- Sponsor type
- Research network
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
- Investigators
- Margaret Rosenfeld, MD, MPH, Principal investigator, Seattle Children's HospitalStephanie Davis, MD, Principal investigator, University of North CarolinaFelix Ratjen, MD, PhD, Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00546663
- Sponsor's protocol ID
- ISIS001
- Acronym
- ISIS Pilot
- Status last verified
- Aug 2011
- First submitted
- 17 Oct 2007
- Last updated
- 24 Aug 2011
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study to Evaluate the Tolerability of Inhaled 7% Hypertonic Saline in Infants With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00546663
- Acronym / sponsor ID
- ISIS Pilot / ISIS001
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, hypertonic saline
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- CF Therapeutics Development Network Coordinating CenterResearch network
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- Sep 2007
- Primary completion
- May 2008 (actual)
- Study completion
- Jul 2008 (actual)
- First posted
- 19 Oct 2007 (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 is an open-label pilot study of the safety and tolerability of 7% hypertonic saline inhaled twice daily for 14 days in infants with CF, 12 to 30 months of age.
Detailed description
The efficacy and safety of hypertonic saline (HS) in CF patients over 6 years of age has been demonstrated in clinical trials of 2 to 48 weeks' duration. Based on these results, a large randomized, placebo-controlled trial of the efficacy and safety of 7% HS administered twice daily for 48 weeks to infants with CF, 4 to 15 months of age at enrollment, is planned (the Infant Study of Inhaled Saline (ISIS) trial). It is anticipated that 150 infants at up to 16 sites will be enrolled in the ISIS trial.
To date, the only evaluations of the safety of HS in infants with CF have been small single-dose studies. There has been no evaluation of the tolerability of chronic HS administration. The goal of this study is to assess the safety and tolerability of exposure to 14 days of 7% HS administered twice daily in infants with CF, prior to enrolling subjects in the planned large, randomized, controlled trial. Conduct of this study will provide evidence for the tolerability of chronic HS administration in infants with CF and estimates of the proportion of infants who do not tolerate chronic HS treatment. The results will be used to establish the appropriate measures of tolerability at enrollment in the ISIS trial, and to refine sample size estimates to account for withdrawal due to intolerance of HS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open-label | Experimental | Drug: inhaled 7% hypertonic saline (HS) |
Who may qualify
- Ages
- 12 to 30 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Diagnosis of CF as defined by one 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 by parent or legal guardian
- 12-30 months of age at enrollment
Key exclusion criteria (7 listed)
- Wheezing at the baseline evaluation at the enrollment visit
- Oxygen saturation < 95 % at the baseline evaluation at the enrollment visit
- Acute intercurrent respiratory infection, defined as an increase in cough, wheezing, or respiratory rate, or new rhinorrhea, nasal congestion or rhinorrhea, with onset in the week preceding the enrollment visit
- Investigational drug use within 30 days prior to the enrollment visit
- Known intolerance of albuterol
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 |
|---|---|---|
| inhaled 7% hypertonic saline (HS)Also: Hyper-Sal™, inhaled saline7% hypertonic saline administered twice daily for 14 days by nebulization. A Pari Sprint Junior nebulizer equipped with a Pari Baby face mask and a Pari Proneb compressor will be used to administer the HS (PARI Respiratory Equipment, Inc., Midlothian, VA). To minimize the risk of cough and bronchospasm with HS inhalation, infants will be pre-treated prior to each dose of HS with albuterol by metered dose inhaler | Drug | Open-label |
Primary outcome
The proportion of infants who are intolerant of single and repeated doses of HS according to protocol-defined criteria
Time frame: At the enrollment visit, during the period of home administration (Days 0 to 14), and at the final study visit.
5 secondary outcomes
New or increased cough, increased qualitative breathing rate, new or increased noisy breathing, or new or increased emesis at home as noted by parents on the daily symptom report
Time frame: During the period of home administration (Days 0 to 14)
Change in respiratory rate, oxygen saturation, or cough frequency between the baseline measurement and measurements obtained after HS administration
Time frame: At the enrollment visit
Change in respiratory rate, oxygen saturation, or cough frequency between the baseline value at the enrollment visit and the value at the final study visit
Time frame: Over two weeks of study participation
Unanticipated adverse events
Time frame: Over two weeks of study participation
Adherence, as measured by (1) the number of doses of HS administered per the home symptom report and (2) returned study drug vials
Time frame: During the period of home administration (Days 0 to 14)
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 24 Aug 2011.
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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.
Margaret Rosenfeld, MD, MPH principal investigator
Seattle Children's Hospital
Stephanie Davis, MD principal investigator
University of North Carolina
Felix Ratjen, MD, PhD principal investigator
The Hospital for Sick Children
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing inhaled 7% hypertonic saline (HS) in people with Cystic Fibrosis, run by CF Therapeutics Development Network Coordinating Center at 3 sites in 2 countries.
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 3 inclusion and 7 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 24 Aug 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, 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.
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?
The proportion of infants who are intolerant of single and repeated doses of HS according to protocol-defined criteria (time frame: At the enrollment visit, during the period of home administration (Days 0 to 14), and at the final study visit.). 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 24 Aug 2011.