Cystic FibrosisNCT00589745
Nasal Potential Difference (NPD) for the Diagnosis of Cystic Fibrosis
A study testing Nasal potential difference measurement in people with Cystic Fibrosis, run by Children's Hospital of Philadelphia at 1 site in United States.
Trial summary
The purpose of this research study is to provide a novel method for the diagnosis of Cystic Fibrosis (CF). This protocol is designed to test the ability of the cells to regulate the movement of salt and water in people with features of CF in whom the diagnosis is not entirely clear. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Nasal potential difference measurement
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2003to 18 Dec 2019
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- All ethnic groups accepted
- Individuals with borderline or normal sweat chloride, one or no identified CFTR mutations and a clinical history suggestive of CF.
- Children less than 6-8 years of age may be unable to cooperate (hold still) for the procedure, or be frightened by the procedure. Sedation may be necessary for performance of the procedure on a young child
Who cannot join
- n/a
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: Measurement of Transepithelial Nasal Potential Difference (NPD) for the Diagnosis of Cystic Fibrosis
The purpose of this research study is to provide a novel method for the diagnosis of Cystic Fibrosis (CF). This protocol is designed to test the ability of the cells to regulate the movement of salt and water in people with features of CF in whom the diagnosis is not entirely clear. We will be studying these cells in the nose, by a technique called nasal transepithelial potential difference (NPD).
Read the detailed description
Cystic Fibrosis (CF) is an autosomal recessive systemic disorder of exocrine glands and secretory epithelia. The disease results from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene which cause a variety of abnormalities. CFTR is absent or dysfunctional in cystic fibrosis. Most of the defects in CFTR result in abnormal chloride ion transport and abnormally viscous mucus. Nasal epithelial CFTR function will be assessed by the NPD procedure.
NPD measurements are useful as a diagnostic tool in individuals with borderline or normal sweat chlorides, one or no identified CFTR mutations, and a clinical history suggestive of CF. The NPD has become the gold standard for detection and quantification of CFTR function in the airways. The assay has also served as an important endpoint since 1981. It has been used extensively and is standardized to assess the ability of new therapeutics in studies designed to replace, repair or restore defective ion transport in CF patients.
The assay basically involves dripping small amounts of the test solutions into the anterior nostril in order to measure uptake or secretion of sodium and chloride. There are five solutions required for the NPD measurement:
- Solution #1: Buffered Ringers
- Solution #2: Solution #1+Amiloride
- Solution #3: Buffered Zero Chloride Solution + Amiloride
- Solution #4: Solution #3 + Isoproterenol
- Solution #5: Solution #4 + Adenosine 5'-Triphosphate-Disodium salt (ATP)
The Cystic Fibrosis Therapeutics Development Network (TDN) is a formal affiliation of CF Research Centers in the US. Two Standard Operating Procedures have been developed by the TDN for the NPD procedure and qualification of those performing the measurement.
Procedure Details:
Briefly, a series of stopcocks is configured to allow perfusion of the above solutions through the port at the tip of an exploring catheter (PE50 tubing. All test solutions are perfused at a rate of 5.0 cc per minute. In each nostril, the PD readings in Ringer's at 0.5, 1.0, 1.5, 2, and 3 cm (lumen negative) are averaged and taken as the average 'baseline' PD. The catheter tip is then placed at the most negative PD site and maintained for superperfusion measurements with a small piece of tape applied to the nasal tip (to hold the catheter for the duration of the protocol). All solutions are warmed to 37˚C prior to contact with the nasal mucosa.
For each perfusion condition, a steady-state recording is obtained. The recording lasts at least one minute (for Solution #1) and for three minutes (for Solutions #2, 3, and 4) prior to proceeding to the next solution within the sequence. Solution #5 is perfused for a minimum of one minute, and confirms retention of ATP-activated Clˉ secretion in both CF and non-CF individuals. Several readings are obtained for data analysis, and then all nasal P.D. tracings are scored by investigators.
- Conditions
- Cystic Fibrosis
- Being tested
- Nasal potential difference measurement
- Keywords
- Diagnosis
- Medical subject headings
- Cystic Fibrosis, Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nasal potential difference measurementOther
Also called NPD
Nasal potential difference measurement involves dripping small amounts of 5 test solutions into the anterior nostril. This is designed to test the ability of the cells to regulate the movement of salt and water in people with features of CF in whom the diagnosis is not entirely clear.
| Study group | Role | What they receive |
|---|---|---|
| Subjects being evaluated for CF | Other | Subjects will be referred from physicians who are clinically concerned about the possibility of Cystic Fibrosis. Nasal potential difference measurement will be obtained to potentially help aid in diagnosis. Receives: Other: Nasal potential difference measurement |
What is being measured
The primary outcome is the main question the study is designed to answer.
The basis of analysis will be the NPD response patterns, which will be interpreted and classified by the PI, as consistent or inconsistent with the diagnosis of CF based on comparison with published standards.
Measured: Every visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2008 (estimated)
- Study startSep 2003
- Primary completion18 Dec 2019
- Study completion18 Dec 2019
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.
- More trials run by Children's Hospital of PhiladelphiaThe 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
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ronald Rubenstein, M.D., PhD., Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00589745
- Sponsor's protocol ID
- 98-001589
- Status last verified
- Jan 2020
- First submitted
- 27 Dec 2007
- Last updated
- 29 Jan 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Measurement of Transepithelial Nasal Potential Difference (NPD) for the Diagnosis of Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00589745
- Acronym / sponsor ID
- 98-001589
- Conditions
- Cystic Fibrosis
- Keywords
- Diagnosis
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Study start
- Sep 2003
- Primary completion
- 18 Dec 2019 (actual)
- Study completion
- 18 Dec 2019 (actual)
- First posted
- 10 Jan 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.
The purpose of this research study is to provide a novel method for the diagnosis of Cystic Fibrosis (CF). This protocol is designed to test the ability of the cells to regulate the movement of salt and water in people with features of CF in whom the diagnosis is not entirely clear. We will be studying these cells in the nose, by a technique called nasal transepithelial potential difference (NPD).
Detailed description
Cystic Fibrosis (CF) is an autosomal recessive systemic disorder of exocrine glands and secretory epithelia. The disease results from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene which cause a variety of abnormalities. CFTR is absent or dysfunctional in cystic fibrosis. Most of the defects in CFTR result in abnormal chloride ion transport and abnormally viscous mucus. Nasal epithelial CFTR function will be assessed by the NPD procedure.
NPD measurements are useful as a diagnostic tool in individuals with borderline or normal sweat chlorides, one or no identified CFTR mutations, and a clinical history suggestive of CF. The NPD has become the gold standard for detection and quantification of CFTR function in the airways. The assay has also served as an important endpoint since 1981. It has been used extensively and is standardized to assess the ability of new therapeutics in studies designed to replace, repair or restore defective ion transport in CF patients.
The assay basically involves dripping small amounts of the test solutions into the anterior nostril in order to measure uptake or secretion of sodium and chloride. There are five solutions required for the NPD measurement:
- Solution #1: Buffered Ringers
- Solution #2: Solution #1+Amiloride
- Solution #3: Buffered Zero Chloride Solution + Amiloride
- Solution #4: Solution #3 + Isoproterenol
- Solution #5: Solution #4 + Adenosine 5'-Triphosphate-Disodium salt (ATP)
The Cystic Fibrosis Therapeutics Development Network (TDN) is a formal affiliation of CF Research Centers in the US. Two Standard Operating Procedures have been developed by the TDN for the NPD procedure and qualification of those performing the measurement.
Procedure Details:
Briefly, a series of stopcocks is configured to allow perfusion of the above solutions through the port at the tip of an exploring catheter (PE50 tubing. All test solutions are perfused at a rate of 5.0 cc per minute. In each nostril, the PD readings in Ringer's at 0.5, 1.0, 1.5, 2, and 3 cm (lumen negative) are averaged and taken as the average 'baseline' PD. The catheter tip is then placed at the most negative PD site and maintained for superperfusion measurements with a small piece of tape applied to the nasal tip (to hold the catheter for the duration of the protocol). All solutions are warmed to 37˚C prior to contact with the nasal mucosa.
For each perfusion condition, a steady-state recording is obtained. The recording lasts at least one minute (for Solution #1) and for three minutes (for Solutions #2, 3, and 4) prior to proceeding to the next solution within the sequence. Solution #5 is perfused for a minimum of one minute, and confirms retention of ATP-activated Clˉ secretion in both CF and non-CF individuals. Several readings are obtained for data analysis, and then all nasal P.D. tracings are scored by investigators.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Subjects being evaluated for CF | Other | Other: Nasal potential difference measurementSubjects will be referred from physicians who are clinically concerned about the possibility of Cystic Fibrosis. Nasal potential difference measurement will be obtained to potentially help aid in diagnosis. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- All ethnic groups accepted
- Individuals with borderline or normal sweat chloride, one or no identified CFTR mutations and a clinical history suggestive of CF.
- Children less than 6-8 years of age may be unable to cooperate (hold still) for the procedure, or be frightened by the procedure. Sedation may be necessary for performance of the procedure on a young child
Key exclusion criteria (1 listed)
- n/a
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 |
|---|---|---|
| Nasal potential difference measurementAlso: NPDNasal potential difference measurement involves dripping small amounts of 5 test solutions into the anterior nostril. This is designed to test the ability of the cells to regulate the movement of salt and water in people with features of CF in whom the diagnosis is not entirely clear. | Other | Subjects being evaluated for CF |
Primary outcome
The basis of analysis will be the NPD response patterns, which will be interpreted and classified by the PI, as consistent or inconsistent with the diagnosis of CF based on comparison with published standards.
Time frame: Every visit
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 29 Jan 2020.
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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.
Ronald Rubenstein, M.D., PhD. principal investigator
Children's Hospital of Philadelphia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Nasal potential difference measurement in people with Cystic Fibrosis, run by Children's Hospital of Philadelphia 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 3 inclusion and 1 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 29 Jan 2020. 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.
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 basis of analysis will be the NPD response patterns, which will be interpreted and classified by the PI, as consistent or inconsistent with the diagnosis of CF based on comparison with published standards. (time frame: Every 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 29 Jan 2020.