Impact of Highly Effective Modulator Therapy on the Cystic Fibrosis Microbiome
NCT ID: NCT05982795
Last Updated: 2025-12-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
30 participants
OBSERVATIONAL
2023-11-08
2026-12-01
Brief Summary
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1. How does this therapy impact bacterial communities in the paranasal sinuses?
2. How does this therapy impact inflammation in the paranasal sinuses and olfactory cleft?
3. How does this therapy impact sense of smell and sinonasal disease burden in individuals with cystic fibrosis?
4. How does this therapy impact disease-specific and general quality of life of individuals with cystic fibrosis?
Participants will be asked to provide samples from their nose, complete testing of their sense of smell, and complete surveys about their quality of life and sense of smell in this study.
Researchers will compare study results between patients who are currently undergoing Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy and patients who are not currently undergoing therapy.
Detailed Description
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A mucus sample from the middle meatus will be obtained and stored at 4ºC until culture analysis. Semi-quantitative bacterial cultures will be performed. Bacterial community composition will be quantified using 16S rRNA variable region (V4) amplicon sequencing and qPCR. Data preprocessing and taxonomic assignment will be performed using QIIME 2 (v 2023.2) and the SILVA 138.1 rRNA database. Abundance data will be further processed in R (v 4.3.0) using packages decontam, phyloseq, and vegan.
Samples from the middle meatus and olfactory cleft will be analyzed to investigate the inflammatory profiles of patients with cystic fibrosis. Both samples will be centrifuged at 4°C, 10,000 rpm for 10 minutes to extract the entire sample from the material. Samples will be transferred by pipette to cryovials, flash-frozen in liquid nitrogen, and stored in a -80°C environment until time of laboratory assay. The study will assess changes in immune status through cytokine arrays. To assess innate immune responses, a Bioplex Pro Human Inflammation Assay for 37 human cytokines (BD Biosciences) will be employed.
The study will also investigate if alterations in nasal microbiome and inflammatory profile correlate with changes in cystic fibrosis-specific quality of life (QOL) and olfaction outcomes. Participants will be asked to complete olfactory function testing using the Sniffin' Sticks extended test kit (MediSense, NL) and/or the University of Pennsylvania Smell Identification Test (UPSIT). Additionally, participants will be asked to complete the four following surveys to assess their quality of life as it relates to sinus symptom severity, disease control, and sense of smell: Cystic Fibrosis Questionnaire-Revised, Sino-nasal Outcome Test-22, Sinus Control Test, and Questionnaire of Olfactory Disorders.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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CFTR-MT Group
The cohort of adult cystic fibrosis patients that are currently on Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy (CFTR-MT) will be asked to complete the following research activities at baseline and every six months following for a two-year period.
1. Nasal endoscopy with microbiome swab and mucus collection through filter paper and sponge
2. The Sniffin' Sticks extended test kit and/or the University of Pennsylvania Smell Identification Test (UPSIT)
3. 4 validated quality of life questionnaires:
1. Cystic Fibrosis Questionnaire Revised (CFQ-R)
2. Sino-Nasal Outcome Test-22 (SNOT-22)
3. Sinus Control Test (SCT)
4. Questionnaire of Olfactory Disorders
Nasal endoscopy with microbiome swab and mucus collection through filter paper and sponge
A CultureSwab MaxV(+) (BD, Franklin Lakes, NJ) will be placed under endoscopic visualization to the middle meatus through a sterile sheath (red rubber urinary catheter) and gently rotated 5 full turns or until the tip is saturated with mucus (no more than 20 seconds).
Under direct visualization, a sterile Leukosorb sponge (Leukosorb; Pall Scientific, Port Washington, NY) will be placed in the middle meatus. A piece of Leukosorb filter paper will additionally be placed in the olfactory cleft. The sponge and paper will be left for 5 minutes and will then be removed under direct visualization and collected for analysis.
Olfactory Function Testing
Olfactory function will be tested using the Sniffin' Sticks extended test kit (MediSense, NL) using the established threshold, discrimination, and identification (TDI) scoring system. Odors will be delivered using felt-tip pens (Sniffin' Sticks, MediSense, NL). The pen cap will be removed from the pen for approximately 3 seconds, and the pen's tip will be brought in front of the subject's nose and carefully moved from left to right nostril. Threshold testing will be obtained by asking the subject to identify the pen containing the odor from two pens containing only solvent. This will be conducted starting with the lowest concentration of odor and moving stepwise until patient is no longer able to identify the odor from the solvent.
Subjects may also be asked to complete the University of Pennsylvania Smell Identification Test (UPSIT). The UPSIT can be self-administered and uses microencapsulated odorants which are released by scratching standardized odor-impregnated test booklets.
Survey Completion
Participants will be asked to complete the validated Cystic Fibrosis Questionnaire-Revised (CFQ-R) survey. This survey evaluates Cystic Fibrosis-specific and general quality of life metrics.
Participants will also be asked to complete the validated Sino-nasal Outcome Test-22 (SNOT-22) which is the most commonly used survey for evaluating outcomes in patients with chronic rhinosinusitis.
Participants will also be asked to complete the validated Sinus Control Test (SCT) - a 4-question questionnaire that assesses disease control in chronic rhinosinusitis.
Participants will be asked to complete the questionnaire of olfactory disorders (QOD). This is a 25-question validated survey used to assess the impact of olfactory dysfunction on patients with sinonasal disease.
Non-CFTR-MT Group
The cohort of adult cystic fibrosis patients that are not currently on Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy (CFTR-MT) will be asked to complete the following research activities at baseline and every six months following for a two-year period.
1. Nasal endoscopy with microbiome swab and mucus collection through filter paper and sponge
2. The Sniffin' Sticks extended test kit and/or the University of Pennsylvania Smell Identification Test (UPSIT)
3. 4 validated quality of life questionnaires:
1. Cystic Fibrosis Questionnaire Revised (CFQ-R)
2. Sino-Nasal Outcome Test-22 (SNOT-22)
3. Sinus Control Test (SCT)
4. Questionnaire of Olfactory Disorders
Nasal endoscopy with microbiome swab and mucus collection through filter paper and sponge
A CultureSwab MaxV(+) (BD, Franklin Lakes, NJ) will be placed under endoscopic visualization to the middle meatus through a sterile sheath (red rubber urinary catheter) and gently rotated 5 full turns or until the tip is saturated with mucus (no more than 20 seconds).
Under direct visualization, a sterile Leukosorb sponge (Leukosorb; Pall Scientific, Port Washington, NY) will be placed in the middle meatus. A piece of Leukosorb filter paper will additionally be placed in the olfactory cleft. The sponge and paper will be left for 5 minutes and will then be removed under direct visualization and collected for analysis.
Olfactory Function Testing
Olfactory function will be tested using the Sniffin' Sticks extended test kit (MediSense, NL) using the established threshold, discrimination, and identification (TDI) scoring system. Odors will be delivered using felt-tip pens (Sniffin' Sticks, MediSense, NL). The pen cap will be removed from the pen for approximately 3 seconds, and the pen's tip will be brought in front of the subject's nose and carefully moved from left to right nostril. Threshold testing will be obtained by asking the subject to identify the pen containing the odor from two pens containing only solvent. This will be conducted starting with the lowest concentration of odor and moving stepwise until patient is no longer able to identify the odor from the solvent.
Subjects may also be asked to complete the University of Pennsylvania Smell Identification Test (UPSIT). The UPSIT can be self-administered and uses microencapsulated odorants which are released by scratching standardized odor-impregnated test booklets.
Survey Completion
Participants will be asked to complete the validated Cystic Fibrosis Questionnaire-Revised (CFQ-R) survey. This survey evaluates Cystic Fibrosis-specific and general quality of life metrics.
Participants will also be asked to complete the validated Sino-nasal Outcome Test-22 (SNOT-22) which is the most commonly used survey for evaluating outcomes in patients with chronic rhinosinusitis.
Participants will also be asked to complete the validated Sinus Control Test (SCT) - a 4-question questionnaire that assesses disease control in chronic rhinosinusitis.
Participants will be asked to complete the questionnaire of olfactory disorders (QOD). This is a 25-question validated survey used to assess the impact of olfactory dysfunction on patients with sinonasal disease.
Interventions
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Nasal endoscopy with microbiome swab and mucus collection through filter paper and sponge
A CultureSwab MaxV(+) (BD, Franklin Lakes, NJ) will be placed under endoscopic visualization to the middle meatus through a sterile sheath (red rubber urinary catheter) and gently rotated 5 full turns or until the tip is saturated with mucus (no more than 20 seconds).
Under direct visualization, a sterile Leukosorb sponge (Leukosorb; Pall Scientific, Port Washington, NY) will be placed in the middle meatus. A piece of Leukosorb filter paper will additionally be placed in the olfactory cleft. The sponge and paper will be left for 5 minutes and will then be removed under direct visualization and collected for analysis.
Olfactory Function Testing
Olfactory function will be tested using the Sniffin' Sticks extended test kit (MediSense, NL) using the established threshold, discrimination, and identification (TDI) scoring system. Odors will be delivered using felt-tip pens (Sniffin' Sticks, MediSense, NL). The pen cap will be removed from the pen for approximately 3 seconds, and the pen's tip will be brought in front of the subject's nose and carefully moved from left to right nostril. Threshold testing will be obtained by asking the subject to identify the pen containing the odor from two pens containing only solvent. This will be conducted starting with the lowest concentration of odor and moving stepwise until patient is no longer able to identify the odor from the solvent.
Subjects may also be asked to complete the University of Pennsylvania Smell Identification Test (UPSIT). The UPSIT can be self-administered and uses microencapsulated odorants which are released by scratching standardized odor-impregnated test booklets.
Survey Completion
Participants will be asked to complete the validated Cystic Fibrosis Questionnaire-Revised (CFQ-R) survey. This survey evaluates Cystic Fibrosis-specific and general quality of life metrics.
Participants will also be asked to complete the validated Sino-nasal Outcome Test-22 (SNOT-22) which is the most commonly used survey for evaluating outcomes in patients with chronic rhinosinusitis.
Participants will also be asked to complete the validated Sinus Control Test (SCT) - a 4-question questionnaire that assesses disease control in chronic rhinosinusitis.
Participants will be asked to complete the questionnaire of olfactory disorders (QOD). This is a 25-question validated survey used to assess the impact of olfactory dysfunction on patients with sinonasal disease.
Eligibility Criteria
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Inclusion Criteria
* English-speaking
* Diagnosed with CF as established by genetic testing combined with clinical assessment and/or sweat chloride
* Patients being seen at the New Hampshire Cystic Fibrosis Center
* Adults able to sign informed consent
Exclusion Criteria
* Individuals who are not yet adults
* Prisoners
18 Years
99 Years
ALL
No
Sponsors
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Dartmouth-Hitchcock Medical Center
OTHER
Responsible Party
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Ryan.E.Little
Assistant Professor of Surgery, Geisel School of Medicine
Principal Investigators
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Ryan E Little, MD
Role: PRINCIPAL_INVESTIGATOR
Dartmouth-Hitchcock Medical Center
Locations
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Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, United States
Countries
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Central Contacts
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Other Identifiers
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STUDY02001953
Identifier Type: -
Identifier Source: org_study_id