Cystic FibrosisNCT04056702
Impact of Triple Combination CFTR Therapy on Sinus Disease.
An observational study of Cystic Fibrosis, run by Jennifer Taylor-Cousar at 1 site in United States.
Trial summary
The study's main goal is to observe how effective elexacaftor-tezacaftor-ivacaftor is for improving the symptoms and signs of CF-related sinus disease. Read more about this study
- Who is it for?
- Ages 18 to 89 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Elexacaftor-tezacaftor-ivacaftor exposure
- How many people
- 31 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Sep 2019to Jul 2022
- Phase
- Observational
- 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 18 to 89 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: People with cystic fibrosis and chronic sinusitis.
Who may be able to join
- Subjects from 18 to 89 years old.
- Subjects with cystic fibrosis and comorbid chronic sinus disease (historic diagnosis based on physician assessment of signs and symptoms and medical record).
- Subjects who clinically elect to initiate elexacaftor-tezacaftor-ivacaftor therapy and/or that are ineligible for CFTR modulator therapy based on their genotype, but have class I/II mutations based on sweat chloride > 90 mmol/L.
Who cannot join
- Subjects under the age of 18 or over the age of 89.
- Subjects who do not elect to initiate elexacaftor-tezacaftor-ivacaftor for clinical reasons.
- Subjects who had sinus surgery within the last 6 months or will have sinus surgery during the study period.
- Subjects who have had a recent pulmonary exacerbation or viral infection within two weeks of initial visit.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
National Jewish Health
Denver, Colorado, 80206
Connor Balkissoon, 303-270-2768, balkissoonc@njhealth.org
About this study
Official title: Impact of Elexacaftor-tezacaftor-ivacaftor Triple Combination CFTR Therapy on Sinus Disease: Quantitative Sinus Computed Tomography, Patient Reported Outcomes and Cellular and Molecular Changes
The study's main goal is to observe how effective elexacaftor-tezacaftor-ivacaftor is for improving the symptoms and signs of CF-related sinus disease.
Read the detailed description
To determine if elexacfator-tezacaftor-ivacaftor improves the signs and symptoms of CF-related sinus disease, before and after initiation of triple combination therapy, each subject will undergo sinus CT scan and complete questionnaires related to signs and symptoms of sinus disease.
- Conditions
- Cystic Fibrosis
- Being tested
- Elexacaftor-tezacaftor-ivacaftor exposure
- Keywords
- Chronic Sinusitis
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Elexacaftor-tezacaftor-ivacaftor exposureDrug
Once a clinical decision has been made to prescribed a patient triple combination therapy, people with CF are eligible to participate in this study.
| Study group | Role | What they receive |
|---|---|---|
| Prescribed triple combination | A group of 60 people with CF who are clinically prescribed elexacaftor-tezacaftor-ivacaftor and have chronic sinusitis Receives: Drug: Elexacaftor-tezacaftor-ivacaftor exposure | |
| Not eligible for modulators | A group of 10 patients with two class I/II mutations who are ineligible for elexacaftor-tezacaftor-ivacaftor based on their genotype and have chronic sinusitis. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Sinus CT opacification.
Calculated from an automated quantification of the size of sinuses.
Measured: Change in Sinus CT opacification between the initial and 6 month visits.
Secondary outcomes (2)
Change in 22-item Sino-Nasal Outcome Test (SNOT-22) score.
Validated, disease-specific measure of sinus symptoms. Contains 22 questions that are all scored from 0 to 5, when summed a total score ranges form 0 to 110. Higher scores represent worse sinus symptoms.
Measured: Change in SNOT-22 score between the initial and 6 month visits.
Change in Questionnaire for Olfactory Disorders (QOD) score
Validated, disease-specific measure of olfaction, i.e. the ability to smell. Contains 19 questions that are scored form 0 to 3, when summed a total score can range form 0 to 57. Higher scores indicate greater impact on a subjects sense of smell.
Measured: Change in QOD score between the initial and 6 month visits.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2019
- Study start5 Sep 2019
- Primary completionJul 2021 (estimated)
- Study completionJul 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 31 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Jennifer Taylor-Cousar
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor-investigator, Jennifer Taylor-Cousar, National Jewish Health
- Investigators
- Jennifer TaylorCousar, M.D., Principal investigator, National Jewish Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04056702
- Sponsor's protocol ID
- HS-3236
- Status last verified
- Mar 2021
- First submitted
- 27 Jul 2019
- Last updated
- 11 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Miller JE, Taylor-Cousar JL, Overdevest JB, Khatiwada A, Mace JC, Alt JA, Bodner TE, Chowdhury NI, DiMango EA, Eshaghian PH, Getz AE, Gudis DA, Han EJ, Hwang PH, Keating CL, Khanwalkar A, Kimple AJ, Lee JT, Li D, Markarian K, Norris M, Nayak JV, Owens C, Patel ZM, Poch K, Schlosser RJ, Smith KA, Smith TL, Soler ZM, Suh JD, Tervo JP, Turner GA, Wang MB, Saavedra MT, Beswick DM. Determining the minimal clinically important difference for the questionnaire of olfactory disorders in people with cystic fibrosis and factors associated with improvement after highly effective modulator therapy. Int Forum Allergy Rhinol. 2024 Jun;14(6):1079-1087. doi: 10.1002/alr.23312. Epub 2023 Dec 25.
PubMed 38145393
Study at a glance
From the registry record, last updated by the sponsor on 11 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Impact of Elexacaftor-tezacaftor-ivacaftor Triple Combination CFTR Therapy on Sinus Disease: Quantitative Sinus Computed Tomography, Patient Reported Outcomes and Cellular and Molecular Changes
- ClinicalTrials.gov ID
- NCT04056702
- Acronym / sponsor ID
- HS-3236
- Conditions
- Cystic Fibrosis
- Keywords
- Chronic Sinusitis
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 31 participants (estimated)
- Lead sponsor
- Jennifer Taylor-CousarUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- 5 Sep 2019 (actual)
- Primary completion
- Jul 2021 (estimated)
- Study completion
- Jul 2022 (estimated)
- First posted
- 14 Aug 2019 (actual)
- 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 study's main goal is to observe how effective elexacaftor-tezacaftor-ivacaftor is for improving the symptoms and signs of CF-related sinus disease.
Detailed description
To determine if elexacfator-tezacaftor-ivacaftor improves the signs and symptoms of CF-related sinus disease, before and after initiation of triple combination therapy, each subject will undergo sinus CT scan and complete questionnaires related to signs and symptoms of sinus disease.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Prescribed triple combination | – | Drug: Elexacaftor-tezacaftor-ivacaftor exposureA group of 60 people with CF who are clinically prescribed elexacaftor-tezacaftor-ivacaftor and have chronic sinusitis |
| Not eligible for modulators | – | –A group of 10 patients with two class I/II mutations who are ineligible for elexacaftor-tezacaftor-ivacaftor based on their genotype and have chronic sinusitis. |
Who may qualify
- Ages
- 18 to 89 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- People with cystic fibrosis and chronic sinusitis.
Key inclusion criteria (3 listed)
- Subjects from 18 to 89 years old.
- Subjects with cystic fibrosis and comorbid chronic sinus disease (historic diagnosis based on physician assessment of signs and symptoms and medical record).
- Subjects who clinically elect to initiate elexacaftor-tezacaftor-ivacaftor therapy and/or that are ineligible for CFTR modulator therapy based on their genotype, but have class I/II mutations based on sweat chloride > 90 mmol/L.
Key exclusion criteria (4 listed)
- Subjects under the age of 18 or over the age of 89.
- Subjects who do not elect to initiate elexacaftor-tezacaftor-ivacaftor for clinical reasons.
- Subjects who had sinus surgery within the last 6 months or will have sinus surgery during the study period.
- Subjects who have had a recent pulmonary exacerbation or viral infection within two weeks of initial visit.
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 |
|---|---|---|
| Elexacaftor-tezacaftor-ivacaftor exposureOnce a clinical decision has been made to prescribed a patient triple combination therapy, people with CF are eligible to participate in this study. | Drug | Prescribed triple combination |
Primary outcome
Change in Sinus CT opacification.
Time frame: Change in Sinus CT opacification between the initial and 6 month visits.
Calculated from an automated quantification of the size of sinuses.
2 secondary outcomes
Change in 22-item Sino-Nasal Outcome Test (SNOT-22) score.
Time frame: Change in SNOT-22 score between the initial and 6 month visits.
Change in Questionnaire for Olfactory Disorders (QOD) score
Time frame: Change in QOD score between the initial and 6 month visits.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Mar 2021.
- Derived from this study: PubMed 38145393Miller JE, Taylor-Cousar JL, Overdevest JB, Khatiwada A, Mace JC, Alt JA, Bodner TE, Chowdhury NI, DiMango EA, Eshaghian PH, Getz AE, Gudis DA, Han EJ, Hwang PH, Keating CL, Khanwalkar A, Kimple AJ, Lee JT, Li D, Markarian K, Norris M, Nayak JV, Owens C, Patel ZM, Poch K, Schlosser RJ, Smith KA, Smith TL, Soler ZM, Suh JD, Tervo JP, Turner GA, Wang MB, Saavedra MT, Beswick DM. Determining the minimal clinically important difference for the questionnaire of olfactory disorders in people with cystic fibrosis and factors associated with improvement after highly effective modulator therapy. Int Forum Allergy Rhinol. 2024 Jun;14(6):1079-1087. doi: 10.1002/alr.23312. Epub 2023 Dec 25.
- Individual participant dataSharing planned: No
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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.
Jennifer TaylorCousar, M.D. principal investigator
National Jewish Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Jennifer Taylor-Cousar 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 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: status not confirmed, as of the sponsor's last update on 11 Mar 2021. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Connor Balkissoon (877-225-5654 ext. 2768, balkissoonc@njhealth.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Sinus CT opacification. (time frame: Change in Sinus CT opacification between the initial and 6 month visits.). 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 NCT04056702, 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 11 Mar 2021.