NCT03439865 · University of Alabama at Birmingham
Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis
What this study is about
The purpose of this pilot study is to explore wither ivacaftor in refractory CRS patients will demonstrate safety and how well patients handle the treatment; restore CFTR-mediated Cl- secretions as measured by EDSPD testing; produce detectable improvements in validated measures of CRS including the SNOT-22 questionnaire, Lund-MacKay CT scan grading, and Lund-Kennedy endoscopic scores; and provide beneficial effects on readily measured markers of sinonasal inflammation and infection (IP-10, IL-8, and Pseudomonas CFUs).
View original scientific description
The purpose of this pilot study is to explore wither ivacaftor in refractory CRS patients will demonstrate safety and tolerability; restore CFTR-mediated Cl- secretions as measured by EDSPD testing; produce detectable improvements in validated measures of CRS including the SNOT-22 questionnaire, Lund-MacKay CT scan grading, and Lund-Kennedy endoscopic scores; and provide beneficial effects on readily measured markers of sinonasal inflammation and infection (IP-10, IL-8, and Pseudomonas CFUs).
Interventions
DRUG
Ivacaftor
150 mg tablet PO BID x 14 days
DRUG
standard of care treatment
topical nasal steroid spray and culture-directed antibiotics x 14 days
Primary outcome measures
Improvement in quality of life measures
Time frame: Screening to Day 30
Comparison of 22-tem Sino-Nasal Outcomes Test (SNOT-22) scores collected at Screening, Day 1, Day 14, and Day 30. The SNOT-22 is a 22-item questionnaire about rhinosinusitis symptoms and the social/emotional consequences for quality of life. The Likert scale ranges from 1-5, where 1 = "No Problem" and 5 = "Problem as bad as it can be." A total score is collected, ranging from 0-110; the higher the score, the worse the outcome.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 18 years of age
- Patient has provided informed consent
- Diagnosis of CRS made by one of the investigators
- Standard of care CT scan with definitive demonstration of isolated or diffuse mucosal thickening, bone changes, and air fluid levels, obtained within 30 days of treatment
- Positive culture of at least one gram negative bacteria (e.g. Pseudomonas, E. coli, Steenotrophomonas) within 30 days prior to testing
- Previous surgery with (at least) exposed maxillary and ethmoid sinuses
- Ability to perform EDSPD testing such that nasal cavity space and sinus openings are sufficient for catheter placement
- Negative pregnancy test for females of childbearing potential within 72 hours of testing and start of study treatment
Exclusion criteria
- \< 18 years of age
- Acute illness other than sinusitis within 2 weeks before start of study treatment that, in the opinion of the investigator, would preclude participation
- Currently taking medications that are moderate or strong CP3A inhibitors
- History of asthma attack requiring emergency room visit or treatment with oral steroids within 2 months prior to study treatment
- History of solid organ or hematological transplantation
- History of known immunodeficiency, autoimmune or granulomatous disorder
- Serum creatinine \> 1.5x upper normal limit
- Abnormal liver function, as defined by serum AST \> 2x upper normal limit, serum ALT \> 2x upper normal limit, Alkaline phosphatase \> 2x upper normal limit, Total bilirubin \> 2x upper normal limit
- Women who are pregnant or breastfeeding
Where
- Birmingham, Alabama
Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 22, 2026 · Source of record for eligibility and locations