Using Ivacaftor to treat chronic rhinosinusitis
Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis (Randomized Pilot Study Utilizing Ivacaftor for the Treatment of Refractory Gram-Negative Bacterial CRS)
This study is testing if the medication ivacaftor can help people with stubborn chronic rhinosinusitis feel better and reduce their symptoms.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | University of Alabama at Birmingham Academic / other |
| Locations | 1 site (Birmingham, Alabama) |
| Trial ID | NCT03439865 on ClinicalTrials.gov |
What this trial studies
This pilot study aims to evaluate the safety and tolerability of ivacaftor in patients with refractory chronic rhinosinusitis (CRS). It will assess whether ivacaftor can restore CFTR-mediated chloride secretions and improve CRS symptoms through various validated measures, including the SNOT-22 questionnaire and CT scan grading. The study will also investigate its effects on markers of sinonasal inflammation and infection. By exploring this novel approach, the study seeks to address the significant clinical need for new treatments for CRS.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older with a diagnosis of chronic rhinosinusitis and evidence of bacterial infection.
Not a fit: Patients under 18 years of age or those with acute illnesses other than sinusitis may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from chronic rhinosinusitis.
How similar studies have performed: While this approach is novel for chronic rhinosinusitis, similar studies targeting CFTR dysfunction have shown promise in other conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Birmingham, Alabama
- University of Alabama at Birmingham — Birmingham, Alabama, United States (Recruiting)
Study contacts
- Principal investigator: Bradford Woodworth, MD — University of Alabama at Birmingham
- Study coordinator: Norma Miller, RN
- Email: ncmiller@uabmc.edu
- Phone: (205) 975-6169
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.