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Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis

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ClinicalTrials.gov Identifier: NCT03439865
Recruitment Status : Not yet recruiting
First Posted : February 20, 2018
Last Update Posted : March 5, 2018
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Brad Woodworth, MD, University of Alabama at Birmingham

Brief Summary:
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).

Condition or disease Intervention/treatment Phase
Chronic Rhinosinusitis (Diagnosis) Drug: Ivacaftor Drug: standard of care treatment Early Phase 1

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis (Randomized Pilot Study Utilizing Ivacaftor for the Treatment of Refractory Gram-Negative Bacterial CRS)
Estimated Study Start Date : July 2019
Estimated Primary Completion Date : July 2021
Estimated Study Completion Date : July 2022

Resource links provided by the National Library of Medicine

Drug Information available for: Ivacaftor

Arm Intervention/treatment
Experimental: standard of care treatment + ivacaftor
topical nasal steroid spray and culture-directed antibiotics + ivacaftor 150 mg tablet
Drug: Ivacaftor
150 mg tablet PO BID x 14 days
Other Name: Kalydeco

Drug: standard of care treatment
topical nasal steroid spray and culture-directed antibiotics x 14 days

Placebo Comparator: standard of care treatment
topical nasal steroid spray and culture-directed antibiotics
Drug: standard of care treatment
topical nasal steroid spray and culture-directed antibiotics x 14 days




Primary Outcome Measures :
  1. 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.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03439865


Contacts
Contact: Lisa Clemons, MSN, RN (205) 934-9714 lkclemons@uabmc.edu

Locations
United States, Alabama
University of Alabama at Birmingham Not yet recruiting
Birmingham, Alabama, United States, 35233
Contact: Lisa Clemons, MSN, RN    205-934-9714    lkclemons@uabmc.edu   
Principal Investigator: Brad Woodworth, MD         
Sponsors and Collaborators
University of Alabama at Birmingham
National Heart, Lung, and Blood Institute (NHLBI)
Investigators
Principal Investigator: Bradford Woodworth, MD University of Alabama at Birmingham

Responsible Party: Brad Woodworth, MD, Principal Investigator, University of Alabama at Birmingham
ClinicalTrials.gov Identifier: NCT03439865     History of Changes
Other Study ID Numbers: IRB-300000168
5R01HL133006-02 ( U.S. NIH Grant/Contract )
First Posted: February 20, 2018    Key Record Dates
Last Update Posted: March 5, 2018
Last Verified: March 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Additional relevant MeSH terms:
Sinusitis
Chronic Disease
Paranasal Sinus Diseases
Nose Diseases
Respiratory Tract Diseases
Respiratory Tract Infections
Otorhinolaryngologic Diseases
Disease Attributes
Pathologic Processes
Anti-Bacterial Agents
Ivacaftor
Anti-Infective Agents
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action