A Phase 2a, randomized, double-blind, placebo-controlled, multi-center study to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of GB001 compared with placebo over 16 weeks of treatment in patients with chronic rhinosinusitis with or without nasal polyposis (NP).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
97
Change From Baseline to Week 16 in Sino-Nasal Outcome Test (SNOT) 22 Total Score
The SNOT-22 is a validated questionnaire to assess the impact of chronic rhinosinusitis (CRS) on quality of life and utilizes a 2-week recall period. It is a 22-item outcome measure on a 5-point category scale applicable to sinonasal conditions and surgical treatments. The total scores range from 0 to 110 with higher total scores implying greater impact of CRS on quality of life.
Time frame: Baseline, Week 16
Change From Baseline to Week 16 in Lund-Mackay Score
Lund-Mackay scores are based on centralized imaging data assessments and are scored by blinded central reading at the imaging core laboratory. The Lund-Mackay system is based on localization with points given for degree of opacification: 0=normal, 1=partial opacification, 2=total opacification. These points are then applied to each sinus (maxillary, anterior ethmoid, posterior ethmoid, sphenoid, and frontal sinus) on each side. The osteomeatal complex on each side is graded as 0=not occluded, or 2=occluded. The maximum score is 12 per side, for a total score ranging from 0 to 24.
Time frame: Baseline, Week 16
Change From Baseline to Week 16 in Nasal Polyp Score (NPS)
The bilateral endoscopic NPS is the sum of the right and left nostril scores, as evaluated by means of blinded, centrally read nasal endoscopy and ranges from 0 to 8. NP is graded based on polyp size: 0 = No polyps, 1 = Small polyps in the middle meatus not reaching below the inferior border of the middle turbinate, 2 = Polyps reaching below the lower border of the middle turbinate, 3 = Large polyps reaching the lower border of the inferior turbinate or polyps medial to the middle turbinate, and 4 = Large polyps causing complete obstruction of the inferior nasal cavity.
Time frame: Baseline, Week 16
Time to First Response in NPS
Response was defined as ≥ 1-point improvement from baseline. The bilateral endoscopic NPS is the sum of the right and left nostril scores, as evaluated by means of blinded, centrally read nasal endoscopy and ranges from 0-8. NP is graded based on polyp size: 0 = No polyps, 1 = Small polyps in the middle meatus not reaching below the inferior border of the middle turbinate, 2 = Polyps reaching below the lower border of the middle turbinate, 3 = Large polyps reaching the lower border of the inferior turbinate or polyps medial to the middle turbinate, and 4 = Large polyps causing complete obstruction of the inferior nasal cavity.
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Banner University of Arizona Medical Center
Tucson, Arizona, United States
NEA Baptist Clinic
Jonesboro, Arkansas, United States
Cedars-Sinai Medical Center
Los Angeles, California, United States
Sacramento Ear Nose and Throat Surgical and Medical Group, Inc.
Roseville, California, United States
Bensch Clinical Research LLC
Stockton, California, United States
Yale School of Medicine
New Haven, Connecticut, United States
Emory University
Atlanta, Georgia, United States
Rush University Medical Center
Chicago, Illinois, United States
ChicagoENT
Chicago, Illinois, United States
Iowa Head & Neck, PC
Des Moines, Iowa, United States
...and 28 more locations
Time frame: up to Week 16
Change From Baseline to Week 16 in Morning (AM) Nasal Congestion (NC) Score
AM NC score was assessed using a 0 to 3 categorical scale (where 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms and 3 = severe symptoms).
Time frame: Baseline, Week 16
Change From Baseline to Week 16 in AM Total Symptom Score (TSS)
AM TSS is the sum of the scores from the 4 AM symptom categories (congestion and/or obstruction, anterior rhinorrhea, posterior rhinorrhea, loss of sense of smell) and ranges from 0-12. Each symptom category was assessed using a 0 to 3 categorical scale (where 0 = no symptoms, 1 = mild symptoms, 2 = moderate symptoms and 3 = severe symptoms).
Time frame: Baseline, Week 16
Change From Baseline to Week 16 in University of Pennsylvania Smell Identification Test (UPSIT) Score
The UPSIT test consists of four booklets, each containing 10 odorants with one odorant per page. The test-time is about 15 minutes. The stimuli are embedded in 10-50 μm diameter plastic microcapsules on brown strips at the bottom of each page. Above each odorant strip is a multiple-choice question with four alternative words to describe the odor. The participant is asked to release the odorant by rubbing the brown-strip with the tip of a pencil and to indicate which of 4 words best describes the odor. An UPSIT result is scored from 0 to 40 where a higher score indicates better olfaction.
Time frame: Baseline, Week 16
Time to First Chronic Rhinosinusitis (CRS) Exacerbation
Chronic rhinosinusitis exacerbation is defined as deterioration of CRS symptoms requiring treatment with an antibiotic, an anti-inflammatory drug, or a symptom reliever; an Emergency Department visit; or hospitalization.
Time frame: up to Week 16
Percentage of Participants With a Treatment-Emergent Adverse Event (AE)
An adverse event (AE) is any untoward medical occurrence in a participant, whether or not considered related to study drug. Abnormal laboratory test results or other safety assessments, including those that worsened from baseline, that were considered clinically significant in the medical and scientific judgment of the investigator were to be reported as AEs.
Time frame: From first dose of study drug through Week 20