GB001 in Adult Participants With Chronic Rhinosinusitis

A Phase 2a, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Effect of GB001 in Patients With Chronic Rhinosinusitis With or Without Nasal Polyps

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 Overview

• Status: Completed
• Conditions: Chronic Rhinosinusitis Without Nasal Polyps (CRSsNP); Chronic Rhinosinusitis With Nasal Polyps (CRSwNP)
• Intervention / Treatment: Drug: GB001; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 97
• Phase: Phase 2

Contacts and Locations

Study Locations

• Czechia
  • Hradec Králové, Czechia, 50005
    • University Hospital Hradec Kralove, Department of Otorhinolaryngology and Head and Neck Surgery
  • Olomouc, Czechia, 77900
    • ENT Outpatient Clinic Pavel Navratil
  • Praha 4, Czechia, 14046
    • Medicon a.s.
  • Rokycany, Czechia, 33722
    • Pulmonary Outpatient Clinic Rokycany s.r.o.
• Ukraine
  • Dnipro, Ukraine, 49006
    • Public Non-Profit Enterprise "Clinical Hospital of Emergency Medical Care" under Dnipro City Council
  • Kharkiv, Ukraine, 61166
    • Public Non-Profit Enterprise under KRC "Regional Clinical Specialized Center for Radiation Protection of the Public"
  • Kyiv, Ukraine, 03680
    • State Institution "O.S. Kolomiychenko Institute of Otolaryngology of National Academy of Medical Sciences of Ukraine"
  • Ternopil', Ukraine, 46023
    • Municipal Non-commercial Enterprise "Ternopil Municipal City Hospital #2"
  • Zaporizhia, Ukraine, 69032
    • Public Non-Profit Enterprise "City Hospital #3" under Zaporizhia City Council
• United States
  • Arizona
    • Tucson, Arizona, United States, 85724
      • Banner University of Arizona Medical Center
  • Arkansas
    • Jonesboro, Arkansas, United States, 72401
      • NEA Baptist Clinic
  • California
    • Los Angeles, California, United States, 90048
      • Cedars-Sinai Medical Center
    • Roseville, California, United States, 95661
      • Sacramento Ear Nose and Throat Surgical and Medical Group, Inc.
    • Stockton, California, United States, 95207
      • Bensch Clinical Research LLC
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale School of Medicine
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Medical Center
    • Chicago, Illinois, United States, 60657
      • ChicagoENT
  • Iowa
    • Des Moines, Iowa, United States, 50312
      • Iowa Head & Neck, PC
  • Kentucky
    • Louisville, Kentucky, United States, 40207
      • Advanced ENT and Allergy
    • Louisville, Kentucky, United States, 40205
      • Kentuckiana Ear, Nose, and Throat
  • Louisiana
    • Marrero, Louisiana, United States, 70072
      • Tandem Clinical Research LLC
  • Maryland
    • White Marsh, Maryland, United States, 21162
      • Chesapeake Clinical Research, Inc.
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Brigham and Women's Hospital
  • Nebraska
    • Bellevue, Nebraska, United States, 68123
      • Nebraska Medical Research Institute, Inc.
  • New Mexico
    • Albuquerque, New Mexico, United States, 87109
      • BreatheAmerica Albuquerque
  • New York
    • New Hyde Park, New York, United States, 11042
      • Northwell Health at ENT and Allergy Associates
    • New York, New York, United States, 10016
      • Northwell Health at ENT and Allergy Associates
    • Rochester, New York, United States, 14618
      • University of Rochester Medicine Otolaryngology
    • White Plains, New York, United States, 10605
      • Northwell Health at ENT and Allergy Associates
  • Oklahoma
    • Tulsa, Oklahoma, United States, 74136
      • Vital Prospects Clinical Research Institute, P.C.
  • Pennsylvania
    • Bethlehem, Pennsylvania, United States, 18017
      • Specialty Physician Associates
    • Philadelphia, Pennsylvania, United States, 19107
      • Thomas Jefferson University Department of Otolaryngology
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina, Department of Otolaryngology
  • Texas
    • Dallas, Texas, United States, 75231
      • AARA Research Center
    • Houston, Texas, United States, 77030
      • Memorial Hermann Medical Plaza
  • Virginia
    • Norfolk, Virginia, United States, 23507
      • Eastern Virginia Medical School
    • Richmond, Virginia, United States, 23235
      • Advanced Otolaryngology, P.C. DBA Richmond Ear, Nose and Throat

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• A diagnosis of chronic rhinosinusitis with nasal polyps (CRSwNP) or chronic rhinosinusitis without nasal polyps (CRSsNP) by a physician at least 12 weeks before Screening Visit
• Treated with stable intranasal corticosteroid (INCS) for at least 2 months prior to Screening Visit
• Presence of at least 2 nasal symptoms: nasal blockage/ obstruction/congestion or nasal discharge or facial pain/pressure or reduction/loss of smell
• Women of childbearing potential must use an acceptable method of contraception

Exclusion Criteria:

• Sino-Nasal Outcome Test-22 (SNOT-22) score < 20 at screening
• Asthma or chronic obstructive pulmonary disease (COPD) patients that are current smokers
• Use of other investigational drugs within 30 days, or within 5 half-lives, whichever is longer, prior to Screening Visit
• Pregnant or breastfeeding
• Pre-existing clinically important co-morbidities
• Regular use of systemic corticosteroids or immunosuppressive treatments

Other protocol-defined inclusion/exclusion criteria will apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: DOUBLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: GB001; GB001 40 mg once per day (QD) for 16 weeks	Drug: GB001; film-coated oral tablet
PLACEBO_COMPARATOR: Placebo; Placebo QD for 16 weeks	Drug: Placebo; film-coated oral tablet

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Baseline, Week 16

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	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).	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).	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.	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.	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.	From first dose of study drug through Week 20

Collaborators and Investigators

• Sponsor: GB001, Inc, a wholly owned subsidiary of Gossamer Bio, Inc.

Study record dates

Study Major Dates

• Study Start (ACTUAL): May 16, 2019
• Primary Completion (ACTUAL): July 9, 2020
• Study Completion (ACTUAL): August 5, 2020

Study Registration Dates

• First Submitted: May 17, 2019
• First Submitted That Met QC Criteria: May 17, 2019
• First Posted (ACTUAL): May 21, 2019

Study Record Updates

• Last Update Posted (ACTUAL): August 23, 2021
• Last Update Submitted That Met QC Criteria: August 19, 2021
• Last Verified: August 1, 2021

More Information

Terms related to this study

• Keywords: CRSwNP; Nasal Polyposis; CRSsNP; GB001; Gossamer Bio; Gossamer
• Additional Relevant MeSH Terms: Infections; Respiratory Tract Infections; Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Pathological Conditions, Anatomical; Paranasal Sinus Diseases; Nose Diseases; Sinusitis; Nasal Polyps; Polyps
• Other Study ID Numbers: GB001-2101; 2019-001682-33 (EUDRACT_NUMBER)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No