Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43871   clinical trials with a EudraCT protocol, of which   7290   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    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

    Summary
    EudraCT number
    		 2019-001682-33
    Trial protocol
    		 CZ  
    Global end of trial date
    05 Aug 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    27 Aug 2021
    First version publication date
    27 Aug 2021
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    GB001-2101
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03956862
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    GB001 Inc., a wholly owned subsidiary of Gossamer Bio, Inc.
    Sponsor organisation address
    3013 Science Park Road, San Diego, United States, 92121
    Public contact
    GB001, Inc. Study Director, GB001, Inc., wholly owned subsidiary of Gossamer Bio Inc., 866 668-4083, ClinicalTrials@gossamerbio.com
    Scientific contact
    GB001, Inc. Study Director, GB001, Inc., wholly owned subsidiary of Gossamer Bio Inc., 866 668-4083, ClinicalTrials@gossamerbio.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    05 Aug 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    09 Jul 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    05 Aug 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    • To evaluate the effect of GB001 on the Sino-Nasal Outcome Test-22 (SNOT-22)
    Protection of trial subjects
    This study was conducted in accordance with consensus ethical principles derived from international guidelines including the Declaration of Helsinki, Council for International Organizations of Medical Sciences (CIOMS) International Ethical Guidelines and International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All applicable local laws and regulations regarding patient safety were also followed.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    16 May 2019
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Czechia: 7
    Country: Number of subjects enrolled
    Ukraine: 42
    Country: Number of subjects enrolled
    United States: 48
    Worldwide total number of subjects
    97
    EEA total number of subjects
    7
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    88
    From 65 to 84 years
    9
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 Participants were recruited from the Czechia, Ukraine, and United States.

    Pre-assignment
    Screening details
    		 The study included a run-in period, during which eligibility for randomization was determined. 192 participants entered the run-in period, 97 of whom were randomized.

    Period 1
    Period 1 title
    overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Placebo QD for 16 weeks
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo QD for 16 weeks

    Arm title
    		 GB001
    Arm description
    		 GB001 40 mg QD for 16 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    GB001
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    GB001 40 mg QD for 16 weeks

    Number of subjects in period 1
    		Placebo GB001
    Started
    50
    47
    Completed
    48
    45
    Not completed
    2
    2
         Consent withdrawn by subject
    1
    1
         Lost to follow-up
    1
    1

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Placebo QD for 16 weeks

    Reporting group title
    GB001
    Reporting group description
    		 GB001 40 mg QD for 16 weeks

    Reporting group values
    		 Placebo GB001 Total
    Number of subjects
    		 50 47 97
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 51.6 ( 10.34 ) 51.7 ( 11.81 ) -
    Gender categorical
    Units: Subjects
        Female
    		 21 28 49
        Male
    		 29 19 48

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Placebo QD for 16 weeks

    Reporting group title
    GB001
    Reporting group description
    		 GB001 40 mg QD for 16 weeks

    Primary: Change From Baseline to Week 16 in Sino-Nasal Outcome Test (SNOT) 22 Total Score

    		 Close Top of page
    End point title
    		 Change From Baseline to Week 16 in Sino-Nasal Outcome Test (SNOT) 22 Total Score
    End point description
    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. Analysis Population Description Intent-to-treat (ITT) population: all participants who were randomized and received at least 1 dose of study treatment. Participants with a baseline and a post-baseline value.
    End point type
    Primary
    End point timeframe
    Baseline, Week 16
    End point values
    		 Placebo GB001
    Number of subjects analysed
    49
    45
    Units: score on a scale
        least squares mean (confidence interval 95%)
    -18.1 (-23.1 to -13.0)
    -18.3 (-23.5 to -13.1)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 v Placebo
    Number of subjects included in analysis
    94
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.9499
    Method
    		 Mixed models analysis
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.2
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -7.6
         upper limit
    		 7.1

    Secondary: Change From Baseline to Week 16 in Lund-Mackay Score

    		 Close Top of page
    End point title
    		 Change From Baseline to Week 16 in Lund-Mackay Score
    End point description
    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. Analysis Population Description ITT population: all participants who were randomized and received at least 1 dose of study treatment.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 16
    End point values
    		 Placebo GB001
    Number of subjects analysed
    50
    47
    Units: score on a scale
        least squares mean (confidence interval 95%)
    -0.6 (-1.6 to 0.4)
    -0.9 (-2.0 to 0.1)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 v Placebo
    Number of subjects included in analysis
    97
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.6778
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.3
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.8
         upper limit
    		 1.2

    Secondary: Change From Baseline to Week 16 in Nasal Polyp Score (NPS)

    		 Close Top of page
    End point title
    		 Change From Baseline to Week 16 in Nasal Polyp Score (NPS)
    End point description
    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. Analysis Population Description ITT population: all participants who were randomized and received at least 1 dose of study treatment. Participants with nasal polyps with a baseline value.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 16
    End point values
    		 Placebo GB001
    Number of subjects analysed
    35
    30
    Units: score on a scale
        least squares mean (confidence interval 95%)
    -0.8 (-1.4 to -0.3)
    -0.7 (-1.3 to -0.1)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 v Placebo
    Number of subjects included in analysis
    65
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.7914
    Method
    		 Mixed models analysis
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.1
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.7
         upper limit
    		 0.9

    Secondary: Time to First Response in NPS

    		 Close Top of page
    End point title
    		 Time to First Response in NPS
    End point description
    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. Analysis Population Description ITT population: all participants who were randomized and received at least 1 dose of study treatment. Participants with nasal polyps. '99999' indicates the value is not estimable due to an insufficient number of observed events.
    End point type
    Secondary
    End point timeframe
    up to Week 16
    End point values
    		 Placebo GB001
    Number of subjects analysed
    35
    32
    Units: weeks
        median (confidence interval 95%)
    16.43 (8.571 to 99999)
    16.14 (8.143 to 99999)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 v Placebo
    Number of subjects included in analysis
    67
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.8916
    Method
    		 Regression, Cox
    Parameter type
    		 Hazard ratio (HR)
    Point estimate
    0.944
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.41
         upper limit
    		 2.173

    Secondary: Change From Baseline to Week 16 in Morning (AM) Nasal Congestion (NC) Score

    		 Close Top of page
    End point title
    		 Change From Baseline to Week 16 in Morning (AM) Nasal Congestion (NC) Score
    End point description
    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). Analysis Population Description ITT population: all participants who were randomized and received at least 1 dose of study treatment. Participants with a baseline value.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 16
    End point values
    		 Placebo GB001
    Number of subjects analysed
    50
    46
    Units: score on a scale
        least squares mean (confidence interval 95%)
    -0.733 (-0.917 to -0.549)
    -0.543 (-0.738 to -0.347)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 v Placebo
    Number of subjects included in analysis
    96
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1635
    Method
    		 Mixed models analysis
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.191
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.077
         upper limit
    		 0.459

    Secondary: Change From Baseline to Week 16 in AM Total Symptom Score (TSS)

    		 Close Top of page
    End point title
    		 Change From Baseline to Week 16 in AM Total Symptom Score (TSS)
    End point description
    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). Analysis Population Description ITT population: all participants who were randomized and received at least 1 dose of study treatment. Participants with a baseline value.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 16
    End point values
    		 Placebo GB001
    Number of subjects analysed
    50
    46
    Units: score on a scale
        least squares mean (confidence interval 95%)
    -2.499 (-3.130 to -1.869)
    -1.867 (-2.526 to -1.209)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 v Placebo
    Number of subjects included in analysis
    96
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1742
    Method
    		 Mixed models analysis
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.632
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.28
         upper limit
    		 1.544

    Secondary: Change From Baseline to Week 16 in University of Pennsylvania Smell Identification Test (UPSIT) Score

    		 Close Top of page
    End point title
    		 Change From Baseline to Week 16 in University of Pennsylvania Smell Identification Test (UPSIT) Score
    End point description
    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. Analysis Population Description ITT population: all participants who were randomized and received at least 1 dose of study treatment. Participants with a baseline value.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 16
    End point values
    		 Placebo GB001
    Number of subjects analysed
    49
    47
    Units: score on a scale
        least squares mean (confidence interval 95%)
    1.5 (-0.3 to 3.2)
    2.3 (0.5 to 4.2)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 v Placebo
    Number of subjects included in analysis
    96
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.4851
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.9
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.6
         upper limit
    		 3.4

    Secondary: Time to First Chronic Rhinosinusitis (CRS) Exacerbation

    		 Close Top of page
    End point title
    		 Time to First Chronic Rhinosinusitis (CRS) Exacerbation
    End point description
    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. Analysis Population Description ITT population: all participants who were randomized and received at least 1 dose of study treatment. '-99999' and '99999' indicate the value is not estimable due to an insufficient number of observed events.
    End point type
    Secondary
    End point timeframe
    up to Week 16
    End point values
    		 Placebo GB001
    Number of subjects analysed
    50
    47
    Units: weeks
        median (confidence interval 95%)
    99999 (-99999 to 99999)
    99999 (-99999 to 99999)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 v Placebo
    Number of subjects included in analysis
    97
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.3898
    Method
    		 Regression, Cox
    Parameter type
    		 Hazard ratio (HR)
    Point estimate
    0.544
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.136
         upper limit
    		 2.178

    Secondary: Incidence of Treatment-Emergent Adverse Events (TEAEs)

    		 Close Top of page
    End point title
    		 Incidence of Treatment-Emergent Adverse Events (TEAEs)
    End point description
    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. Analysis Population Description Safety Population: all participants who received at least 1 dose of study treatment.
    End point type
    Secondary
    End point timeframe
    From first dose of study drug through Week 20
    End point values
    		 Placebo GB001
    Number of subjects analysed
    50
    47
    Units: percentage of participants
        number (not applicable)
    36.0
    44.7
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    From first dose of study drug through Week 20
    Adverse event reporting additional description
    [Not specified]
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 -

    Reporting group title
    GB001
    Reporting group description
    		 -

    Serious adverse events
    		 Placebo GB001
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 50 (0.00%)
    1 / 47 (2.13%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Reproductive system and breast disorders
    Endometriosis
         subjects affected / exposed
    0 / 50 (0.00%)
    1 / 47 (2.13%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Placebo GB001
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    10 / 50 (20.00%)
    12 / 47 (25.53%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 50 (6.00%)
    5 / 47 (10.64%)
         occurrences all number
    5
    5
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    2 / 50 (4.00%)
    3 / 47 (6.38%)
         occurrences all number
    2
    3
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    0 / 50 (0.00%)
    3 / 47 (6.38%)
         occurrences all number
    0
    3
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    3 / 50 (6.00%)
    0 / 47 (0.00%)
         occurrences all number
    3
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    3 / 50 (6.00%)
    2 / 47 (4.26%)
         occurrences all number
    3
    2

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Apr 2019
    Amendment 1 (v2.0.0)
    02 May 2019
    Amendment 2 (v2.1)
    28 Aug 2019
    Amendment 3 (v3.0)
    18 Feb 2020
    Amendment 4 (v4.0)
    16 Apr 2020
    Amendment 5 (v5.0)

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Fri May 03 14:15:38 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA