Trial Outcomes & Findings for GB001 in Adult Participants With Chronic Rhinosinusitis (NCT NCT03956862)

NCT ID: NCT03956862

Last Updated: 2021-08-23

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 97 participants
• Primary outcome timeframe: Baseline, Week 16
• Results posted on: 2021-08-23

Participant Flow

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.

Participant milestones

Measure	Placebo Placebo once per day (QD) for 16 weeks	GB001 GB001 40 mg QD for 16 weeks
Overall Study STARTED	50	47
Overall Study COMPLETED	48	45
Overall Study NOT COMPLETED	2	2

Reasons for withdrawal

Measure	Placebo Placebo once per day (QD) for 16 weeks	GB001 GB001 40 mg QD for 16 weeks
Overall Study Lost to Follow-up	1	1
Overall Study Withdrawal by Subject	1	1

Baseline Characteristics

GB001 in Adult Participants With Chronic Rhinosinusitis

Baseline characteristics by cohort

Measure	Placebo n=50 Participants Placebo QD for 16 weeks	GB001 n=47 Participants GB001 40 mg QD for 16 weeks	Total n=97 Participants Total of all reporting groups
Age, Continuous	51.6 years STANDARD_DEVIATION 10.34 • n=5 Participants	51.7 years STANDARD_DEVIATION 11.81 • n=7 Participants	51.7 years STANDARD_DEVIATION 11.02 • n=5 Participants
Sex: Female, Male Female	21 Participants n=5 Participants	28 Participants n=7 Participants	49 Participants n=5 Participants
Sex: Female, Male Male	29 Participants n=5 Participants	19 Participants n=7 Participants	48 Participants n=5 Participants
Race/Ethnicity, Customized White	47 Participants n=5 Participants	41 Participants n=7 Participants	88 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	3 Participants n=5 Participants	5 Participants n=7 Participants	8 Participants n=5 Participants
Race/Ethnicity, Customized Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	46 Participants n=5 Participants	46 Participants n=7 Participants	92 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	4 Participants n=5 Participants	1 Participants n=7 Participants	5 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 16

Population: 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.

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.

Outcome measures

Measure	Placebo n=49 Participants Placebo QD for 16 weeks	GB001 n=45 Participants GB001 40 mg QD for 16 weeks
Change From Baseline to Week 16 in Sino-Nasal Outcome Test (SNOT) 22 Total Score	-18.1 score on a scale Interval -23.1 to -13.0	-18.3 score on a scale Interval -23.5 to -13.1

SECONDARY outcome

Timeframe: Baseline, Week 16

Population: ITT population: all participants who were randomized and received at least 1 dose of study treatment.

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.

Outcome measures

Measure	Placebo n=50 Participants Placebo QD for 16 weeks	GB001 n=47 Participants GB001 40 mg QD for 16 weeks
Change From Baseline to Week 16 in Lund-Mackay Score	-0.6 score on a scale Interval -1.6 to 0.4	-0.9 score on a scale Interval -2.0 to 0.1

SECONDARY outcome

Timeframe: Baseline, Week 16

Population: ITT population: all participants who were randomized and received at least 1 dose of study treatment. Participants with nasal polyps with a baseline value.

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.

Outcome measures

Measure	Placebo n=35 Participants Placebo QD for 16 weeks	GB001 n=30 Participants GB001 40 mg QD for 16 weeks
Change From Baseline to Week 16 in Nasal Polyp Score (NPS)	-0.8 score on a scale Interval -1.4 to -0.3	-0.7 score on a scale Interval -1.3 to -0.1

SECONDARY outcome

Timeframe: up to Week 16

Population: ITT population: all participants who were randomized and received at least 1 dose of study treatment. Participants with nasal polyps.

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.

Outcome measures

Measure	Placebo n=35 Participants Placebo QD for 16 weeks	GB001 n=32 Participants GB001 40 mg QD for 16 weeks
Time to First Response in NPS	16.43 weeks Interval 8.571 to NA (Not Available) indicates the value is not estimable due to an insufficient number of observed events.	16.14 weeks Interval 8.143 to NA (Not Available) indicates the value is not estimable due to an insufficient number of observed events.

SECONDARY outcome

Timeframe: Baseline, Week 16

Population: ITT population: all participants who were randomized and received at least 1 dose of study treatment. Participants with a baseline value.

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).

Outcome measures

Measure	Placebo n=50 Participants Placebo QD for 16 weeks	GB001 n=46 Participants GB001 40 mg QD for 16 weeks
Change From Baseline to Week 16 in Morning (AM) Nasal Congestion (NC) Score	-0.733 score on a scale Interval -0.917 to -0.549	-0.543 score on a scale Interval -0.738 to -0.347

SECONDARY outcome

Timeframe: Baseline, Week 16

Population: ITT population: all participants who were randomized and received at least 1 dose of study treatment. Participants with a baseline value.

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).

Outcome measures

Measure	Placebo n=50 Participants Placebo QD for 16 weeks	GB001 n=46 Participants GB001 40 mg QD for 16 weeks
Change From Baseline to Week 16 in AM Total Symptom Score (TSS)	-2.499 score on a scale Interval -3.13 to -1.869	-1.867 score on a scale Interval -2.526 to -1.209

SECONDARY outcome

Timeframe: Baseline, Week 16

Population: ITT population: all participants who were randomized and received at least 1 dose of study treatment. Participants with a baseline value.

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.

Outcome measures

Measure	Placebo n=49 Participants Placebo QD for 16 weeks	GB001 n=47 Participants GB001 40 mg QD for 16 weeks
Change From Baseline to Week 16 in University of Pennsylvania Smell Identification Test (UPSIT) Score	1.5 score on a scale Interval -0.3 to 3.2	2.3 score on a scale Interval 0.5 to 4.2

SECONDARY outcome

Timeframe: up to Week 16

Population: ITT population: all participants who were randomized and received at least 1 dose of study treatment.

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.

Outcome measures

Measure	Placebo n=50 Participants Placebo QD for 16 weeks	GB001 n=47 Participants GB001 40 mg QD for 16 weeks
Time to First Chronic Rhinosinusitis (CRS) Exacerbation	NA weeks NA (Not Available) indicates the value is not estimable due to an insufficient number of observed events.	NA weeks NA (Not Available) indicates the value is not estimable due to an insufficient number of observed events.

SECONDARY outcome

Timeframe: From first dose of study drug through Week 20

Population: Safety Population: all participants who received at least 1 dose of study treatment.

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.

Outcome measures

Measure	Placebo n=50 Participants Placebo QD for 16 weeks	GB001 n=47 Participants GB001 40 mg QD for 16 weeks
Percentage of Participants With a Treatment-Emergent Adverse Event (AE)	36.0 percentage of participants	44.7 percentage of participants

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

GB001

Serious events: 1 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Measure	Placebo n=50 participants at risk Placebo QD for 16 weeks	GB001 n=47 participants at risk GB001 40 mg QD for 16 weeks
Reproductive system and breast disorders Endometriosis	0.00% 0/50 • From first dose of study drug through Week 20	2.1% 1/47 • From first dose of study drug through Week 20

Other adverse events

Measure	Placebo n=50 participants at risk Placebo QD for 16 weeks	GB001 n=47 participants at risk GB001 40 mg QD for 16 weeks
Infections and infestations Nasopharyngitis	6.0% 3/50 • From first dose of study drug through Week 20	4.3% 2/47 • From first dose of study drug through Week 20
Nervous system disorders Headache	6.0% 3/50 • From first dose of study drug through Week 20	10.6% 5/47 • From first dose of study drug through Week 20
Respiratory, thoracic and mediastinal disorders Asthma	4.0% 2/50 • From first dose of study drug through Week 20	6.4% 3/47 • From first dose of study drug through Week 20
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/50 • From first dose of study drug through Week 20	6.4% 3/47 • From first dose of study drug through Week 20
Musculoskeletal and connective tissue disorders Arthralgia	6.0% 3/50 • From first dose of study drug through Week 20	0.00% 0/47 • From first dose of study drug through Week 20

Additional Information

GB001, Inc. Study Director

GB001, Inc., a wholly owned subsidiary of Gossamer Bio, Inc.

Phone: 1-866-668-4083

Email: ClinicalTrials@gossamerbio.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place