Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2020-002306-12 | EudraCT Number |
Not provided
Not provided
Not provided
Study terminated due to lack of treatment benefit.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
A 2-part study, comprising of a 36-week placebo-controlled period (PCP) and a 24-week open-label extension (OLE) period, to assess the efficacy and safety of 2 dose regimens of GB004 when added to background UC therapy of 5-aminosalicylate (5-ASA) with or without systemic steroids.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PCP Placebo | Placebo Comparator | PCP Placebo for oral administration for 36 weeks |
|
| PCP GB004 480 mg QD | Experimental | PCP GB004 480 mg QD for oral administration for 36 weeks |
|
| PCP GB004 480 mg BID | Experimental | PCP GB004 480 mg BID for oral administration for 36 weeks |
|
| Open-Label Extension (OLE) GB004 480 mg BID | Experimental | OLE GB004 480 mg BID for oral administration for 24 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GB004 | Drug | oral tablet |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Clinical Remission at PCP Week 12 | Clinical remission is defined as a Modified Mayo score ≤ 2, with a rectal bleeding subscore of 0, stool frequency subscore of 0 or 1 (with a ≥ 1 point decrease from baseline), and endoscopic subscore of 0 or 1. The Modified Mayo score is an endpoint measure composed of: Stool frequency, Rectal bleeding, and Endoscopic subscores (where the Endoscopic subscore value of 1 does not include friability), each ranging from 0 to 3, that are summed to give a total score ranging from 0 to 9 points, with higher scores indicating greater severity. | At PCP Week 12 |
| Percentage of Participants With a Treatment Emergent Adverse Event | An adverse event (AE) is any untoward medical occurrence in a participant, whether or not considered related to study treatment. 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. An AE was considered treatment-emergent to the OLE if it started on or after the first dose of OLE study treatment. | From first dose of OLE study treatment through OLE Week 28 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Clinical Response at PCP Week 12 | Clinical response is defined as a reduction in Modified Mayo score of ≥ 2 points and ≥ 35% from baseline, including a decrease in rectal bleeding subscore of ≥ 1 or absolute rectal bleeding subscore of ≤ 1. The Modified Mayo score is an endpoint measure composed of: Stool frequency, Rectal bleeding, and Endoscopic subscores (where the Endoscopic subscore value of 1 does not include friability), each ranging from 0 to 3, that are summed to give a total score ranging from 0 to 9 points, with higher scores indicating greater severity. |
Not provided
Inclusion Criteria:
Adult male and female subjects aged ≥ 18 years at the time of signing the informed consent form (ICF) prior to initiation of any study specific activities/procedures.
UC diagnosed at least 3 months prior to first dose of investigational product (IP) on Day 1.
Currently receiving treatment for UC, on a stable dose for at least 2 weeks prior to flexible sigmoidoscopy or colonoscopy, with oral 5-ASA (eg, mesalamine, sulfasalazine) alone or with one of the following oral treatments:
Exclusion Criteria:
NOTE: Other Inclusion/Exclusion criteria may apply per protocol.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| B G Clinical Research, Inc. | Encinitas | California | 92024 | United States | ||
| Gastro Care Institute |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35014040 | Derived | Danese S, Levesque BG, Feagan BG, Jucov A, Bhandari BR, Pai RK, Taylor Meadows K, Kirby BJ, Bruey JM, Olson A, Osterhout R, Van Biene C, Ford J, Aranda R, Raghupathi K, Sandborn WJ. Randomised clinical trial: a phase 1b study of GB004, an oral HIF-1alpha stabiliser, for treatment of ulcerative colitis. Aliment Pharmacol Ther. 2022 Feb;55(4):401-411. doi: 10.1111/apt.16753. Epub 2022 Jan 10. |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Placebo-Controlled Period (PCP) Placebo | PCP Placebo for oral administration for 36 weeks |
| FG001 | PCP GB004 480 mg QD | PCP GB004 480 mg QD for oral administration for 36 weeks |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| PCP |
|
Not provided
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 15, 2021 | May 22, 2023 |
Not provided
Not provided
Not provided
Not provided
Subjects, investigators and site personnel, and the Sponsor will be blinded to individual subject treatment assignments.
| Placebo | Drug | oral tablet |
|
| At PCP Week 12 |
| Percentage of Participants With Histologic Remission at PCP Week 12 | Histologic remission is defined as Robarts Histopathology Index (RHI) ≤ 3 with lamina propria neutrophils RHI subscore = 0 and neutrophils in epithelium RHI subscore = 0. Histologic remission is evaluated among subjects with both baseline lamina propria neutrophils and neutrophils in epithelium RHI subscores > 0. The RHI is a validated instrument that measures histologic disease activity and consists of 4 subscores (chronic inflammatory infiltrate, lamina propria neutrophils, neutrophils in epithelium, and erosion or ulceration). Each subscore ranges from 0-3, with higher subscores indicating greater histologic disease activity. The RHI score is calculated as: (1 x chronic inflammatory infiltrate) + (2 x lamina propria neutrophils) + (3 x neutrophils in epithelium) + (5 x erosion or ulceration). The RHI therefore ranges from 0-33, with higher scores indicating greater histologic disease activity. | At PCP Week 12 |
| Percentage of Participants With Endoscopic Improvement at PCP Week 12 | Endoscopic improvement is defined as an endoscopic subscore of 0 or 1. The endoscopic subscore is a component of the Modified Mayo score and is assessed on a 0-3 scale: 0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern); 2 = Moderate disease (marked erythema, absent vascular pattern, friability, erosions); and 3 = Severe disease (spontaneous bleeding, ulceration). Higher scores indicate greater endoscopic disease severity. An endoscopic subscore of 1 does not include friability; an endoscopy with friability is assessed an endoscopic subscore of at least 2. | At PCP Week 12 |
| Percentage of Participants With Mucosal Healing at PCP Week 12 | Mucosal healing is defined as endoscopic improvement and histologic remission. Please see Secondary Outcome Measure Descriptions above for Percentage of Participants With Endoscopic Improvement at PCP Week 12 and for Percentage of Participants With Histologic Remission at PCP Week 12 for information on the measures of endoscopic improvement and histologic remission, respectively. | At PCP Week 12 |
| Percentage of Participants With Clinical Remission at PCP Week 36 | Clinical remission is defined as a Modified Mayo score ≤ 2, with a rectal bleeding subscore of 0, stool frequency subscore of 0 or 1 (with a ≥ 1 point decrease from baseline), and endoscopic subscore of 0 or 1. The Modified Mayo score is an endpoint measure composed of: Stool frequency, Rectal bleeding, and Endoscopic subscores (where the Endoscopic subscore value of 1 does not include friability), each ranging from 0 to 3, that are summed to give a total score ranging from 0 to 9 points, with higher scores indicating greater severity. | At PCP Week 36 |
| Percentage of Participants With Clinical Response at PCP Week 36 | Clinical response is defined as a reduction in Modified Mayo score of ≥ 2 points and ≥ 35% from baseline, including a decrease in rectal bleeding subscore of ≥ 1 or absolute rectal bleeding subscore of ≤ 1. The Modified Mayo score is an endpoint measure composed of: Stool frequency, Rectal bleeding, and Endoscopic subscores (where the Endoscopic subscore value of 1 does not include friability), each ranging from 0 to 3, that are summed to give a total score ranging from 0 to 9 points, with higher scores indicating greater severity. | At PCP Week 36 |
| Percentage of Participants With Histologic Remission at PCP Week 36 | Histologic remission is defined as Robarts Histopathology Index (RHI) ≤ 3 with lamina propria neutrophils RHI subscore = 0 and neutrophils in epithelium RHI subscore = 0. The RHI is a validated instrument that measures histologic disease activity and consists of 4 subscores (chronic inflammatory infiltrate, lamina propria neutrophils, neutrophils in epithelium, and erosion or ulceration). Each subscore ranges from 0-3, with higher subscores indicating greater histologic disease activity. The RHI score is calculated as: (1 x chronic inflammatory infiltrate) + (2 x lamina propria neutrophils) + (3 x neutrophils in epithelium) + (5 x erosion or ulceration). The RHI therefore ranges from 0-33, with higher scores indicating greater histologic disease activity. | At PCP Week 36 |
| Percentage of Participants With Endoscopic Improvement at PCP Week 36 | Endoscopic improvement is defined as an endoscopic subscore of 0 or 1. The endoscopic subscore is a component of the Modified Mayo score and is assessed on a 0-3 scale: 0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern); 2 = Moderate disease (marked erythema, absent vascular pattern, friability, erosions); and 3 = Severe disease (spontaneous bleeding, ulceration). Higher scores indicate greater endoscopic disease severity. An endoscopic subscore of 1 does not include friability; an endoscopy with friability is assessed an endoscopic subscore of at least 2. | At PCP Week 36 |
| Percentage of Participants With Mucosal Healing at PCP Week 36 | Mucosal healing is defined as endoscopic improvement and histologic remission. Please see Secondary Outcome Measure Descriptions above for Percentage of Participants With Endoscopic Improvement at PCP Week 36 and for Percentage of Participants With Histologic Remission at PCP Week 36 for information on the measures of endoscopic improvement and histologic remission, respectively. | At PCP Week 36 |
| Lancaster |
| California |
| 93534 |
| United States |
| Texas Digestive Disease Consultants | Baton Rouge | Louisiana | 70809 | United States |
| Gastroenterology Clinic of Acadiana | Lafayette | Louisiana | 70503 | United States |
| Delta Research Partners | Monroe | Louisiana | 71201 | United States |
| Huron Gastroenterology Associates | Ypsilanti | Michigan | 48197 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| Las Vegas Medical Research | Las Vegas | Nevada | 89113 | United States |
| Freehold Endoscopy Associates, LLC d/b/a/ Endoscopy Center of Monmouth County | Freehold | New Jersey | 07728 | United States |
| Great Lakes Gastroenterology Research, LLC | Mentor | Ohio | 44060 | United States |
| Texas Digestive Disease Consultant | Southlake | Texas | 76092 | United States |
| Washington Gastroenterology, PLLC | Bellevue | Washington | 98004 | United States |
| Washington Gastroenterology, PLCC | Tacoma | Washington | 98405 | United States |
| JSC Infectious Diseases, AIDS and Clinical Immunology Research Center | Tbilisi | 0160 | Georgia |
| LTD Aversi Clinic | Tbilisi | 0160 | Georgia |
| LTD Central University Clinic After Academic N. Kipshidze | Tbilisi | 0160 | Georgia |
| LTD Coloproctological Center of Georgia | Tbilisi | 0160 | Georgia |
| Malkhaz Katsiashvili Multiprofile Emergency Medicine Center LTD | Tbilisi | 0172 | Georgia |
| PMSI Republican Clinical Hospital "Timofei Mosneaga" | Chisinau | MD2025 | Moldova |
| CLINSANTE Clinical Research Center Civil Law Partnership Ewa Galczak-Nowak, Malgorzata Trzaska | Bydgoszcz | 85-794 | Poland |
| St. John Paul 2 Municipal Hospital in Elblag, Department of Internal Medicine | Elblag | 82-300 | Poland |
| Clinical Research Center of Karkonosze - Lexmedica Limited Liability Company, KCBK - LEXMEDICA | Jelenia Góra | 58-500 | Poland |
| Professor K. Gibinski University Clinical Centre of the Medical University of Silesia in Katowice | Katowice | 40-752 | Poland |
| "LANDA" Katarzyna Agata Landa, Landa" Specialist Doctor's Offices | Krakow | 31-156 | Poland |
| Medicome Limited Liability Company, Oswiecim Clinical Trial Centre | Oświęcim | 32-600 | Poland |
| Marek Horynski, MD, Ph.D. Individual Specialist Medical Practice [Specjialistyczna Praktyka Lekarska Dr med. Marek Horynski] | Sopot | 81-756 | Poland |
| "NOWE ZDROWIE-CK" Kieltucki and Partners General Partnership, NOWE ZDROWIE-CK | Staszów | 28-200 | Poland |
| "Gastromed" Torun Gastrology Centre [Torunskie Centrum Gastrologiczne "Gastromed"] | Torun | 87-100 | Poland |
| EB GROUP Limited Liability Company, MDM Health Centre | Warsaw | 00-635 | Poland |
| WIP Warsaw IBD Point Profesor Kierkus | Warsaw | 00-728 | Poland |
| Reuma Park Clinic Limited Liability Company Limited Partnership, Reuma Park Medical Center | Warsaw | 02-665 | Poland |
| VIVAMED Non-Public Healthcare Facility | Warsaw | 03-580 | Poland |
| Clinical Research Center Piotr Napora Medical Doctors Professional Partnership | Wroclaw | 51-162 | Poland |
| Colentina Clinical Hospital | Bucharest | 020125 | Romania |
| Limited Liability Company Joint Venture Diagnostic Center "Biotherm" | Barnaul | 656015 | Russia |
| "Myod" Ltd. | Bataysk | 346880 | Russia |
| Federal Siberian Research Clinical Center under the Federal Medical Biological Agency | Krasnoyarsk | 660037 | Russia |
| Moscow State-Funded Healthcare Institution City Clinical Hospital n.a. V.M. Buyanov under Moscow Healthcare Department | Moscow | 115516 | Russia |
| Medical Center SibNovoMed, Limited Liability Company | Novosibirsk | 630005 | Russia |
| Novosibirskiy Gastrocenter, LLC | Novosibirsk | 630007 | Russia |
| Institute of Cytology and Genetics, Siberian Branch of the Russian Academy of Sciences | Novosibirsk | 630089 | Russia |
| Medical Diagnostic Center, Limited Liability Company | Orenburg | 460051 | Russia |
| Penza Regional Clinical Hospital named after N.N. Burdenko | Penza | 440026 | Russia |
| Clinic UZI 4D, Limited Liability Company | Pyatigorsk | 357502 | Russia |
| S.M. Kirov Miltiary Medical Academy | Saint Petersburg | 191015 | Russia |
| St. Petersburg State-Funded Healthcare Institution: City Outpatient Care Unit No. 38 | Saint Petersburg | 191015 | Russia |
| Consultation and Diagnostics Center and Outpatient Care Unit under the Department of Presidential Affairs | Saint Petersburg | 197110 | Russia |
| Regional State-Funded Healthcare Institution: Novgorod Regional Clinic Hospital | Veliky Novgorod | 173008 | Russia |
| Clinical Hospital Center "Dr Dragisa Misovic Dedinje'' local lab | Belgrade | 11000 | Serbia |
| Clinical Hospital Center "Dr Dragisa Misovic Dedinje'' | Belgrade | 11000 | Serbia |
| Clinical Hospital Center Zemun | Belgrade | 11000 | Serbia |
| Zvezdara University Medical Center-local lab | Belgrade | 11000 | Serbia |
| Clinical Center Kragujevac | Kragujevac | 34000 | Serbia |
| General Hospital "Djordje Joanovic" | Zrenjanin | 23000 | Serbia |
| Inje University Heaundae Paik Hospital | Busan | 48018 | South Korea |
| Dong-A University Hospital | Busan | 49201 | South Korea |
| Kyungpook National University Chilgok Hospital | Daegu | 41404 | South Korea |
| Communal Nonprofit Enterprise "Cherkasy Regional Hospital of Cherkasy Oblast Council" | Cherkasy | 18009 | Ukraine |
| Regional Communal Noncommercial Enterprise "Chernivtsi Regional Clinical Hospital" Site 9519 | Chernivtsi | 58001 | Ukraine |
| Regional Communal Noncommercial Enterprise "Chernivtsi Regional Clinical Hospital" Site 9527 | Chernivtsi | 58001 | Ukraine |
| Public Non-Profit Enterprise "Regional Clinical Hospital under Ivano-Frankivsk Regional Council" | Ivano-Frankivsk | 76008 | Ukraine |
| PNPE "Prof. O.O. Shalimov City Clinical Hospital #2" under Kharkiv City Council | Kharkiv | 61037 | Ukraine |
| Public Non-Profit Enterprise under Kharkiv Regional Council "Regional Clinical Hospital" | Kharkiv | 61058 | Ukraine |
| PNPE "Kyiv City Clinical Hospital #18" under the Executive Body of Kyiv City Council | Kyiv | 01030 | Ukraine |
| Medical Center of the Limited Liability Company "Harmoniia Krasy" | Kyiv | 01135 | Ukraine |
| Medical Center "OK!Clinic+" of the Company with Limited Liability "International Institute of Clinical Research" | Kyiv | 02091 | Ukraine |
| Medical Center of the Limited Liability Company "Medical Center "CONSILIUM MEDICAL" | Kyiv | 04050 | Ukraine |
| Public Non-Profit Enterprise under Kyiv Regional Council "Kyiv Regional Hospital" | Kyiv | 04078 | Ukraine |
| The Municipal Enterprise "Volyn Regional Clinical Hospital" of the Volyn Regional Council | Lutsk | 43005 | Ukraine |
| Communal Noncommercial Enterprise of Lviv Regional Council "Lviv Regional Clinical Hospital" | Lviv | 79010 | Ukraine |
| Public Non-Profit Enterprise "Odesa Regional Clinical Hospital" under Odesa Regional Council | Odesa | 65025 | Ukraine |
| Public Enterprise "Poltava M.V. Sklifosovsky Regional Clinical Hospital under Poltava Regional Council" | Poltava | 36011 | Ukraine |
| MNPE "Vinnytsia Regional Clinical Hospital named after M.I. Pirogov Vinnytsia Regional Council" | Vinnytsia | 21028 | Ukraine |
| Communal Non-Commercial Enterprise "Vinnytsia City Clinical Hospital #1 | Vinnytsia | 21029 | Ukraine |
| PNPE "City Hospital of Urgent and Emergency Medical Care under Zaporizhia City Council" | Zaporizhia | 69005 | Ukraine |
| MNPE "Zaporizhia Regional Clinical Hospital" of Zaporizhia Regional Council | Zaporizhia | 69600 | Ukraine |
| Limited Liability Company "Medibor" | Zhytomyr | 10002 | Ukraine |
| FG002 | PCP GB004 480 mg BID | PCP GB004 480 mg BID for oral administration for 36 weeks |
| FG003 | Open-Label Extension (OLE) GB004 480 mg BID | OLE GB004 480 mg BID for oral administration for 24 weeks |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| OLE |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | PCP Placebo | PCP Placebo for oral administration for 36 weeks |
| BG001 | PCP GB004 480 mg QD | PCP GB004 480 mg QD for oral administration for 36 weeks |
| BG002 | PCP GB004 480 mg BID | PCP GB004 480 mg BID for oral administration for 36 weeks |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants | No |
| |||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants | No |
| |||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants | No |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants With Clinical Remission at PCP Week 12 | Clinical remission is defined as a Modified Mayo score ≤ 2, with a rectal bleeding subscore of 0, stool frequency subscore of 0 or 1 (with a ≥ 1 point decrease from baseline), and endoscopic subscore of 0 or 1. The Modified Mayo score is an endpoint measure composed of: Stool frequency, Rectal bleeding, and Endoscopic subscores (where the Endoscopic subscore value of 1 does not include friability), each ranging from 0 to 3, that are summed to give a total score ranging from 0 to 9 points, with higher scores indicating greater severity. | PCP Intent to Treat (ITT) Population: all participants who were randomized and received at least 1 dose of PCP study treatment | Posted | Number | 95% Confidence Interval | percentage of participants | At PCP Week 12 |
|
|
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Primary | Percentage of Participants With a Treatment Emergent Adverse Event | An adverse event (AE) is any untoward medical occurrence in a participant, whether or not considered related to study treatment. 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. An AE was considered treatment-emergent to the OLE if it started on or after the first dose of OLE study treatment. | OLE Safety Population: all participants who received at least 1 dose of OLE study treatment | Posted | Number | percentage of participants | From first dose of OLE study treatment through OLE Week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Clinical Response at PCP Week 12 | Clinical response is defined as a reduction in Modified Mayo score of ≥ 2 points and ≥ 35% from baseline, including a decrease in rectal bleeding subscore of ≥ 1 or absolute rectal bleeding subscore of ≤ 1. The Modified Mayo score is an endpoint measure composed of: Stool frequency, Rectal bleeding, and Endoscopic subscores (where the Endoscopic subscore value of 1 does not include friability), each ranging from 0 to 3, that are summed to give a total score ranging from 0 to 9 points, with higher scores indicating greater severity. | PCP ITT Population: all participants who were randomized and received at least 1 dose of PCP study treatment | Posted | Number | 95% Confidence Interval | percentage of participants | At PCP Week 12 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Histologic Remission at PCP Week 12 | Histologic remission is defined as Robarts Histopathology Index (RHI) ≤ 3 with lamina propria neutrophils RHI subscore = 0 and neutrophils in epithelium RHI subscore = 0. Histologic remission is evaluated among subjects with both baseline lamina propria neutrophils and neutrophils in epithelium RHI subscores > 0. The RHI is a validated instrument that measures histologic disease activity and consists of 4 subscores (chronic inflammatory infiltrate, lamina propria neutrophils, neutrophils in epithelium, and erosion or ulceration). Each subscore ranges from 0-3, with higher subscores indicating greater histologic disease activity. The RHI score is calculated as: (1 x chronic inflammatory infiltrate) + (2 x lamina propria neutrophils) + (3 x neutrophils in epithelium) + (5 x erosion or ulceration). The RHI therefore ranges from 0-33, with higher scores indicating greater histologic disease activity. | Participants in the PCP ITT Population (all participants who were randomized and received at least 1 dose of PCP study treatment) with baseline lamina propria neutrophils and neutrophils in the epithelium RHI subscores > 0. | Posted | Number | 95% Confidence Interval | percentage of participants | At PCP Week 12 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Endoscopic Improvement at PCP Week 12 | Endoscopic improvement is defined as an endoscopic subscore of 0 or 1. The endoscopic subscore is a component of the Modified Mayo score and is assessed on a 0-3 scale: 0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern); 2 = Moderate disease (marked erythema, absent vascular pattern, friability, erosions); and 3 = Severe disease (spontaneous bleeding, ulceration). Higher scores indicate greater endoscopic disease severity. An endoscopic subscore of 1 does not include friability; an endoscopy with friability is assessed an endoscopic subscore of at least 2. | PCP Intent to Treat (ITT) Population: all participants who were randomized and received at least 1 dose of PCP study treatment | Posted | Number | 95% Confidence Interval | percentage of participants | At PCP Week 12 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Mucosal Healing at PCP Week 12 | Mucosal healing is defined as endoscopic improvement and histologic remission. Please see Secondary Outcome Measure Descriptions above for Percentage of Participants With Endoscopic Improvement at PCP Week 12 and for Percentage of Participants With Histologic Remission at PCP Week 12 for information on the measures of endoscopic improvement and histologic remission, respectively. | Participants in the PCP ITT Population (all participants who were randomized and received at least 1 dose of PCP study treatment) with baseline lamina propria neutrophils and neutrophils in the epithelium RHI subscores > 0. | Posted | Number | 95% Confidence Interval | percentage of participants | At PCP Week 12 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Clinical Remission at PCP Week 36 | Clinical remission is defined as a Modified Mayo score ≤ 2, with a rectal bleeding subscore of 0, stool frequency subscore of 0 or 1 (with a ≥ 1 point decrease from baseline), and endoscopic subscore of 0 or 1. The Modified Mayo score is an endpoint measure composed of: Stool frequency, Rectal bleeding, and Endoscopic subscores (where the Endoscopic subscore value of 1 does not include friability), each ranging from 0 to 3, that are summed to give a total score ranging from 0 to 9 points, with higher scores indicating greater severity. | PCP ITT Population: all participants who were randomized and received at least 1 dose of PCP study treatment. Participants who did not consent to protocol version 2.0 and participants who withdrew from PCP due to study termination by Sponsor are excluded from the analysis. | Posted | Number | 95% Confidence Interval | percentage of participants | At PCP Week 36 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Clinical Response at PCP Week 36 | Clinical response is defined as a reduction in Modified Mayo score of ≥ 2 points and ≥ 35% from baseline, including a decrease in rectal bleeding subscore of ≥ 1 or absolute rectal bleeding subscore of ≤ 1. The Modified Mayo score is an endpoint measure composed of: Stool frequency, Rectal bleeding, and Endoscopic subscores (where the Endoscopic subscore value of 1 does not include friability), each ranging from 0 to 3, that are summed to give a total score ranging from 0 to 9 points, with higher scores indicating greater severity. | PCP ITT Population: all participants who were randomized and received at least 1 dose of PCP study treatment. Participants who did not consent to protocol version 2.0 and participants who withdrew from PCP due to study termination by Sponsor are excluded from the analysis. | Posted | Number | 95% Confidence Interval | percentage of participants | At PCP Week 36 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Histologic Remission at PCP Week 36 | Histologic remission is defined as Robarts Histopathology Index (RHI) ≤ 3 with lamina propria neutrophils RHI subscore = 0 and neutrophils in epithelium RHI subscore = 0. The RHI is a validated instrument that measures histologic disease activity and consists of 4 subscores (chronic inflammatory infiltrate, lamina propria neutrophils, neutrophils in epithelium, and erosion or ulceration). Each subscore ranges from 0-3, with higher subscores indicating greater histologic disease activity. The RHI score is calculated as: (1 x chronic inflammatory infiltrate) + (2 x lamina propria neutrophils) + (3 x neutrophils in epithelium) + (5 x erosion or ulceration). The RHI therefore ranges from 0-33, with higher scores indicating greater histologic disease activity. | Participants in the PCP ITT Population (all participants who were randomized and received at least 1 dose of PCP study treatment) with baseline lamina propria neutrophils and neutrophils in the epithelium RHI subscores > 0. Participants who did not consent to protocol version 2.0, participants who withdrew from PCP due to study termination by Sponsor, and participants for whom biopsies were collected and not centrally read due to study termination by Sponsor are excluded from the analysis. | Posted | Number | 95% Confidence Interval | percentage of participants | At PCP Week 36 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Endoscopic Improvement at PCP Week 36 | Endoscopic improvement is defined as an endoscopic subscore of 0 or 1. The endoscopic subscore is a component of the Modified Mayo score and is assessed on a 0-3 scale: 0 = Normal or inactive disease; 1 = Mild disease (erythema, decreased vascular pattern); 2 = Moderate disease (marked erythema, absent vascular pattern, friability, erosions); and 3 = Severe disease (spontaneous bleeding, ulceration). Higher scores indicate greater endoscopic disease severity. An endoscopic subscore of 1 does not include friability; an endoscopy with friability is assessed an endoscopic subscore of at least 2. | PCP ITT Population: all participants who were randomized and received at least 1 dose of PCP study treatment. Subjects who did not consent to protocol version 2.0 and subjects who withdrew from PCP due to study termination by Sponsor are excluded from the analysis. | Posted | Number | 95% Confidence Interval | percentage of participants | At PCP Week 36 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Mucosal Healing at PCP Week 36 | Mucosal healing is defined as endoscopic improvement and histologic remission. Please see Secondary Outcome Measure Descriptions above for Percentage of Participants With Endoscopic Improvement at PCP Week 36 and for Percentage of Participants With Histologic Remission at PCP Week 36 for information on the measures of endoscopic improvement and histologic remission, respectively. | Participants in the PCP ITT Population (all participants who were randomized and received at least 1 dose of PCP study treatment) with baseline lamina propria neutrophils and neutrophils in the epithelium RHI subscores > 0. Participants who did not consent to protocol version 2.0, participants who withdrew from PCP due to study termination by Sponsor, and participants for whom biopsies were collected and not centrally read due to study termination by Sponsor are excluded from the analysis. | Posted | Number | 95% Confidence Interval | percentage of participants | At PCP Week 36 |
|
PCP: From first dose in PCP through first dose in OLE (if participant was dosed in OLE) or through PCP Week 40 (if participant was not dosed in OLE) OLE: From first dose in OLE through OLE Week 28
Number of participants at risk based on PCP safety population (all participants who received at least 1 dose of PCP study treatment, grouped according to highest dose of GB004 received) for PCP Reporting Groups and OLE safety population (all participants who received at least 1 dose of OLE study treatment) for OLE Reporting Group. Differences between PCP ITT and safety populations due to 2 participants randomized to GB004 480 mg QD but erroneously received and dosed GB004 480 mg BID.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | PCP Placebo | PCP Placebo for oral administration for 36 weeks | 0 | 78 | 2 | 78 | 13 | 78 |
| EG001 | PCP GB004 480 mg QD | PCP GB004 480 mg QD for oral administration for 36 weeks | 0 | 76 | 2 | 76 | 29 | 76 |
| EG002 | PCP GB004 480 mg BID | PCP GB004 480 mg BID for oral administration for 36 weeks | 0 | 82 | 4 | 82 | 27 | 82 |
| EG003 | OLE GB004 480 mg BID | OLE GB004 480 mg BID for oral administration for 24 weeks | 1 | 130 | 9 | 130 | 8 | 130 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Colitis ulcerative | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Anal abscess | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Prostate cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Calculus urinary | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dizziness | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| GB004, Inc. Study Director | GB004, Inc., a wholly owned subsidiary of Gossamer Bio, Inc. | 1-866-668-4083 | ClinicalTrials@gossamerbio.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 11, 2022 | May 23, 2023 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D003093 | Colitis, Ulcerative |
| D015212 | Inflammatory Bowel Diseases |
| ID | Term |
|---|---|
| D003092 | Colitis |
| D005759 | Gastroenteritis |
| D005767 | Gastrointestinal Diseases |
| D004066 | Digestive System Diseases |
| D003108 | Colonic Diseases |
| D007410 | Intestinal Diseases |
Not provided
Not provided
| Withdrawal by Subject |
|
| Lack of Efficacy |
|
| Adverse Event |
|
| Other, Not Specified |
|
| Death |
|
| Lost to Follow-up |
|
| Male |
|
| Asian |
|
| Black or African American |
|
| Other |
|
| Hispanic or Latino |
|
| 0.4719 |
| Risk Difference (RD) |
| 4.6 |
| 2-Sided |
| 95 |
| -9.0 |
| 17.8 |
PCP GB004 480 mg BID vs Placebo. |
| Superiority |
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
| OG002 | PCP GB004 480 mg BID | PCP GB004 480 mg BID for oral administration for 36 weeks |
|
|
|
|
|
|
| Units | Counts |
|---|
| Participants |
|
|
|
|
|
|
|
|
|
PCP GB004 480 mg QD for oral administration for 36 weeks |
| OG002 | PCP GB004 480 mg BID | PCP GB004 480 mg BID for oral administration for 36 weeks |
|
|
|
PCP GB004 480 mg BID for oral administration for 36 weeks
|
|
|
PCP GB004 480 mg BID for oral administration for 36 weeks
|
|
|