A Phase 2 interventional study of GB004 and Placebo in Ulcerative Colitis, sponsored by GB004, Inc.. Terminated at 77 sites in 9 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-08-01.
Sponsored by GB004, Inc. · Phase 2, Interventional, and Treatment
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.
1,530 studies on the registry are indexed under Colitis, Ulcerative; 407 are open to participants now.
This study's enrollment of 236 is above the median of 70 across 1,069 interventional studies indexed under Colitis, Ulcerative.
Browse Colitis, Ulcerative studies →This is the only study on the registry with GB004, Inc. as lead sponsor.
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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.
PCP Placebo for oral administration for 36 weeks
Drug: Placebo
PCP GB004 480 mg QD for oral administration for 36 weeks
Drug: GB004
PCP GB004 480 mg BID for oral administration for 36 weeks
Drug: GB004
OLE GB004 480 mg BID for oral administration for 24 weeks
Drug: GB004
oral tablet
oral tablet
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.
Time frame: 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.
Time frame: From first dose of OLE study treatment through OLE Week 28
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: At PCP Week 36
| Milestone | Placebo-Controlled Period (PCP) Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID | Open-Label Extension (OLE) GB004 480 mg BID |
|---|---|---|---|---|
| Started | 78 | 78 | 80 | 0 |
| Completed | 23 | 25 | 26 | 0 |
| Not completed | 55 | 53 | 54 | 0 |
| Withdrew: Lack of efficacy | 28 | 23 | 23 | 0 |
| Withdrew: Study terminated by sponsor | 15 | 13 | 16 | 0 |
| Withdrew: Withdrawal by subject | 8 | 6 | 6 | 0 |
| Withdrew: Adverse event | 1 | 7 | 7 | 0 |
| Withdrew: Other, not specified | 1 | 2 | 1 | 0 |
| Withdrew: Protocol violation | 1 | 0 | 1 | 0 |
| Withdrew: Lost to follow-up | 1 | 0 | 0 | 0 |
| Withdrew: Physician decision | 0 | 1 | 0 | 0 |
| Withdrew: Site terminated by sponsor | 0 | 1 | 0 | 0 |
| Milestone | Placebo-Controlled Period (PCP) Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID | Open-Label Extension (OLE) GB004 480 mg BID |
|---|---|---|---|---|
| Started | 0 | 0 | 0 | 130 |
| Completed | 0 | 0 | 0 | 39 |
| Not completed | 0 | 0 | 0 | 91 |
| Withdrew: Study terminated by sponsor | 0 | 0 | 0 | 68 |
| Withdrew: Withdrawal by subject | 0 | 0 | 0 | 10 |
| Withdrew: Lack of efficacy | 0 | 0 | 0 | 7 |
| Withdrew: Adverse event | 0 | 0 | 0 | 2 |
| Withdrew: Other, not specified | 0 | 0 | 0 | 2 |
| Withdrew: Death | 0 | 0 | 0 | 1 |
| Withdrew: Lost to follow-up | 0 | 0 | 0 | 1 |
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.
| percentage of participants | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID |
|---|---|---|---|
| Percentage of Participants With Clinical Remission at PCP Week 12 | 17.9 (11.0 to 27.9) | 15.4 (9.0 to 25.0) | 22.5 (14.7 to 32.8) |
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.
| percentage of participants | OLE GB004 480 mg BID |
|---|---|
| Percentage of Participants With a Treatment Emergent Adverse Event | 32.3 |
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.
| percentage of participants | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID |
|---|---|---|---|
| Percentage of Participants With Clinical Response at PCP Week 12 | 42.3 (32.0 to 53.4) | 34.6 (25.0 to 45.7) | 52.5 (41.7 to 63.1) |
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.
| percentage of participants | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID |
|---|---|---|---|
| Percentage of Participants With Histologic Remission at PCP Week 12 | 26.1 (17.2 to 37.5) | 26.5 (17.4 to 38.0) | 32.9 (23.0 to 44.5) |
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.
| percentage of participants | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 m BID |
|---|---|---|---|
| Percentage of Participants With Endoscopic Improvement at PCP Week 12 | 21.8 (14.1 to 32.2) | 23.1 (15.1 to 33.6) | 30.0 (21.1 to 40.8) |
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.
| percentage of participants | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID |
|---|---|---|---|
| Percentage of Participants With Mucosal Healing at PCP Week 12 | 17.4 (10.2 to 28.0) | 16.2 (9.3 to 26.7) | 20.0 (12.3 to 30.8) |
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.
| percentage of participants | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID |
|---|---|---|---|
| Percentage of Participants With Clinical Remission at PCP Week 36 | 14.3 (7.7 to 25.0) | 7.8 (3.4 to 17.0) | 12.9 (6.7 to 23.4) |
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.
| percentage of participants | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID |
|---|---|---|---|
| Percentage of Participants With Clinical Response at PCP Week 36 | 20.6 (12.5 to 32.2) | 23.4 (14.7 to 35.1) | 29.0 (19.2 to 41.3) |
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.
| percentage of participants | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID |
|---|---|---|---|
| Percentage of Participants With Histologic Remission at PCP Week 36 | 14.3 (7.1 to 26.7) | 8.3 (3.3 to 19.6) | 16.0 (8.3 to 28.5) |
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.
| percentage of participants | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID |
|---|---|---|---|
| Percentage of Participants With Endoscopic Improvement at PCP Week 36 | 15.9 (8.9 to 26.8) | 15.6 (8.7 to 26.4) | 12.9 (6.7 to 23.4) |
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.
| percentage of participants | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID |
|---|---|---|---|
| Percentage of Participants With Mucosal Healing at PCP Week 36 | 14.3 (7.1 to 26.7) | 6.3 (2.1 to 16.8) | 12.0 (5.6 to 23.8) |
Collected over 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. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| PCP Placebo | 0/78 (0%) | 2/78 (2.6%) | 13/78 (16.7%) |
| PCP GB004 480 mg QD | 0/76 (0%) | 2/76 (2.6%) | 29/76 (38.2%) |
| PCP GB004 480 mg BID | 0/82 (0%) | 4/82 (4.9%) | 27/82 (32.9%) |
| OLE GB004 480 mg BID | 1/130 (0.8%) | 9/130 (6.9%) | 8/130 (6.2%) |
| Event | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID | OLE GB004 480 mg BID |
|---|---|---|---|---|
| Colitis ulcerativeGastrointestinal disorders | 1/78 | 2/76 | 0/82 | 4/130 |
| AnaemiaBlood and lymphatic system disorders | 1/78 | 0/76 | 0/82 | 0/130 |
| CholecystitisHepatobiliary disorders | 0/78 | 0/76 | 1/82 | 0/130 |
| Anal abscessInfections and infestations | 0/78 | 0/76 | 1/82 | 1/130 |
| Prostate cancerNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/78 | 0/76 | 1/82 | 0/130 |
| Calculus urinaryRenal and urinary disorders | 0/78 | 0/76 | 1/82 | 0/130 |
| Atrial fibrillationCardiac disorders | 0/78 | 0/76 | 0/82 | 1/130 |
| COVID-19Infections and infestations | 0/78 | 0/76 | 0/82 | 1/130 |
| COVID-19 pneumoniaInfections and infestations | 0/78 | 0/76 | 0/82 | 1/130 |
| SyncopeNervous system disorders | 0/78 | 0/76 | 0/82 | 1/130 |
| Event | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID | OLE GB004 480 mg BID |
|---|---|---|---|---|
| DizzinessNervous system disorders | 1/78 | 14/76 | 8/82 | 0/130 |
| NauseaGastrointestinal disorders | 2/78 | 11/76 | 4/82 | 0/130 |
| SomnolenceNervous system disorders | 1/78 | 5/76 | 7/82 | 0/130 |
| AnaemiaBlood and lymphatic system disorders | 1/78 | 5/76 | 0/82 | 0/130 |
| COVID-19Infections and infestations | 5/78 | 4/76 | 4/82 | 8/130 |
| FatigueGeneral disorders | 1/78 | 1/76 | 5/82 | 0/130 |
| HeadacheNervous system disorders | 0/78 | 4/76 | 2/82 | 0/130 |
| Upper respiratory tract infectionInfections and infestations | 4/78 | 2/76 | 2/82 | 0/130 |
| Age, Continuous(Years) | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID | Total |
|---|---|---|---|---|
| Mean | 45.1 ± 14.86 | 44.3 ± 13.79 | 45.1 ± 16.76 | 44.8 ± 15.14 |
| Sex: Female, Male(Participants) | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID | Total |
|---|---|---|---|---|
| Female | 37 | 34 | 31 | 102 |
| Male | 41 | 44 | 49 | 134 |
| Race/Ethnicity, Customized(Participants) | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID | Total |
|---|---|---|---|---|
| Race — White | 77 | 74 | 79 | 230 |
| Race — Asian | 0 | 3 | 0 | 3 |
| Race — Black or African American | 1 | 0 | 1 | 2 |
| Race — Other | 0 | 1 | 0 | 1 |
| Race/Ethnicity, Customized(Participants) | PCP Placebo | PCP GB004 480 mg QD | PCP GB004 480 mg BID | Total |
|---|---|---|---|---|
| Ethnicity — Not Hispanic or Latino | 75 | 75 | 80 | 230 |
| Ethnicity — Hispanic or Latino | 3 | 3 | 0 | 6 |
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Plan to share: No
Ran from Oct 2020 to primary completion in Jun 2022, 19 months.
Results were first posted in Aug 2023, 1 year 2 months after primary completion.
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