A Study With GB004 in Adult Subjects With Active Ulcerative Colitis (UC)
- Registration Number
- NCT04556383
- Lead Sponsor
- GB004, Inc.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 236
-
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:
- prednisone ≤ 20 mg/day or equivalent or
- beclomethasone ≤ 5 mg/day or
- budesonide or budesonide multi-matrix (MMX) of ≤ 9 mg/day
- Prior approved biologic therapy used for the treatment of UC.
- Diagnosis of Crohn's disease, indeterminate colitis, or pouchitis, or presence of bacterial or parasitic infection.
- Tofacitinib, oral cyclosporine, sirolimus or mycophenolate mofetil within 8 weeks of Day 1.
- Azathioprine, or 6-mercaptopurine within 1 day of Day 1.
NOTE: Other Inclusion/Exclusion criteria may apply per protocol.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description PCP Placebo Placebo PCP Placebo for oral administration for 36 weeks PCP GB004 480 mg QD GB004 PCP GB004 480 mg QD for oral administration for 36 weeks PCP GB004 480 mg BID GB004 PCP GB004 480 mg BID for oral administration for 36 weeks Open-Label Extension (OLE) GB004 480 mg BID GB004 OLE GB004 480 mg BID for oral administration for 24 weeks
- Primary Outcome Measures
Name Time Method Percentage of Participants With Clinical Remission at PCP Week 12 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.Percentage of Participants With a Treatment Emergent Adverse Event From first dose of OLE study treatment through OLE Week 28 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.
- Secondary Outcome Measures
Name Time Method Percentage of Participants With Clinical Response at PCP Week 12 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.Percentage of Participants With Histologic Remission at PCP Week 12 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.Percentage of Participants With Endoscopic Improvement at PCP Week 12 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.Percentage of Participants With Clinical Response at PCP Week 36 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.Percentage of Participants With Histologic Remission at PCP Week 36 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.Percentage of Participants With Mucosal Healing at PCP Week 36 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.Percentage of Participants With Mucosal Healing at PCP Week 12 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.Percentage of Participants With Clinical Remission at PCP Week 36 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.Percentage of Participants With Endoscopic Improvement at PCP Week 36 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.
Trial Locations
- Locations (77)
CLINSANTE Clinical Research Center Civil Law Partnership Ewa Galczak-Nowak, Malgorzata Trzaska
🇵🇱Bydgoszcz, Poland
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
LTD Coloproctological Center of Georgia
🇬🇪Tbilisi, Georgia
PMSI Republican Clinical Hospital "Timofei Mosneaga"
🇲🇩Chisinau, Moldova, Republic of
Las Vegas Medical Research
🇺🇸Las Vegas, Nevada, United States
Texas Digestive Disease Consultant
🇺🇸Southlake, Texas, United States
Freehold Endoscopy Associates, LLC d/b/a/ Endoscopy Center of Monmouth County
🇺🇸Freehold, New Jersey, United States
Great Lakes Gastroenterology Research, LLC
🇺🇸Mentor, Ohio, United States
Washington Gastroenterology, PLLC
🇺🇸Bellevue, Washington, United States
Huron Gastroenterology Associates
🇺🇸Ypsilanti, Michigan, United States
JSC Infectious Diseases, AIDS and Clinical Immunology Research Center
🇬🇪Tbilisi, Georgia
Medicome Limited Liability Company, Oswiecim Clinical Trial Centre
🇵🇱Oswiecim, Poland
EB GROUP Limited Liability Company, MDM Health Centre
🇵🇱Warsaw, Poland
VIVAMED Non-Public Healthcare Facility
🇵🇱Warsaw, Poland
Limited Liability Company Joint Venture Diagnostic Center "Biotherm"
🇷🇺Barnaul, Russian Federation
Clinical Research Center Piotr Napora Medical Doctors Professional Partnership
🇵🇱Wroclaw, Poland
Institute of Cytology and Genetics, Siberian Branch of the Russian Academy of Sciences
🇷🇺Novosibirsk, Russian Federation
S.M. Kirov Miltiary Medical Academy
🇷🇺St Petersburg, Russian Federation
St. Petersburg State-Funded Healthcare Institution: City Outpatient Care Unit No. 38
🇷🇺St. Petersburg, Russian Federation
Regional State-Funded Healthcare Institution: Novgorod Regional Clinic Hospital
🇷🇺Veliky Novgorod, Russian Federation
Clinical Hospital Center "Dr Dragisa Misovic Dedinje'' local lab
🇷🇸Belgrade, Serbia
Clinical Hospital Center "Dr Dragisa Misovic Dedinje''
🇷🇸Belgrade, Serbia
Clinical Center Kragujevac
🇷🇸Kragujevac, Serbia
Communal Nonprofit Enterprise "Cherkasy Regional Hospital of Cherkasy Oblast Council"
🇺🇦Cherkasy, Ukraine
Clinical Hospital Center Zemun
🇷🇸Belgrade, Serbia
Zvezdara University Medical Center-local lab
🇷🇸Belgrade, Serbia
General Hospital "Djordje Joanovic"
🇷🇸Zrenjanin, Serbia
Regional Communal Noncommercial Enterprise "Chernivtsi Regional Clinical Hospital" Site 9519
🇺🇦Chernivtsi, Ukraine
Public Non-Profit Enterprise under Kyiv Regional Council "Kyiv Regional Hospital"
🇺🇦Kyiv, Ukraine
Communal Noncommercial Enterprise of Lviv Regional Council "Lviv Regional Clinical Hospital"
🇺🇦Lviv, Ukraine
PNPE "City Hospital of Urgent and Emergency Medical Care under Zaporizhia City Council"
🇺🇦Zaporizhia, Ukraine
Limited Liability Company "Medibor"
🇺🇦Zhytomyr, Ukraine
Gastro Care Institute
🇺🇸Lancaster, California, United States
B G Clinical Research, Inc.
🇺🇸Encinitas, California, United States
Texas Digestive Disease Consultants
🇺🇸Baton Rouge, Louisiana, United States
Washington Gastroenterology, PLCC
🇺🇸Tacoma, Washington, United States
Professor K. Gibinski University Clinical Centre of the Medical University of Silesia in Katowice
🇵🇱Katowice, Poland
"Gastromed" Torun Gastrology Centre [Torunskie Centrum Gastrologiczne "Gastromed"]
🇵🇱Toruń, Poland
"NOWE ZDROWIE-CK" Kieltucki and Partners General Partnership, NOWE ZDROWIE-CK
🇵🇱Staszow, Poland
WIP Warsaw IBD Point Profesor Kierkus
🇵🇱Warsaw, Poland
Marek Horynski, MD, Ph.D. Individual Specialist Medical Practice [Specjialistyczna Praktyka Lekarska Dr med. Marek Horynski]
🇵🇱Sopot, Poland
Reuma Park Clinic Limited Liability Company Limited Partnership, Reuma Park Medical Center
🇵🇱Warsaw, Poland
Federal Siberian Research Clinical Center under the Federal Medical Biological Agency
🇷🇺Krasnoyarsk, Russian Federation
"Myod" Ltd.
🇷🇺Bataysk, Russian Federation
Consultation and Diagnostics Center and Outpatient Care Unit under the Department of Presidential Affairs
🇷🇺St. Petersburg, Russian Federation
PNPE "Kyiv City Clinical Hospital #18" under the Executive Body of Kyiv City Council
🇺🇦Kyiv, Ukraine
Public Non-Profit Enterprise "Regional Clinical Hospital under Ivano-Frankivsk Regional Council"
🇺🇦Ivano-Frankivsk, Ukraine
Medical Center of the Limited Liability Company "Harmoniia Krasy"
🇺🇦Kyiv, Ukraine
Medical Center of the Limited Liability Company "Medical Center "CONSILIUM MEDICAL"
🇺🇦Kyiv, Ukraine
Medical Center "OK!Clinic+" of the Company with Limited Liability "International Institute of Clinical Research"
🇺🇦Kyiv, Ukraine
The Municipal Enterprise "Volyn Regional Clinical Hospital" of the Volyn Regional Council
🇺🇦Lutsk, Ukraine
Public Non-Profit Enterprise "Odesa Regional Clinical Hospital" under Odesa Regional Council
🇺🇦Odesa, Ukraine
Public Enterprise "Poltava M.V. Sklifosovsky Regional Clinical Hospital under Poltava Regional Council"
🇺🇦Poltava, Ukraine
Communal Non-Commercial Enterprise "Vinnytsia City Clinical Hospital #1
🇺🇦Vinnytsia, Ukraine
MNPE "Vinnytsia Regional Clinical Hospital named after M.I. Pirogov Vinnytsia Regional Council"
🇺🇦Vinnytsia, Ukraine
MNPE "Zaporizhia Regional Clinical Hospital" of Zaporizhia Regional Council
🇺🇦Zaporizhia, Ukraine
LTD Central University Clinic After Academic N. Kipshidze
🇬🇪Tbilisi, Georgia
Gastroenterology Clinic of Acadiana
🇺🇸Lafayette, Louisiana, United States
Inje University Heaundae Paik Hospital
🇰🇷Busan, Korea, Republic of
Regional Communal Noncommercial Enterprise "Chernivtsi Regional Clinical Hospital" Site 9527
🇺🇦Chernivtsi, Ukraine
LTD Aversi Clinic
🇬🇪Tbilisi, Georgia
Dong-A University Hospital
🇰🇷Busan, Korea, Republic of
Colentina Clinical Hospital
🇷🇴Bucharest, Romania
Medical Center SibNovoMed, Limited Liability Company
🇷🇺Novosibirsk, Russian Federation
PNPE "Prof. O.O. Shalimov City Clinical Hospital #2" under Kharkiv City Council
🇺🇦Kharkiv, Ukraine
Malkhaz Katsiashvili Multiprofile Emergency Medicine Center LTD
🇬🇪Tbilisi, Georgia
Kyungpook National University Chilgok Hospital
🇰🇷Daegu, Korea, Republic of
St. John Paul 2 Municipal Hospital in Elblag, Department of Internal Medicine
🇵🇱Elblag, Poland
"LANDA" Katarzyna Agata Landa, Landa" Specialist Doctor's Offices
🇵🇱Krakow, Poland
Penza Regional Clinical Hospital named after N.N. Burdenko
🇷🇺Penza, Russian Federation
Delta Research Partners
🇺🇸Monroe, Louisiana, United States
Clinical Research Center of Karkonosze - Lexmedica Limited Liability Company, KCBK - LEXMEDICA
🇵🇱Jelenia Gora, Poland
Medical Diagnostic Center, Limited Liability Company
🇷🇺Orenburg, Russian Federation
Novosibirskiy Gastrocenter, LLC
🇷🇺Novosibirsk, Russian Federation
Clinic UZI 4D, Limited Liability Company
🇷🇺Pyatigorsk, Russian Federation
Public Non-Profit Enterprise under Kharkiv Regional Council "Regional Clinical Hospital"
🇺🇦Kharkiv, Ukraine
Moscow State-Funded Healthcare Institution City Clinical Hospital n.a. V.M. Buyanov under Moscow Healthcare Department
🇷🇺Moscow, Russian Federation