E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Mild-to-moderate active ulcerative colitis |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10009900 |
E.1.2 | Term | Colitis ulcerative |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the effect of GB004 compared to placebo on clinical remission at PCP Week 12 |
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E.2.2 | Secondary objectives of the trial |
o To evaluate the effect of GB004 on clinical response, histologic remission, endoscopic improvement and mucosal healing at PCP Week 12 o To evaluate the effect of GB004 on clinical remission, clinical response, histologic remission, endoscopic improvement, and mucosal healing at PCP Week 36 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
o 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. o UC diagnosed at least 3 months prior to first dose of investigational product (IP) on Day 1. o 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 treatments: a. prednisone ≤ 20 mg/day or equivalent or b. beclomethasone ≤ 5 mg/day or c. budesonide or budesonide multi-matrix (MMX) of ≤ 9 mg/day |
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E.4 | Principal exclusion criteria |
o Prior approved biologic therapy used for the treatment of UC. o Evidence of Crohn's disease, indeterminate colitis, or presence of bacterial or parasitic infection. o Tofacitinib, oral cyclosporine, sirolimus or myocophenolate mofetil within 8 weeks of Day 1. o Azathioprine, or 6-mercaptopurine within 1 day of Day 1. o Diagnosis of Crohn's disease or indeterminate colitis, pouchitis.
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E.5 End points |
E.5.1 | Primary end point(s) |
Proportion of subjects with clinical remission at PCP Week 12, 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 |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1. Proportion of subjects with clinical response at PCP Week 12, defined as reduction in the Modified Mayo score of ≥ 2 points and ≥ 35 percent reduction from baseline, including a decrease in Rectal bleeding subscore of ≥1 or absolute Rectal bleeding subscore of ≤ 1 2. Proportion of subjects with histologic remission at PCP Week 12, defined as Robarts Histopathology Index (RHI) ≤ 3 with lamina propria neutrophils subscore = 0 and neutrophils in epithelium subscore = 0 3. Proportion of subjects with endoscopic improvement at PCP Week 12, defined as endoscopic subscore of 0 or 1 4. Proportion of subjects with mucosal healing at PCP Week 12, defined as endoscopic improvement and histologic remission 5. Proportion of subjects with clinical remission at PCP Week 36 6. Proportion of subjects with clinical response at PCP Week 36 7. Proportion of subjects with histologic remission at PCP Week 36 8. Proportion of subjects with endoscopic improvement at PCP Week 36 9. Proportion of subjects with mucosal healing at PCP Week 36 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 3 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 15 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 22 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Belarus |
Georgia |
Korea, Republic of |
Moldova, Republic of |
Russian Federation |
Serbia |
Ukraine |
United States |
Italy |
Poland |
Romania |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 21 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 22 |