E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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 |
Study M16-067 comprises two sub-studies: The objective of Sub-Study 1 are to characterize the efficacy, safety, and pharmacokinetics of risankizumab as induction treatment in subjects with moderately to severely active ulcerative colitis (UC) and to identify the appropriate induction dose of risankizumab for further evaluation in Sub-Study 2. The objective of Sub-Study 2 is to evaluate the efficacy and safety of risankizumab compared to placebo in inducing clinical remission in subjects with moderately to severely active UC. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Male or female aged >=18 to <= 80 years, or minimum age of adult consent according to local regulations at the Baseline Visit. In addition for sub-study 2 only: Where locally permissible, subjects 16 to < 18 years of age who meet the definition of Tanner stage 5 for development at the Baseline Visit - Confirmed diagnosis of ulcerative colitis (UC) for at least 3 months prior to Baseline. - Active UC. - Demonstrated intolerance or inadequate response to one or more of the following categories of drugs: aminosalicylates, oral locally acting steroids, systemic steroids, immunomodulators, and/or biologic therapies |
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E.4 | Principal exclusion criteria |
- Subject with a current diagnosis of Crohn's disease (CD), inflammatory bowel disease-unclassified (IBD-U) or a history of radiation or ischemic colitis. - Subject receiving prohibited medications and treatment. - Extent of inflammatory disease limited to the rectum as assessed by screening endoscopy. - Subject with currently known complications of UC. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Proportion of subjects with clinical remission per Adapted Mayo score at Week 12. |
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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) |
Sub-study 1 1. Proportion of subjects with endoscopic improvement at Week 12. 2. Proportion of subjects achieving clinical remission at Week 12 in subjects with a Full Mayo score of 6 to 12 at Baseline. 3. Proportion of subjects achieving clinical response at Week 12. 4. Proportion of subjects achieving clinical response at Week 4. 5. Proportion of subjects with endoscopic remission at Week 12. 6. Proportion of subjects with hospitalizations through Week 12. 7. Proportion of subjects with mucosal healing at Week 12. 8. Change from Baseline in UC-Symptom Questionnaire (UC-SQ) at Week 12. 9. Change from Baseline in Inflammatory Bowel Disease Questionnaire (IBDQ) at Week 12. 10. Change from Baseline in Short Form-36 at Week 12. 11. Change from Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACITFatigue) at Week 12. 12. Proportion of subjects with UC-related surgeries through Week
Sub-study 2 1. Proportion of subjects achieving clinical remission per Full Mayo score at Week 12 in subjects with a Full Mayo score of 6 to 12 at Baseline 2. Proportion of subjects achieving clinical response per Adapted Mayo score at Week 12 3. Proportion of subjects achieving clinical response per Partial Adapted Mayo score at Week 4 4. Proportion of subjects who reported no abdominal pain at Week 12 5. Proportion of subjects who reported no bowel urgency at Week 12 6. Proportion of subjects with endoscopic remission at Week 12 7. Proportion of subjects with endoscopic improvement at Week 12 8. Change from Baseline to Week 12 in Inflammatory Bowel Disease Questionnaire (IBDQ) total score 9. Change from Baseline to Week 12 in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) 10. Proportion of subjects with histological endoscopic improvement of the mucosa at Week 12 11. Proportion of subjects who reported no nocturnal bowel movements at Week 12 12. Proportion of subjects who reported no tenesmus at Week 12 13. Change from Baseline to Week 12 in number of fecal incontinence episodes per week 14. Change from Baseline to Week 12 in number of days per week with sleep interrupted due to UC symptoms 15. Proportion of subjects achieving clinical response per Adapted Mayo score at Week 12 in subjects with pancolitis at Baseline 16. Proportion of subjects with UC-related hospitalizations through Week 12 17. Change from Baseline to Week 12 in Short Form-36 (SF-36)
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Sub-study 1 1. Week 12 2. Baseline, Week 12 3. Week 12 4. Week 4 5. Week 12 6. Week 12 7. Week 12 8. Baseline, Week 12 9. Baseline, Week 12 10. Baseline, Week 12 11. Baseline, Week 12 12. 12 weeks
Sub-study 2 1. Week 12 2. Week 12 3. Week 4 4. Week 12 5. Week 12 6. Week 12 7. Week 12 8. Week 12 9. Week 12 10. Week 12 11. Week 12 12. Week 12 13. Week 12 14. Week 12 15. Week 12 16. Week 12 17. Week 12
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
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) | Yes |
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 | 4 |
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 | 7 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 138 |
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 |
Argentina |
Brazil |
Canada |
Chile |
China |
Colombia |
Egypt |
Israel |
Japan |
Korea, Republic of |
Malaysia |
Mexico |
New Zealand |
Russian Federation |
Serbia |
South Africa |
Taiwan |
Turkey |
Ukraine |
United States |
Austria |
Belgium |
Bulgaria |
Croatia |
Czechia |
Denmark |
France |
Germany |
Greece |
Italy |
Latvia |
Lithuania |
Netherlands |
Poland |
Portugal |
Romania |
Slovakia |
Slovenia |
Spain |
Sweden |
Switzerland |
United Kingdom |
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E.8.7 | Trial has a data monitoring committee | Yes |
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 | 1 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | 7 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 18 |