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) |
The achievement of 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. The achievement of endoscopic improvement at Week 12 2. The achievement of clinical remission per Full Mayo score (defined as a Full Mayo score ≤ 2 with no subscore > 1) at Week 12 in subjects with a Full Mayo score of 6 to 12 at Baseline 3. The achievement of clinical response per Adapted Mayo score at Week 12 4. The achievement of clinical response per Partial Adapted Mayo score at Week 4 5. The achievement of endoscopic remission at Week 12 6. Occurrence of subjects with hospitalizations through Week 12 7. The achievement of HEMR 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. UC-related surgeries through Week 12
Sub-study 2 1. The achievement of clinical response per Adapted Mayo score at Week 12 2. The achievement of endoscopic improvement at Week 12 3. The achievement of histologic endoscopic mucosal improvement (HEMI: Endoscopic subscore of 0 or 1 without evidence of friability and Geboes score ≤ 3.1) at Week 12 4. The achievement of endoscopic remission at Week 12 5. The achievement of clinical response per Partial Adapted Mayo score at Week 4 6. The achievement of no bowel urgency at Week 12 7. The achievement of no abdominal pain at Week 12 8. The achievement of histologic endoscopic mucosal remission (HEMR: Endoscopy subscore of 0 and Geboes score < 2.0) at Week 12 9. Change from Baseline to Week 12 in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) 10. Change from Baseline to Week 12 in Inflammatory Bowel Disease Questionnaire (IBDQ) total score 11. Occurrence of UC-related hospitalizations through Week 12 12. The achievement of no nocturnal bowel movements at Week 12 13. The achievement of no tenesmus at Week 12 14. Change from Baseline to Week 12 in number of fecal incontinence episodes per week 15. Change from Baseline to Week 12 in number of days per week with sleep interrupted due to UC symptoms.
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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 12 4. Week 12 5. Week 4 6. Week 12 7. Week 12 8. Week 12 9. Baseline, Week 12 10. Baseline, Week 12 11. Week 12 12. Week 12 13. Week 12 14. Baseline, Week 12 15. Baseline, 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 | 3 |
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 |
Malaysia |
Mexico |
New Zealand |
South Africa |
United States |
Switzerland |
Russian Federation |
Turkey |
Ukraine |
Serbia |
Austria |
Belgium |
Bulgaria |
Croatia |
Czechia |
Denmark |
France |
Germany |
Greece |
Italy |
Latvia |
Lithuania |
Netherlands |
Poland |
Portugal |
Romania |
Slovakia |
Slovenia |
Spain |
Sweden |
Korea, Republic of |
Taiwan |
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 | 4 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 18 |
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 |