E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic antibiotic refractory and relapsing pouchitis |
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E.1.1.1 | Medical condition in easily understood language |
Chronic antibiotic refractory and relapsing inflammation of the pouch |
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E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Percentage of subjects achieving clinically relevant steroid-free remission |
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E.2.2 | Secondary objectives of the trial |
• Percentage of subjects achieving partial response • Time to clinically relevant steroid-free remission • Change in mPDAI endoscopic subscore • Change in mPDAI symptomatic subscore • Change in total mPDAI score • Change in European Quality of Life 5 Dimensions (EQ-5D)
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• The subject is male or female and aged 18 to 80 years inclusive • The subject has a history of ileal pouch-anal anastomosis (IPAA) for ulcerative colitis (UC) • The subject has pouchitis that is (a) relapsing or (b) chronic antibiotic refractory, defined by an mPDAI score ≥5 assessed as the average from 3 days immediately prior to the baseline endoscopy visit and a minimum endoscopic subscore of 2 (outside the staple or suture line) with either (a) ≥3 recurrent episodes within the last year, each treated with ≥2 weeks of antibiotic or other prescription therapy, or (b) requiring maintenance antibiotic therapy taken continuously for ≥4 weeks immediately prior to the baseline endoscopy visit
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E.4 | Principal exclusion criteria |
• Crohn’s disease (CD), CD-related complications of the pouch (pouch fistula, pouch strictures, ulcerations in the pre-pouch ileum without pouchitis), irritable pouch syndrome (IPS), isolated or predominant cuffitis, infectiouw pouchitis, diverting ostomy or mechanical complications of the pouch • Previous treatment with an anti-IL12/23 or an anti-IL23 antibody • Any investigational or approved biologic agent within 30 days of baseline • Nonbiologic investigational therapy or tofacitinib within 30 days prior to baseline • Active or untreated latent tuberculosis (TB) • Chronic hepatitis B virus (HBV) infection, chronic hepatitis C virus (HCV) infection, a known history of human immunodeficiency virus (HIV) infection (or is found to be seropositive at screening) or subject is immunodeficient • Active severe infection (e.g. sepsis, cytomegalovirus, listeriosis or C. difficile) • History of malignancy or current malignancy |
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E.5 End points |
E.5.1 | Primary end point(s) |
The percentage of subjects achieving clinically relevant steroid-free remission (mPDAI score <5 and a reduction by ≥2 points from baseline) after 16 weeks of treatment |
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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) |
• The percentage of subjects achieving clinically relevant steroid-free remission (mPDAI score <5 and a reduction by ≥2 points from baseline) after 48 weeks of treatment • The percentage of subjects achieving partial response (reduction of mPDAI score by ≥2 points from baseline) after 16 weeks of treatment • The percentage of subjects achieving partial response (reduction of mPDAI score by ≥2 points from baseline) after 48 weeks of treatment • Time to clinically relevant remission • Change in mPDAI endoscopic subscore at Week 16 and 48 compared to baseline • Change in mPDAI symptomatic subscore at Week 16 and 48 compared to baseline • Change in total mPDAI score at Week 16 and 48 compared to baseline • Change in European Quality of Life 5 Dimensions (EQ-5D) at Week 16, 32 and 48 compared to baseline
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
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) | No |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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 | No |
E.8.2.3 | Other | No |
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 | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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 | |
E.8.9.1 | In the Member State concerned days | |