E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Fibrostenotic Crohn’s Disease |
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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 | 10011401 |
E.1.2 | Term | Crohn's disease |
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 demonstrate that spesolimab is effective in maintaining Symptomatic Stenosis Response and/or inducing Radiographic Stenosis Response in patients with symptomatic CD-related small bowel stenosis, who have achieved Symptomatic Stenosis Response after standard medical therapy. |
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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 and female patients, 18 to 75 years when signing informed consent at screening
- Established diagnosis of clinical CD prior to screening
- Suspicion of symptomatic small bowel stenosis
- Presence of abdominal pain after eating or limitation in amount or types of food at screening
- 1 or 2 naïve or anastomotic stenoses in the terminal ileum at screening, confirmed by MRE at randomization
- Have achieved Symptomatic Stenosis Response before randomization (i.e. 7 day average scores <2 for diary questions on the abdominal pain
after eating AND on the limitation in amount or types of food)
- Endoscopic activity defined by Colonic Simple Endoscopic Score in CD (SES-CD) ≤12 after Lead-in Period, at the time of randomization |
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E.4 | Principal exclusion criteria |
- No stenosis in reach of ileocolonoscopy
- Systemic corticosteroid treatment of current obstructive symptoms for >1 week prior to screening
- Endoscopic balloon dilation (EBD) or surgical treatment of the same small bowel stenosis within the last 6 months prior to screening Visit 1
- More than 2 smal intestinal stenoses
- Patients who require immediate EBD or surgical intervention as per the investigator´s discretion
- Failure of >2 different biological drug classes prior to screening (e.g. TNF inhibitors, Integrin Receptor antagonists and IL-12 / IL-23 antagonists)
- Current complications of CD at screening Visit 1 and randomization (Day 1) that would possibly confound the evaluation of benefit from treatment with spesolimab
- Current stenosis in the colon
- Previous strictureplasty on current stricture
- Current ileostomy or colostomy
- Any kind of bowel resection or diversion within 6 months of screening Visit 1
- Any other intra-abdominal surgery (except for abscess drainage) within 3 months prior to screening Visit
- Colorectal cancer (CRC) present and past (<5 years) history |
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E.5 End points |
E.5.1 | Primary end point(s) |
1) Proportion of patients with maintained Symptomatic Stenosis Response
2) Proportion of patients with Radiographic Stenosis Response |
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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 patients with maintained Symptomatic Stenosis Response
2) Proportion of patients with Radiographic Stenosis Response
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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 | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | Yes |
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 | 2 |
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 | 70 |
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 |
Hong Kong |
Mexico |
Serbia |
Austria |
Belgium |
Brazil |
Canada |
China |
Denmark |
Germany |
Hungary |
Ireland |
Israel |
Italy |
Japan |
Korea, Republic of |
Netherlands |
Norway |
Poland |
Portugal |
Romania |
Spain |
Sweden |
Switzerland |
Taiwan |
United Kingdom |
United States |
France |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | 3 |
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 | 9 |
E.8.9.2 | In all countries concerned by the trial days | 3 |