E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Ulcerative colitis |
Colitis Ulcerosa |
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E.1.1.1 | Medical condition in easily understood language |
Ulcerative colitis |
Colitis Ulcerosa |
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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 | 16.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10045365 |
E.1.2 | Term | Ulcerative colitis |
E.1.2 | System Organ Class | 100000004856 |
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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 the efficacy and safety, and to assess the pharmacokinetics of adalimumab administered subcutaneously (SC) in pediatric subjects with moderate to severe ulcerative colitis (UC). |
Para demostrar la eficacia y seguridad, y estudiar la farmacocinética de Adalimumab administrado subcutáneamente (SC) en sujetos pediátricos con colitis ulcerosa (CU) de moderada a severa |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Pharmacogenetic testing |
Prueba farmacocinetica |
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E.3 | Principal inclusion criteria |
? Subjects from the ages of 4 to 17 prior to baseline dosing. ? Diagnosis of UC for at least 12 weeks prior to screening, confirmed by endoscopy with biopsy during the Screening Period ? Active ulcerative colitis with a Mayo Score of 6 ? 12 points and endoscopy subscore of 2 ? 3 despite concurrent treatment with oral corticosteroids or immunosuppressants or both |
?Sujetos de 4 a 17 años antes de la dosis basal ?Diagnóstico de CU 12 semanas antes de la selección, confirmado por endoscopia con biopsia durante el periodo de selección ?Colitis ulcerosa activa con un índice de Mayo de 6 a 12 puntos y una subpuntuación en la endoscopia de 2 a 3, a pesar de recibir tratamiento con corticoesteroides o inmunosupresores, o ambos |
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E.4 | Principal exclusion criteria |
? Subject with Crohn's disease (CD) or indeterminate colitis (IC). ? Current diagnosis of fulminant colitis and/or toxic megacolon. ? Subjects with disease limited to the rectum (ulcerative proctitis) during the screening endoscopy. ? Chronic recurring infections or active TB |
?Sujetos con enfermedad de Crohn o colitis indeterminada. ?Diagnóstico actual de colitis fulminante y/o megalocolon tóxico. ?Sujetos con enfermedad limitada al recto (proscitis ulcerativa) durante la endoscopia de selección. ?Infecciones recurrentes crónicas o tuberculosis activa. |
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E.5 End points |
E.5.1 | Primary end point(s) |
? The proportion of subjects who achieve clinical remission at Week 8 as measured by PMS ? The proportion of subjects who responded at Week 8 per PMS and achieve clinical remission at Week 52 as measured by Mayo score |
?Porcentaje de sujetos que logran la remisión clínica en semana 8 medido según la PMS ?Porcentaje de sujetos que responden en semana 8 según PMS, y presentan remisión clínica en semana 52 según la puntuación de Mayo |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Week 8, Week 52 |
Semana 8, Semana 52 |
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E.5.2 | Secondary end point(s) |
? Proportion of subjects who achieve mucosal healing at Week 52 as measured by Mayo endoscopy subscore (defined as ? 1) in Week 8 responders per PMS ? Proportion of subjects in PUCAI remission (defined as < 10) in Week 8 responders per PMS at Week 52 |
?Porcentaje de sujetos que logran una curación de la mucosa en semana 52, medida según la subpuntuación de Mayo en la endoscopia. ?Porcentaje de sujetos en remisión según PUCA (definido como <10) en sujetos que responden en semana 8 según la PMS en semana 52. |
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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 | 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) | 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 | 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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 30 |
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 |
Canada |
New Zealand |
Australia |
Israel |
Switzerland |
United States |
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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
|
The end of study is defined as the date of the Last subject last visit, or the last follow-up contact, whichever is longer. |
El final del estudio se define como la fecha de la última visita del último paciente, o la última visita de seguimiento, lo que sea más tardío. |
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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 | 4 |
E.8.9.1 | In the Member State concerned days | |
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 | 4 |