E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Active Ulcerative Colitis (UC) or Crohn's disease (CD) |
Colitis ulcerosa (CU) o enfermedad de Crohn (EC) activa |
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E.1.1.1 | Medical condition in easily understood language |
Active UC or CD. UC is a long-term condition that results in inflammation and ulcers of the colon and rectum. CD is a disease that causes inflammation of the digestive system. |
CU o EC activa. La CU es una enfermedad crónica que produce inflamación y úlceras en el colon y el recto. La EC es una enfermedad que causa inflamación del sistema digestivo. |
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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 | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of this study is to evaluate the safety profile of long-term vedolizumab IV treatment in pediatric subjects with UC or CD. |
El objetivo principal de este estudio consiste en evaluar el perfil de seguridad del tratamiento a largo plazo con vedolizumab IV en sujetos pediátricos con CU o EC. |
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E.2.2 | Secondary objectives of the trial |
• To evaluate the effect of long-term vedolizumab IV treatment on time to major inflammatory bowel disease (IBD)-related events (eg, hospitalizations, surgeries, and procedures) in pediatric subjects with UC or CD. • To evaluate quality of life in subjects aged 9 to 17 years who were treated with vedolizumab IV using the IMPACT-III questionnaire. |
• Evaluar el efecto del tratamiento a largo plazo con vedolizumab IV sobre el tiempo transcurrido hasta la aparición de episodios importantes relacionados con la enfermedad inflamatoria intestinal (EII) (por ejemplo, hospitalizaciones, intervenciones quirúrgicas y procedimientos) en sujetos pediátricos con CU o EC. • Evaluar la calidad de vida de sujetos de entre 9 y 17 años tratados con vedolizumab IV mediante el cuestionario IMPACT-III. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. In the opinion of the investigator, the subject, parent, or legal guardian is capable of understanding and complying with protocol requirements. 2. The subject, parent, or legal guardian signs and dates a written, informed consent form and any required privacy authorization before the initiation of any study procedures. 3. The subject is male or female with UC or CD and aged <18 years. (Note: Subjects who reach adult age [18 years of age in most jurisdictions/regions] during the study should be considered to transition to commercial drug if available in their country, especially if transition to an adult care setting is required by local regulation.). 4. The subject has completed Study MLN0002-3024 or Study MLN0002-3025 and has achieved corticosteroid-free clinical response at Week 54 (and has tapered off of steroids, as applicable, at least 12 weeks before Week 54) as defined by a reduction of partial Mayo score of ≥2 points and ≥25% from baseline for subjects with UC; or a reduction of PCDAI of ≥15 points for subjects with CD from baseline of the parent study. 5. A male subject who is sexually active with a female partner of childbearing potential agrees to use barrier method of contraception (eg, condom with or without spermicide) from signing of informed consent throughout the duration of the study and for 18 weeks after last dose. The female partner of a male subject should also be advised to use a highly effective method of contraception. 6. A female subject of childbearing potential who is sexually active with a nonsterilized male partner agrees to use a highly effective method of contraception from signing of informed consent throughout the duration of the study and 18 weeks after the last dose. |
1. En opinión del investigador, el sujeto, progenitor o tutor legal es capaz de entender y cumplir los requisitos del protocolo. 2. El sujeto, progenitor o tutor legal firma y fecha un documento de consentimiento informado por escrito y cualquier autorización necesaria en materia de privacidad antes del inicio de cualquiera de los procedimientos del estudio. 3. El sujeto es un varón o mujer con CU o EC y tiene una edad menor de 18 años. (Nota: En los sujetos que alcancen la edad adulta [18 años en la mayoría de las jurisdicciones o regiones] durante el estudio deberá contemplarse la transición al fármaco comercial, si está disponible en su país, especialmente si la normativa local exige la transición a un entorno asistencial para adultos). 4. El sujeto ha completado el estudio MLN0002-3024 o el estudio MLN0002-3025 y ha logrado una respuesta clínica sin corticosteroides en la semana 54 (y ha reducido progresivamente los corticosteroides, según proceda, al menos 12 semanas antes de la semana 54), definida como una reducción de la puntuación parcial de Mayo ≥2 puntos y ≥25% con respecto al momento basal en los sujetos con CU o una reducción de la puntuación PCDAI ≥15 puntos en los sujetos con EC con respecto al momento basal del estudio original. 5. Los varones que mantengan relaciones sexuales con una pareja con capacidad reproductiva deben comprometerse a utilizar un método anticonceptivo de barrera (por ejemplo, preservativo con o sin espermicida) a partir de la firma del consentimiento informado, durante la totalidad del estudio y hasta 18 semanas después de la última dosis. También debe aconsejarse a las parejas de los varones participantes que utilicen un método anticonceptivo altamente eficaz. 6. Las mujeres con capacidad reproductiva que mantengan relaciones sexuales con una pareja masculina no esterilizada deben comprometerse a utilizar un método anticonceptivo altamente eficaz a partir de la firma del consentimiento informado, durante la totalidad del estudio y hasta 18 semanas después de la última dosis. |
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E.4 | Principal exclusion criteria |
1. The subject is female and is lactating or pregnant. 2. The subject has hypersensitivity or allergies to any of the vedolizumab excipients. 3. The subject currently requires major surgical intervention for UC or CD (eg, bowel resection), or is anticipated to require major surgical intervention for UC or CD during the study. 4. The subject has developed any new unstable or uncontrolled cardiovascular, heart failure moderate to severe (New York Class Association III or IV), pulmonary, hepatic, renal, gastrointestinal, genitourinary, hematological, coagulation, immunological, endocrine/metabolic, neurological, or other medical disorder that, in the opinion of the investigator, would confound the study results or compromise subject safety. 5. The subject has other serious comorbidities that will limit their ability to complete the study. 6. The subject is unable to comply with all study assessments. |
1. El sujeto es una mujer en período de lactancia o embarazada. 2. El sujeto tiene hipersensibilidad o alergia a cualquiera de los excipientes de vedolizumab. 3. El sujeto requiere actualmente una intervención de cirugía mayor para la CU o EC (por ejemplo, resección intestinal) o se prevé que la necesitará durante el estudio. 4. El sujeto ha presentado insuficiencia cardíaca moderada o grave (clase III o IV de la New York Class Association) o cualquier trastorno cardiovascular, pulmonar, hepático, renal, digestivo, genitourinario, hematológico, de coagulación, inmunológico, endocrino/metabólico, neurológico o de otro tipo inestable o no controlado nuevo que, en opinión del investigador, podría confundir los resultados del estudio o comprometer la seguridad del sujeto. 5. El sujeto presenta otras enfermedades concomitantes graves que limitarán su capacidad para completar el estudio. 6. El sujeto no puede cumplir todas las evaluaciones del estudio. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint for this study is adverse events (AEs). |
El criterio de valoración principal de este estudio son los acontecimientos adversos (AA). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Once every 16 weeks until Week 48, thereafter once every 24 weeks. |
Cada 16 semanas hasta la semana 48 y cada 24 semanas a partir de entonces. |
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E.5.2 | Secondary end point(s) |
• Time to major IBD-related events (eg, hospitalizations, surgeries, or procedures). • Changes from baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III total and subscale scores for subjects aged 9 to 17 years as measured every 24 weeks. |
• Tiempo transcurrido hasta episodios importantes relacionados con la EII (por ejemplo, hospitalizaciones, intervenciones quirúrgicas o procedimientos). • Variaciones con respecto al momento basal del estudio MLN0002-3024 (CU) o MLN0002-3025 (EC) de las puntuaciones total y de subescalas del cuestionario IMPACT-III en los sujetos de entre 9 y 17 años, medidas cada 24 semanas. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Time to major IBD-related events - through-out duration of the study. Changes from baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III total and subscale scores for subjects aged 9 to 17 years as measured - every 24 weeks. |
Tiempo transcurrido hasta episodios importantes relacionados con la EII a lo largo del estudio. Variaciones con respecto al momento basal del estudio MLN0002-3024 (CU) o MLN0002-3025 (EC) de las puntuaciones total y de subescalas del cuestionario IMPACT-III en los sujetos de entre 9 y 17 años, medidas cada 24 semanas. |
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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 | Yes |
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 | 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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
Diferentes dosis de medicamento en investigación en los brazos de tratamiento |
Different doses of IMP are used in treatment arms |
|
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 | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 41 |
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 |
Canada |
China |
Israel |
Japan |
Korea, Republic of |
United States |
Lithuania |
Poland |
Spain |
Czechia |
Greece |
Italy |
Belgium |
Croatia |
Hungary |
Russian Federation |
Slovakia |
Ukraine |
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 | 7 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 7 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |