| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
| Moderately to Severely Active Crohn’s Disease |
| Enfermedad de Crohn moderada a gravemente activa |
|
| E.1.1.1 | Medical condition in easily understood language |
| Moderately to Severely Active Crohn’s Disease |
| Enfermedad de Crohn moderada a gravemente activa |
|
| 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 |
|
| 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 evaluate the efficacy and safety of guselkumab in pediatric participants with CD at the end of maintenance therapy among participants who were in clinical response to guselkumab at Week 12 |
| Evaluar la eficacia y la seguridad de guselkumab en participantes pediátricos con EC al final de la terapia de mantenimiento entre los participantes que respondieron clínicamente a guselkumab en la semana 12 |
|
| E.2.2 | Secondary objectives of the trial |
1. To evaluate the clinical efficacy of guselkumab in pediatric participants with CD
2. To evaluate the efficacy of treatment with guselkumab in clinical remission by PRO at Week 12 and/or Week 52
3. To evaluate the PK and immunogenicity of guselkumab in pediatric participants with CD
4. To assess the impact of guselkumab therapy on growth
5. To evaluate the safety of guselkumab in pediatric participants with CD
6. To evaluate the efficacy of treatment with guselkumab in participants who are assigned at Week 12 to q4w maintenance therapy and do not receive non investigational product (IP) rescue therapy |
1. Evaluar la eficacia clínica de guselkumab en participantes pediátricos con EC
2. Evaluar la eficacia del tratamiento con guselkumab en remisión clínica por PRO en la Semana 12 y/o Semana 52
3. Evaluar la farmacocinética y la inmunogenicidad de guselkumab en participantes pediátricos con EC
4. Evaluar el impacto del tratamiento con guselkumab sobre el crecimiento
5. Evaluar la seguridad de guselkumab en participantes pediátricos con EC
6. Evaluar la eficacia del tratamiento con guselkumab en participantes asignados en la semana 12 a terapia de mantenimiento cada 4 semanas y que no reciben terapia de rescate con productos que no están en investigación (IP). |
|
| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
1. 2 to <18 years of age, inclusive (at the time of consent for screening).
2. Medically stable based on physical examination, medical history, and vital signs performed at screening. Medically stable based on clinical laboratory tests performed at screening.
3. Have a diagnosis of CD or fistulizing CD, with active colitis, ileitis, or ileocolitis, confirmed at any time in the past by clinical, endoscopic, and histologic criteria. Diagnosis based on prior surgical resection and histology is also acceptable. Radiographic findings may provide supportive evidence.
4. Have moderately to severely active CD (as defined by a screening PCDAI score >30).
5. Have endoscopy with evidence of active CD defined as SES-CD score ≥6 (or ≥4 for participants with isolated ileal disease) within 4 weeks of receiving study intervention at Week 0.
Please refer to the protocol for more exclusion criteria |
1. 2 a <18 años de edad, inclusive (en el momento del consentimiento para la detección).
2. Médicamente estable según el examen físico, el historial médico y los signos vitales realizados en la selección. Médicamente estable según las pruebas de laboratorio clínico realizadas en la selección.
3. Tener un diagnóstico de EC o CD fistulizante, con colitis activa, ileítis o ileocolitis, confirmado en cualquier momento en el pasado por criterios clínicos, endoscópicos e histológicos. También es aceptable el diagnóstico basado en la resección quirúrgica previa y la histología. Los hallazgos radiográficos pueden proporcionar evidencia de apoyo.
4. Tener EC activa de moderada a grave (según lo definido por una puntuación PCDAI de detección> 30).
5. Tener una endoscopia con evidencia de EC activa definida como puntuación SES-CD ≥6 (o ≥4 para participantes con enfermedad ileal aislada) dentro de las 4 semanas posteriores a recibir la intervención del estudio en la semana 0.
Consulte el protocolo para conocer más criterios de exclusión. |
|
| E.4 | Principal exclusion criteria |
1. Has complications of CD such as symptomatic strictures or stenosis, short gut syndrome, or any other manifestation that might be anticipated to require surgery, that could preclude the use of the PCDAI to assess response to therapy or would possibly confound the ability to assess the effect of the treatment. Of note, surgical procedures related to fistula treatment are not necessarily exclusionary; discuss with medical monitor.
2. Currently has or is suspected to have an abscess. Recent cutaneous and perianal abscesses are not exclusionary if drained and adequately treated at least 3 weeks prior to Week 0, or 8 weeks prior to Week 0 for intra-abdominal abscesses, provided that there is no anticipated need for any further surgery.
3. Has had any kind of bowel resection within 26 weeks or any other intra-abdominal surgery within 12 weeks of baseline.
4. Presence of a stoma, ileoanal pouch, or ostomy.
5. Has high grade dysplasia, history of or current evidence of polypoid or non-polypoid dysplasia, or any adenoma that has not been removed.
Please refer to the protocol for more exclusion criteria |
1. Tiene complicaciones de la EC, como estenosis o estenosis sintomáticas, síndrome del intestino corto o cualquier otra manifestación que pueda anticiparse que requiera cirugía, que podría impedir el uso del PCDAI para evaluar la respuesta a la terapia o posiblemente confundiría la capacidad de evaluar el efecto del tratamiento. Cabe destacar que los procedimientos quirúrgicos relacionados con el tratamiento de la fístula no son necesariamente excluyentes; discutir con el monitor médico.
2. Actualmente tiene o se sospecha que tiene un absceso. Los abscesos cutáneos y perianales recientes no son excluyentes si se drenaron y trataron adecuadamente al menos 3 semanas antes de la semana 0 u 8 semanas antes de la semana 0 para los abscesos intraabdominales, siempre que no haya necesidad anticipada de cirugía adicional.
3. Ha tenido algún tipo de resección intestinal dentro de las 26 semanas o cualquier otra cirugía intraabdominal dentro de las 12 semanas anteriores.
4. Presencia de estoma, bolsa ileoanal u ostomía.
5. Tiene displasia de alto grado, antecedentes o evidencia actual de displasia polipoide o no polipoide, o cualquier adenoma que no se haya extirpado.
Consulte el protocolo para conocer más criterios de exclusión. |
|
| E.5 End points |
| E.5.1 | Primary end point(s) |
1. Clinical remission (defined as PCDAI score ≤10)
2. Endoscopic response (≥50% reduction from SES-CD score at baseline) |
1. Remisión clínica (definida como puntuación PCDAI ≤10)
2. Respuesta endoscópica (≥50 % de reducción de la puntuación SES-CD al inicio) |
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. At Week 52
2. At Week 52 |
1. En la semana 52
2. En la semana 52 |
|
| E.5.2 | Secondary end point(s) |
1. Clinical response (decrease from baseline/LOR reference in the PCDAI score ≥12.5; total score ≤30)
2. Clinical response (PCDAI)
3. Clinical remission (PCDAI)
4. Endoscopic response (SES-CD)
5. Endoscopic remission (SES-CD)
6.Corticosteroid-free clinical remission (defined as PCDAI score ≤10 at
Week 52 and not receiving corticosteroids for at least 90 days before Week 52)
7. Sustained clinical remission (defined as PCDAI ≤10)
8. Serum guselkumab concentration during induction
9. Serum guselkumab concentration during maintenance (at least Ctrough)
10. Change from baseline in:
-Weight
-Weight percentiles and z-scores
-Height
-Height percentiles and z-scores
-Height Velocity
11. AEs, including SAEs |
1. Respuesta clínica (disminución desde el valor inicial/referencia LOR en la puntuación PCDAI ≥12,5; puntuación total ≤30)
2. Respuesta clínica (PCDAI)
3. Remisión clínica (PCDAI)
4. Respuesta endoscópica (SES-CD)
5. Remisión endoscópica (SES-CD)
6. Remisión clínica sin corticosteroides (definida como puntuación PCDAI ≤10 al
semana 52 y no recibir corticosteroides durante al menos 90 días antes de la semana 52)
7. Remisión clínica sostenida (definida como PCDAI ≤10)
8. Concentración sérica de guselkumab durante la inducción
9. Concentración sérica de guselkumab durante el mantenimiento (al menos Cvalle)
10. Cambio desde la línea de base en:
-Peso
-Percentiles de peso y puntuaciones z
-Altura
-Percentiles de altura y puntuaciones z
-Altura Velocidad
11. EA, incluidos SAE |
|
| E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. At Week 12
2. At Week 52
3. At Week 12
4. At Week 12
5. At Week 52
6. At Week 52
7. At Weeks 12, 24, and 52
8. From Week 0 through Week 12
9. N/A
10. At Week 12, 24 and 52
11. N/A |
1. En la semana 12
2. En la semana 52
3. En la semana 12
4. En la semana 12
5. En la semana 52
6. En la semana 52
7. En las semanas 12, 24 y 52
8. Desde la semana 0 hasta la semana 12
9. N/D
10. En la semana 12, 24 y 52
11. N/A |
|
| 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) | 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 | 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 | 43 |
| 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 |
| Brazil |
| Canada |
| Israel |
| Japan |
| Korea, Republic of |
| United States |
| Austria |
| France |
| Poland |
| Netherlands |
| Spain |
| Switzerland |
| Czechia |
| Italy |
| Belgium |
| Norway |
| Portugal |
| United Kingdom |
|
| 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
|
| The end of the platform program (which includes the master and both ISAs) is considered when the last randomized participant in the last intervention cohort has either completed the Week 52 visit and transitioned into the LTE study or completed their final safety visit (as specified in the applicable ISA), or terminated study participation. |
| El final del programa de plataforma (que incluye el maestro y ambos ISA) se considera cuando el último participante aleatorizado en la última cohorte de intervención completó la visita de la Semana 52 y pasó al estudio LTE o completó su visita de seguridad final (como se especifica en la ISA aplicable), o terminó la participación en el estudio. |
|
| E.8.9 Initial estimate of the duration of the trial |
| E.8.9.1 | In the Member State concerned years | 3 |
| E.8.9.1 | In the Member State concerned months | |
| E.8.9.1 | In the Member State concerned days | |
| E.8.9.2 | In all countries concerned by the trial years | 3 |
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