| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
| Moderately to Severely Active Crohn's Disease |
| Malattia di Crohn da moderatamente a gravemente attiva |
|
| E.1.1.1 | Medical condition in easily understood language |
| Moderately to Severely Active Crohn's Disease |
| Malattia di Crohn da moderatamente a gravemente attiva |
|
| 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 |
| Valutare l’efficacia e la sicurezza di guselkumab in partecipanti pediatrici affetti da MC alla fine della terapia di mantenimento tra i partecipanti che presentavano una risposta clinica a guselkumab alla Settimana 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. Valutare l’efficacia clinica di guselkumab in partecipanti pediatrici affetti da MC
2. Valutare l’efficacia del trattamento con guselkumab nella remissione clinica in base ai PRO alla Settimana 12 e/o alla Settimana 52
3. Valutare la PK e l’immunogenicità di guselkumab in partecipanti pediatrici affetti da MC
4. Valutare l’impatto della terapia con guselkumab sulla crescita
5. Valutare la sicurezza di guselkumab in partecipanti pediatrici affetti da MC
6. Valutare l’efficacia del trattamento con guselkumab in partecipanti assegnati alla terapia di mantenimento alla Settimana 12 e q4s e non ricevono la terapia di soccorso con il prodotto sperimentale (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. Da 2 a < 18 anni di età inclusi (al momento del consenso allo screening).
2. Stabilità medica basata su esame obiettivo, anamnesi medica e segni vitali eseguiti allo screening. Stabilità medica basata sugli esami clinici di laboratorio eseguiti allo screening.
3. Diagnosi di MC o MC fistolizzante, con colite, ileite o ileocolite attiva, confermata in qualsiasi momento in passato dai criteri clinici, endoscopici e istologici. È inoltre accettabile la diagnosi basata su pregressa resezione chirurgica e istologia. I risultati radiografici potrebbero fornire evidenza a supporto.
4. Presenza di MC in fase attiva da moderata a grave (come definito da un punteggio PCDAI di screening >30).
5. Presentare endoscopia con evidenza di MC attiva definita da un punteggio SES-CD =6 (o =4 per i partecipanti con malattia ileale isolata) entro 4 settimane dalla ricezione dell’intervento dello studio alla Settimana 0.
Fare riferimento al protocollo per ulteriori criteri di esclusione |
|
| 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. Presenza di complicanze della MC, quali aderenze o stenosi sintomatiche, sindrome dell’intestino corto o qualsiasi altra manifestazione che si prevede possa richiedere un intervento chirurgico, che potrebbe precludere l’uso del PCDAI per valutare la risposta alla terapia o che potrebbe confondere la capacità di valutare l’effetto del trattamento. Si noti che le procedure chirurgiche correlate al trattamento delle fistole non rappresentano necessariamente dei criteri di esclusione, discuterne con il responsabile del monitoraggio medico.
2. Attualmente ha o si sospetta abbia un ascesso. Recenti ascessi cutanei e perianali non determinano l’esclusione se drenati e adeguatamente trattati almeno 3 settimane prima della Settimana 0 o 8 settimane prima della Settimana 0 per gli ascessi intra-addominali, a condizione che non si preveda alcuna necessità di un ulteriore intervento chirurgico.
3. Ha subito un qualsiasi tipo di resezione intestinale entro 26 settimane o qualsiasi altro intervento chirurgico intra-addominale entro 12 settimane dal basale.
4. Presenza di stoma, pouch ileo-anale o stomia.
5. Presenta displasia di alto grado, anamnesi o attuale evidenza di displasia polipoide o non polipoide, o qualsiasi adenoma che non è stato rimosso.
Fare riferimento al protocollo per ulteriori criteri di esclusione |
|
| 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. Remissione clinica (definita come punteggio PCDAI =10)
2. Risposta endoscopica (riduzione =50% dal punteggio SES-CD al basale) |
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. At Week 52
2. At Week 52 |
1. Alla Settimana 52
2. Alla Settimana 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. Risposta clinica (diminuzione del riferimento rispetto al basale/LOR nel punteggio PCDAI =12,5; punteggio totale =30)
2. Risposta clinica (PCDAI)
3. Remissione clinica (PCDAI)
4. Risposta endoscopica (SES-CD)
5. Remissione endoscopica (SES-CD)
6. Remissione clinica senza corticosteroidi (definita come punteggio PCDAI =10 alla Settimana 52 e non riceve corticosteroidi per almeno 90 giorni prima della Settimana 52)
7. Remissione clinica sostenuta (definita come PCDAI =10)
8. Concentrazione sierica di guselkumab durante l’induzione
9. Concentrazione sierica di guselkumab durante il mantenimento (almeno Ctrough)
10. Variazione rispetto al basale in:
- Peso
- Percentili e z-score di peso
- Altezza
- Percentili e z-score di altezza
- Velocità di crescita
11. Eventi avversi (EA), compresi gli Eventi avversi seri (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. Alla Settimana 12
2. Alla Settimana 52
3. Alla Settimana 12
4. Alla Settimana 12
5. Alla Settimana 52
6. Alla Settimana 52
7. Alle Settimane 12, 24 e 52
8. Dalla Settimana 0 alla fine della Settimana 12
9. N/D
10. Alle Settimane 12, 24 e 52
11. N/D |
|
| 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 | 4 |
| 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 | Information not present in EudraCT |
| 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. |
| La fine del programma della piattaforma (che include l’originale ed entrambi gli ISA) viene presa in considerazione quando l’ultimo partecipante randomizzato nell’ultima coorte di intervento avrà completato la visita della Settimana 52 e sarà passato allo studio LTE o avrà completato la visita di sicurezza finale (come specificato nell’ISA applicabile) o concluso la partecipazione allo studio. |
|
| 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 | 0 |
| E.8.9.1 | In the Member State concerned days | 0 |
| E.8.9.2 | In all countries concerned by the trial years | 3 |
| 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 |
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