A Study of Guselkumab in Pediatric Participants with Moderately to Severely Active Crohn's Disease

JRCT ID: jRCT2031230352

Registered date:21/09/2023

Basic Information

• Recruitment status: Pending
• Health condition(s) or Problem(s) studied: Crohn's Disease
• Date of first enrollment: 22/12/2023
• Target sample size: 120
• Countries of recruitment: Australia,Japan,Belgium,Japan,Brazil,Japan,Canada,Japan,Switzerland,Japan,Czechia,Japan,Spain,Japan,France,Japan,United Kingdom Of Great Britain,Japan,Israel,Japan,Italy,Japan,Korea.Republic Of,Japan,Netherlands,Japan,Norway,Japan,Poland,Japan,Portugal,Japan,United States Of America,Japan
• Study type: Interventional

Intervention(s)

Guselkumab:Open-label induction phase:Guselkuma bIntravenously (IV) Open-label induction phase:Guselkumab Subcutaneously(SC) Double-blind maintenance phase:Guselkumab SC or Guselkumab SC and Placebo SC Open-label maintenance phase:Guselkumab SC:Guselkumab will be administered either intravenously or subcutaneously.
Placebo:Double-blindmaintenance phase:Guselkumab SC or Guselkumab SC and Placebo SC:Week 12 induction responders will be administered placebo (matching guselkumab up to Week 48) SC at protocol specified time points to maintain the blind.

Outcome(s)

Primary Outcome

-Percentage of Participants with Clinical Remission at Week 52:Percentage of participants with clinical remission at Week 52 will be assessed. Clinical remission is defined as pediatric Crohn's Disease activity index (PCDAI) less than or equal to (<=) 10.
-Percentage of Participants Who Achieve Endoscopic Response at Week 52::Percentage of participants who achieve endoscopic response at Week 52 will be assessed. Endoscopic response is defined as greater than or equal to (>=) 50 percent (%) reduction from simplified endoscopic score-Crohn's Disease (SES-CD) score at baseline.

Secondary Outcome

- Percentage of Participants with Clinical Response at Week 12: Percentage of participants with clinical response at Week 12 will be assessed. Clinical responder is defined as a decrease from baseline/loss of response (LOR) in the PCDAI score of >=12.5 points with a total PCDAI score <=30.
- Percentage of Participants with Clinical Response at Week 52: Percentage of participants with clinical response at Week 52 will be assessed. Clinical responder is defined as a decrease from baseline/LOR in the PCDAI score of >=12.5 points with a total PCDAI score <=30.
- Percentage of Participants with Clinical Remission at Week 12: Percentage of participants with clinical remission at Week 12 will be assessed. Clinical remission is defined as PCDAI score <=10.
- Percentage of Participants Who Achieve Endoscopic Response at Week 12: Percentage of participants who achieve endoscopic response at Week 12 will be assessed. Endoscopic response is defined as >=50% reduction from SES-CD score at baseline.
- Percentage of Participants with Endoscopic Remission at Week 52: Percentage of participants with endoscopic remission at Week 52 will be assessed. Endoscopic remission is defined as SES-CD total score <=4 and at least a 2-point reduction from baseline and no subscore >1.
- Percentage of Participants with Corticosteroid-free Remission at Week 52: Percentage of participants with corticosteroid-free remission at Week 52 will be assessed. Corticosteroid-free remission is defined as PCDAI score <=10 at Week 52 and not receiving corticosteroids for at least 90 days before Week 52.
- Percentage of Participants with Sustained Clinical Remission at Weeks 12, 24, and 52: Percentage of participants with sustained clinical remission at Weeks 12, 24, and 52 will be assessed. Sustained clinical remission is defined as PCDAI <=10 at Weeks 12, 24, and 52.
- Percentage of Participants with Clinical remission by Patient-Reported Outcome (PRO):Week 12 and/or Week 52:Percentage of participants with clinical remission by PRO will be assessed. Clinical remission by PRO is defined as stool frequency (SF) <=3 and abdominal pain (AP) <=1 and no worsening of SF and AP from baseline.
- Serum Concentration of Guselkumab During Induction Phase:From Week 0 to Week 12:Serum concentrations of guselkumab will be assessed. Serum samples will be analyzed to determine concentrations of guselkumab using a validated, specific, and sensitive immunoassay method.
- Trough Plasma Concentration (Ctrough) of Guselkumab During Maintenance Phase:At Weeks 16, 24, 36, 48 and 52:Ctrough is defined as the serum concentration of guselkumab immediately prior (pre-dose) to the next drug administration.
- Change from Baseline in Body Weight at Weeks 12, 24, and 52: Change from baseline in body weight at Weeks 12, 24, and 52 will be assessed.
- Change from Baseline in Body Weight Percentiles at Weeks 12, 24, and 52: Change from baseline in body weight percentiles at Weeks 12, 24, and 52 will be assessed.
- Change from Baseline in Body Weight z-scores at Weeks 12, 24, and 52: Change from baseline in body weight z-scores at Weeks 12, 24, and 52 will be assessed.
- Change from Baseline in Height at Weeks 12, 24, and 52: Change from baseline in height at Weeks 12, 24, and 52 will be assessed.
- Change from Baseline in Height Percentiles at Weeks 12, 24, and 52: Change from baseline in height percentiles at Weeks 12, 24, and 52 will be assessed.
- Change from Baseline in Height z-scores at Weeks 12, 24, and 52: Change from baseline in height z-scores at Weeks 12, 24, and 52 will be assessed.
- Change from Baseline in Height Velocity at Weeks 12, 24, and 52: Change from baseline in height velocity at Weeks 12, 24, and 52 will be assessed.
- Percentage of Participants with Clinical Remission:Week 52:Percentage of participants with clinical remission who were assigned to q4w maintenance therapy and did not receive rescue therapy at Week 52 will be assessed. Clinical remission is defined as PCDAI score <=10.
- Percentage of Participants Who Achieve Endoscopic Response:Week 52:Percentage of participants who achieve endoscopic response who were assigned to q4w maintenance therapy and did not receive rescue therapy at Week 52 will be assessed. Endoscopic response is defined as >=50% reduction from SES-CD score at baseline.

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: < 18age old
• Gender: Both

Include criteria

-Participants must have a diagnosis of Crohn's Disease (CD) or fistulizing CD, with active colitis, ileitis, or ileocolitis, confirmed at any time in the past by clinical, endoscopic, and histologic criteria.
- Participants must have moderately to severely active CD (as defined by a baseline Pediatric Crohn's Disease Activity Index [PCDAI] score greater than [>] 30)
- Participants must have endoscopy with evidence of active CD defined as Simple Endoscopic Score for Crohn's Disease (SES-CD) score greater than or equal to (>=) 6 (or >=4 for participants with isolated ileal disease) during screening into this study
- Participants must have a prior or current CD medication history that includes either inadequate response, loss of response to or failure to tolerate current treatment immunomodulators or with oral or intravenously (IV) corticosteroids or have received biologic therapy/JAK inhibitor for the treatment of CD and have a documented history of inadequate response, loss of response (LOR), or intolerance to the biologic therapy/JAK inhibitor

Exclude criteria

- Participants has complications of CD such as symptomatic strictures or stenosis, short gut syndrome, or any other manifestation that might be anticipated to require surgery.
- Participants must not have an abscess
- Participants must not have any kind of bowel resection within 26 weeks or any other intra-abdominal surgery within 12 weeks of baseline

Related Information

• Primary Sponsor: Nakama Takahiro
• Secondary ID(s): 2021-006282-37,NCT05923073