A Study of Guselkumab in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis

JRCT ID: jRCT2071230116

Registered date:15/02/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Colitis, Ulcerative
• Date of first enrollment: 08/03/2024
• Target sample size: 120
• Countries of recruitment: Australia,Japan,Belgium,Japan,Canada,Japan,China,Japan,Denmark,Japan,Finland,Japan,France,Japan,Greece,Japan,Italy,Japan,Norway,Japan,Poland,Japan,Portugal,Japan,Spain,Japan,Sweden,Japan,Turkey,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

Open-label Induction Phase: Guselkumab Intravenously (IV)
Participants will receive a guselkumab dose IV based on their body weight (BW) during the 12-week open-label induction phase.

Open-label Induction Phase: Guselkumab Subcutaneously (SC)
Participants will receive a guselkumab dose SC based on their BW during the 12-week open-label induction phase.

Double-blind Maintenance Phase: Guselkumab SC or Guselkumab SC and Placebo SC
At the end of the induction phase, Week 12 responders will be randomized into the double-blind maintenance phase to receive a guselkumab dose SC based on their BW or a guselkumab dose SC based on their BW and placebo SC up to Week 56.

Open-label Maintenance Phase: Guselkumab SC
Week 12 non-responders will enter an open-label maintenance phase to receive guselkumab SC dosing regimen based on their body weight up to Week 56.

Guselkumab Subcutaneous
Guselkumab will be administered subcutaneously.

Matching Placebo
Week 12 induction responders will be administered placebo (matching guselkumab up to Week 56) SC at protocol specified time points to maintain the blind.

Guselkumab Intravenous
Guselkumab will be administered intravenously.

Outcome(s)

Primary Outcome

Percentage of Participants with Clinical Remission at Week 56
Percentage of participants with clinical remission as assessed by modified Mayo score at Week 56 among participants who were induction responders will be reported. Clinical remission per modified Mayo score is defined as a stool frequency subscore of 0 or 1, a rectal bleeding subscore of 0, and an endoscopy subscore of 0 or 1 with no friability present on the endoscopy, where the stool frequency subscore has not increased from induction baseline.

Secondary Outcome

Percentage of Participants with Clinical Remission at Week 12
Percentage of participants with clinical remission at Week 12 as assessed by modified Mayo score will be reported. Clinical remission per modified Mayo score is defined as a stool frequency subscore of 0 or 1, a rectal bleeding subscore of 0, and an endoscopy subscore of 0 or 1 with no friability present on the endoscopy, where the stool frequency subscore has not increased from induction baseline.

Percentage of Participants With Pediatric Ulcerative Colitis Activity Index (PUCAI) Remission at Week 12
Percentage of participants with PUCAI remission at Week 12 will be reported. It comprises 6 scales and ranges between 0 and 85 points. The scales are abdominal pain, rectal bleeding, stool consistency, number of stools, nocturnal bowel movement, and activity level. The PUCAI score is calculated as the sum of the 6 subscores. A PUCAI score of less than (<) 10 indicates remission.

Percentage of Participants with Symptomatic Remission at Week 12
Percentage of participants with symptomatic remission at Week 12 will be reported. Symptomatic remission is defined as a stool frequency subscore of 0 or 1 and a rectal bleeding subscore of 0, where the stool frequency subscore has not increased from induction baseline.

United States: Percentage of Participants with Endoscopic Improvement at Week 12
Percentage of participants with endoscopic improvement as assessed by Mayo endoscopy subscore at Week 12 will be reported. Endoscopic improvement is defined as the Mayo endoscopy subscore of 0 or 1 with no friability present on the endoscopy.

European Union: Percentage of Participants with Endoscopic Healing at Week 12
Percentage of participants with endoscopic healing as assessed by Mayo endoscopy subscore at Week 12 will be reported. Endoscopic healing is defined as the Mayo endoscopy subscore of 0 or 1 with no friability present on the endoscopy.

Percentage of Participants with Clinical Response at Week 12
Percentage of participants with clinical response as assessed by modified Mayo score at Week 12 will be reported. Modified Mayo score is a 3-component (stool frequency, rectal bleeding, and endoscopy subscores) assessment and does not include the physician's global assessment. A decrease from baseline in the modified Mayo score by greater than or equal to (>=) 30 percent and >= 2 points, with either a decrease from baseline in the rectal bleeding subscore of >= 1 or a rectal bleeding subscore of 0 or 1.

Percentage of Participants with Symptomatic Remission at Week 56
Percentage of participants with symptomatic remission at Week 56 will be reported. Symptomatic remission is defined as a stool frequency subscore of 0 or 1 and a rectal bleeding subscore of 0, where the stool frequency subscore has not increased from induction baseline.

United States: Percentage of Participants With Endoscopic Improvement at Week 56
Percentage of participants with endoscopic improvement as assessed by Mayo endoscopy subscore at Week 56 will be reported. Endoscopic improvement is defined as the Mayo endoscopy subscore of 0 or 1 with no friability present on the endoscopy.

European Union: Percentage of Participants With Endoscopic Healing at Week 56
Percentage of participants with endoscopic healing as assessed by Mayo endoscopy subscore at Week 56 will be reported. Endoscopic healing is defined as the Mayo endoscopy subscore of 0 or 1 with no friability present on the endoscopy.

Percentage of Participants with Corticosteroid-free Clinical Remission at Week 56
Percentage of participants with corticosteroid-free clinical remission at Week 56 will be reported. Corticosteroid free clinical remission is defined as a Mayo stool frequency subscore of 0 or 1, a rectal bleeding subscore of 0, and an endoscopy subscore of 0 or 1 with no friability present on the endoscopy, where the stool frequency subscore has not increased from induction baseline (Week 0), and not receiving corticosteroids for at least 8 weeks prior to Week 56

Percentage of Participants with Clinical Response at Week 56
Percentage of participants with clinical response as assessed by modified Mayo score at Week 56 will be reported. Modified Mayo score is a 3-component (stool frequency, rectal bleeding, and endoscopy subscores) assessment and does not include the physician's global assessment. A decrease from baseline in the modified Mayo score by >= 30 percent and >= 2 points, with either a decrease from baseline in the rectal bleeding subscore of >= 1 or a rectal bleeding subscore of 0 or 1.

Percentage of Participants Histo-endoscopic Mucosal Improvement at Week 56
Percentage of participants histo-endoscopic mucosal healing per endoscopy subscore and histologic improvement at Week 56 will be reported. Histologic-endoscopic mucosal healing is defined as achieving a combination of histologic improvement and endoscopic improvement (US) or endoscopic healing (EU) (endoscopy subscore of 0 or 1).

Percentage of Participants with Symptomatic Remission at Week 56 Among Participants who had Symptomatic Remission at Week 12
Percentage of participants with symptomatic remission at Week 56 among participants who had symptomatic remission at Week 12 will be reported. Symptomatic remission score is defined as a stool frequency subscore of 0 or 1 and a rectal bleeding subscore of 0, where the stool frequency subscore has not increased from induction baseline.

Percentage of Participants Who Achieve Endoscopic Normalization at Week 56
Percentage of participants who achieve endoscopic normalization with an endoscopy subscore of 0 at Week 56 will be reported.

Percentage of Participants With PUCAI Remission at Week 56
Percentage of participants with PUCAI remission at Week 56 will be reported. PUCAI comprises of 6 scales and ranges between 0 and 85 points. The scales are: abdominal pain, rectal bleeding, stool consistency, number of stools, nocturnal bowel movement, and activity level. The PUCAI score is calculated as the sum of the 6 subscores. A PUCAI score of less than (<) 10 indicates remission.

Serum Concentration of Guselkumab During Induction Phase
Serum samples will be analyzed to determine concentrations of guselkumab overtime.

Serum Concentration of Guselkumab During Maintenance Phase
Serum samples will be analyzed to determine concentrations of guselkumab over time.

Number of Participants with Incidence of Anti-guselkumab Antibodies
Number of participants with anti-guselkumab antibodies for all study treatment regimens will be assessed.

Percentage of Participants with Adverse Events (AEs)
Percentage of participants with AEs will be reported. An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention.

Percentage of Participants with Serious Adverse Events (SAEs)
Percentage of participants with SAEs will be reported. An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying);persistent or significant disability or incapacity; congenital anomaly.

Percentage of Participants with AEs Leading to Discontinuation of Study Intervention
Percentage of participants with AEs leading to discontinuation of study intervention will be reported.

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: <= 17age old
• Gender: Both

Include criteria

Weight greater than or equal to (>=) 10 kilogram (kg) at the time of consent for screening

A pathology report to support a documented diagnosis of Ulcerative Colitis (UC) must be available in the source documents. There is no maximum duration for which a participant needs to be diagnosed with UC. If the pathology report to support a documented diagnosis of UC is not available in the source documents, the screening endoscopy with biopsies (obtained within 3 weeks before first study intervention administration) needs to support the diagnosis of UC.

Moderately to severely active UC, defined by a baseline modified Mayo (without physician's global assessment) score of 5 through 9 inclusive, with a screening Mayo endoscopy subscore >= 2 as determined by a central review of the video of the endoscopy, and a baseline Mayo rectal bleeding subscore >=1

Medically stable on the basis of physical examination, medical history, and vital signs, performed at screening. Any abnormalities must be consistent with the underlying illness in the study population and this determination must be recorded in the participant's source documents and acknowledged by the investigator

Participants must have had an inadequate response and/or intolerance to biologic therapy and/or conventional therapies or be dependent upon corticosteroids

Exclude criteria

- Have UC limited to the rectum only or to less than (<) 20 centimeter of the colon
- Presence of a stoma
- Has had any kind of bowel resection within 6 months or any other intra-abdominal surgery within 3 months of baseline
- Have severe colitis or have evidence of Crohn's Disease (CD)

Related Information

• Primary Sponsor: Nakama Takahiro
• Secondary ID(s): 2022-001285-35,2022-502238-22-00,NCT06260163