A Study of Vedolizumab in Children With Ulcerative Colitis (UC) or Crohn's Disease (CD)

JRCT ID: jRCT2071230036

Registered date:05/07/2023

Basic Information

• Recruitment status: Recruiting

Health condition(s) or Problem(s) studied

Ulcerative Colitis
Crohn's Disease

• Date of first enrollment: 16/05/2023
• Target sample size: 240
• Countries of recruitment: United States,Japan,Australia,Japan,Belgium,Japan,Canada,Japan,China,Japan,Croatia,Japan,Greece,Japan,Hungary,Japan,Israel,Japan,Italy,Japan,Korea,Japan,Poland,Japan,Slovakia,Japan,United Kingdom,Japan
• Study type: Interventional

Intervention(s)

Treatment Cohort: Participants 10 to =<15 kg, Vedolizumab 150 mg
Eligible participants from studies MLN0002-3024 or MLN0002-3025 weighing 10 to =<15 kg will receive vedolizumab 150 mg, IV infusion, Q8W, (same as their Week 46 dose in parent study) in this study for up to approximately 5 years.

Treatment Cohort: Participants 10 to =<15 kg, Vedolizumab 100 mg
Eligible participants from studies MLN0002-3024 or MLN0002-3025 weighing 10 to =<15 kg will receive vedolizumab 100 mg, IV infusion, Q8W, (same as their Week 46 dose in parent study) in this study for up to approximately 5 years.

Treatment Cohort: Participants >15 to <30 kg, Vedolizumab 200 mg
Eligible participants from studies MLN0002-3024 or MLN0002-3025 weighing >15 to <30 kg will receive vedolizumab 200 mg, IV infusion, Q8W, (same as their Week 46 dose in parent study) in this study for up to approximately 5 years.

Treatment Cohort: Participants >15 to <30 kg, Vedolizumab 100 mg
Eligible participants from studies MLN0002-3024 or MLN0002-3025 weighing >15 to <30 kg will receive vedolizumab 100 mg, IV infusion, Q8W, (same as their Week 46 dose in parent study) in this study for up to approximately 5 years.

Treatment Cohort: Participants >=30 kg, Vedolizumab 300 mg
Eligible participants from studies MLN0002-3024 or MLN0002-3025 weighing >=30 kg will receive vedolizumab 300 mg, IV infusion, Q8W, (same as their Week 46 dose in parent study) in this study for up to approximately 5 years.

Treatment Cohort: Participants >=30 kg, Vedolizumab 150 mg
Eligible participants from studies MLN0002-3024 or MLN0002-3025 weighing >=30 kg will receive vedolizumab 150 mg, IV infusion, Q8W, (same as their Week 46 dose in parent study) in this study for up to approximately 5 years.

Observational Cohort: Early Terminated Participants From Parent Studies
Participants will have assessment visits at Day 1 and Weeks 8, 34, 60, and 86 as part of a long-term follow-up period to assess prespecified safety events of interest and to monitor growth and pubertal development for approximately 2 years after their last dose of study drug in parent study.

Outcome(s)

Primary Outcome

1.Treatment Cohort: Number of Participants With at Least One Adverse Event (AE)
Time Frame: From first dose of study drug up to approximately 5 years
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have causal relationship with this treatment. AE can be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug whether or not it is considered related to drug.

2.Observational Cohort: Number of Participants With Prespecified Safety Events
Time Frame: Up to approximately 2 years
Prespecified safety events will include serious infections, malignancies, progressive multifocal leukoencephalopathy (PML), concerns about growth and pubertal development, and bowel surgery.

Secondary Outcome

1.Treatment Cohort: Time to Major Inflammatory Bowel Disease (IBD)-related Events
Time Frame: Up to approximately 5 years
Major IBD-related events include hospitalizations, surgeries, and procedures in pediatric participants with UC or CD.

2.Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Total Score for Participants Aged 9 to 17 Years for Every 24 Weeks
Time Frame: Baseline, every 24 weeks in this study (up to approximately 5 years)
The IMPACT-III questionnaire is a self-reported measure with 35 closed questions encompassing 6 domains: Bowel Symptoms (7 items), Systemic Symptoms (3 items), Social Functioning (12 items), Body Image (3 items), Treatment/Interventions (3 items), and Emotional Functioning (7 items). The IMPACT-III uses a 5-point Likert scale ranging from 1 (bad 'quality of life' condition) to 5 (good 'quality of life' condition) for all answers. The total score is an average of all item scores. The outcome score ranges from 35 to 175, with higher scores suggesting better quality of life. This outcome will be assessed in participants who were aged 9 to 17 years at the time of first dose of study drug in study MLN0002-3024 or MLN0002-3025. Baseline refers to the Baseline of study MLN0002-3024 or MLN0002-3025.

3.Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Bowel Symptom Subscale Score for Participants Aged 9 to 17 Years for Every 24 Weeks
Time Frame: Baseline, every 24 weeks in this study (up to approximately 5 years)
The IMPACT-III Bowel Symptom Subscale is a self-reported measure with 7 closed questions. It uses a 5-point Likert scale ranging from 1 (bad 'quality of life' condition) to 5 (good 'quality of life' condition) for all answers. The bowel symptom subscale score ranges from 1 to 35, with higher scores indicating lesser bowel symptoms. This outcome will be assessed in participants who were aged 9 to 17 years at the time of first dose of study drug in study MLN0002-3024 or MLN0002-3025. Baseline refers to the Baseline of study MLN0002-3024 or MLN0002-3025.

4.Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Systemic Symptom Subscale Score for Participants Aged 9 to 17 Years for Every 24 Weeks
Time Frame: Baseline, every 24 weeks in this study (up to approximately 5 years)
The IMPACT-III Systemic Symptom Subscale is a self-reported measure with 3 closed questions. It uses a 5-point Likert scale ranging from 1 (bad 'quality of life' condition) to 5 (good 'quality of life' condition) for all answers. The Systemic symptom subscale score ranges from 1 to 15, with higher scores indicating lesser systemic symptoms. This outcome will be assessed in participants who were aged 9 to 17 years at the time of first dose of study drug in study MLN0002-3024 or MLN0002-3025. Baseline refers to the Baseline of study MLN0002-3024 or MLN0002-3025.

5.Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Social Functioning Subscale Score for Participants Aged 9 to 17 Years for Every 24 Weeks
Time Frame: Baseline, every 24 weeks in this study (up to approximately 5 years)
The IMPACT-III Social Functioning Subscale is a self-reported measure with 12 closed questions. It uses a 5-point Likert scale ranging from 1 (bad 'quality of life' condition) to 5 (good 'quality of life' condition) for all answers. The social functioning subscale score ranges from 1 to 60, with higher scores indicating better social functioning. This outcome will be assessed in participants who were aged 9 to 17 years at the time of first dose of study drug in study MLN0002-3024 or MLN0002-3025. Baseline refers to the Baseline of study MLN0002-3024 or MLN0002-3025.

6.Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Body Image Subscale Score for Participants Aged 9 to 17 Years for Every 24 Weeks
Time Frame: Baseline, every 24 weeks in this study (up to approximately 5 years)
The IMPACT-III Body Image Subscale is a self-reported measure with 3 closed questions. It uses a 5-point Likert scale ranging from 1 (bad 'quality of life' condition) to 5 (good 'quality of life' condition) for all answers. The body image subscale score ranges from 1 to 15, with higher scores indicating better body image. This outcome will be assessed in participants who were aged 9 to 17 years at the time of first dose of study drug in study MLN0002-3024 or MLN0002-3025. Baseline refers to the Baseline of study MLN0002-3024 or MLN0002-3025.

7.Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Treatment/Intervention Subscale Score for Participants Aged 9 to 17 Years for Every 24 Weeks
Time Frame: Baseline, every 24 weeks in this study (up to approximately 5 years)
The IMPACT-III Treatment/Intervention Subscale is a self-reported measure with 3 closed questions. It uses a 5-point Likert scale ranging from 1 (bad 'quality of life' condition) to 5 (good 'quality of life' condition) for all answers. The treatment/intervention subscale score ranges from 1 to 15, with higher scores indicating ease of administration of treatment/interventions. This outcome will be assessed in participants who were aged 9 to 17 years at the time of first dose of study drug in study MLN0002-3024 or MLN0002-3025. Baseline refers to the Baseline of study MLN0002-3024 or MLN0002-3025.

8.Treatment Cohort: Change From Baseline of Studies MLN0002-3024 (UC) or MLN0002-3025 (CD) in IMPACT-III Emotional Functioning Subscale Score for Participants Aged 9 to 17 Years for Every 24 Weeks
Time Frame: Baseline, every 24 weeks in this study (up to approximately 5 years)
The IMPACT-III Emotional Functioning Subscale is a self-reported measure with 7 closed questions. It uses a 5-point Likert scale ranging from 1 (bad 'quality of life' condition) to 5 (good 'quality of life' condition) for all answers. The emotional functioning subscale score ranges from 1 to 35, with higher scores indicating better emotional functioning. This outcome will be assessed in participants who were aged 9 to 17 years at the time of first dose of study drug in study MLN0002-3024 or MLN0002-3025. Baseline refers to the Baseline of study MLN0002-3024 or MLN0002-3025.

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

For Treatment Cohort:
1.The participant should have completed Study MLN0002-3024 or Study MLN0002-3025 and 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 participants with UC, or by a decrease of pediatric Crohn's disease activity index (PCDAI) of >=15 points for participants with CD and with total PCDAI =<30.
2.A male participant who is sexually active with a female partner of childbearing potential agrees to use barrier method of contraception (e.g., 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 participant should also be advised to use a highly effective method of contraception.
3.A female participant 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.
For Observational Cohort:
1.The participant has received at least 1 dose of vedolizumab during Study MLN0002-3024 or Study MLN0002-3025 and early terminated OR completed the Week 54 visit of Study MLN0002-3024 or Study MLN0002-3025 but was not eligible to enroll in the treatment cohort of this study.

Exclude criteria

For Treatment Cohort only:
1.The participant currently requires major surgical intervention for UC or CD (e.g., bowel resection), or is anticipated to require major surgical intervention for UC or CD during the study.
2.The participant has developed any new unstable or uncontrolled cardiovascular, heart failure moderate to severe (New York Class Association III or IV), pulmonary, hepatic, renal, gastrointestinal (GI), 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 participant safety.
3.The participant has other serious comorbidities that will limit their ability to complete the study.
4.The participant is unable to comply with all study assessments.
5.The participant has hypersensitivity or allergies to any of the vedolizumab excipients.
6.The participant is lactating or pregnant.

Related Information

• Primary Sponsor: Shikamura Mitsuhiro
• Secondary ID(s): NCT05442567,2021-000630-34