E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Ulcerative Colitis, Crohn’s Disease |
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E.1.1.1 | Medical condition in easily understood language |
Ulcerative Colitis, Crohn’s Disease |
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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 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10045365 |
E.1.2 | Term | Ulcerative colitis |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
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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 |
The purpose of this study is to determine if the long-term vedolizumab intravenous treatment is safe in pediatric participants with ulcerative colitis or Crohn's disease. The objectives for the observational cohort are to assess prespecified safety events of interest and to monitor growth and pubertal development for approximately 2 years after the last dose of study drug in Studies MLN0002-3024 or MLN0002-3025. |
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E.2.2 | Secondary objectives of the trial |
- The effect of long-term vedolizumab intravenous treatment on time to major inflammatory bowel disease-related events (hospitalizations, surgeries, and procedures) in pediatric subjects with ulcerative colitis or Crohn's disease - Quality of life in subjects aged 9 to 17 years who were treated with vedolizumab intravenous by using the IMPACT-III questionnaire |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Main Inclusion 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. |
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E.4 | Principal exclusion criteria |
Main Exclusion 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. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Treatment Cohort: - Number of Participants With at Least One Adverse Event Observational Cohort: -Number of Participants With Prespecified Safety Events |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Treatment Cohort: From first dose of study drug up to approximately 5 years
Observational Cohort: Up to approximately 2 years |
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E.5.2 | Secondary end point(s) |
Treatment Cohort: - Time to Major Inflammatory Bowel Disease (IBD)-related Events - Change From Baseline in IMPACT-III Total Score for Participants Aged 9 to 17 Years - Change From Baseline in IMPACT-III Bowel Symptom Subscale Score for Participants Aged 9 to 17 Years - Change From Baseline in IMPACT-III Systemic Symptom Subscale Score for Participants Aged 9 to 17 Years - Change From Baseline in IMPACT-III Social Functioning Subscale Score for Participants Aged 9 to 17 Years - Change From Baseline in IMPACT-III Body Image Subscale Score for Participants Aged 9 to 17 Years - Change From Baseline in IMPACT-III Treatment/Intervention Subscale Score for Participants Aged 9 to 17 Years - Change From Baseline in IMPACT-III Emotional Functioning Subscale Score for Participants Aged 9 to 17 Years |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Treatment Cohort: From first dose of study drug up to approximately 5 years
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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 | Yes |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
Australia |
Canada |
China |
Israel |
Japan |
Korea, Republic of |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
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
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It has been defined as the last subject last visit |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 7 |