A Phase 2b, Extension Study to Determine the Long-term Safety of Vedolizumab IV in Pediatric Subjects With Ulcerative Colitis or Crohn's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 59
- 试验地点
- 23
- 主要终点
- Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
The purpose of this study is to determine the safety profile of long-term vedolizumab IV treatment in pediatric participants with UC or CD.
详细描述
The drug being tested in this study is called Vedolizumab. Vedolizumab is being tested to treat pediatric participants who have moderately to severely active UC or CD.
This study will look at the long-term safety profile in participants who take vedolizumab IV. Participants will continue receiving the same dose assigned from the parent study MLN0002-2003 [NCT03138655], which will remain blinded until week 40.
The dosing regimen selected for the long-term study is intended to maintain clinical response at the lowest possible exposure.
At the discretion of the investigator, participants receiving the low dose (150 or 100 milligram [mg]) of vedolizumab IV may be escalated to the high dose (300 or 200 mg) if the participants demonstrate disease worsening at 2 consecutive visits (scheduled or unscheduled).
Participants who experience continued disease worsening during the study despite being administered vedolizumab 300 or 200 mg every 8 weeks (Q8W) will be discontinued from the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Is male or female with UC or CD and was between 2 to 17 years, inclusive, at the time of randomization for Study MLN0002-
- •(Note: A participant remains eligible to participate in this study after they reach 18 years of age if they continue to meet the inclusion criteria and do not meet any
排除标准
- •Has completed Study MLN0002-2003 and, at Week 22, achieved clinical response as defined by a reduction of partial Mayo score of >=2 points and >=25% from Baseline, or a reduction of the Paediatric Ulcerative Colitis Activity Index (PUCAI) of >=20 points from baseline for participants with UC; or a reduction of the CDAI as defined by a >=70-point decrease from Baseline or a decrease of Pediatric Crohn's Disease Activity Index (PCDAI) of >=15 points for participants with CD.
- •May be receiving a therapeutic dose of the following drugs:
- •Oral 5-aminosalicylic acid (5-ASA) compounds.
- •Oral corticosteroid therapy (prednisone or equivalent steroid at a dose less than or equal to [<=] 50 milligram per day [mg/day]) provided the participant was receiving this medication during prior participation in MLN0002-
- •Topical (rectal) treatment with 5-ASA or corticosteroids.
- •Probiotics (example, Saccharomyces boulardii).
- •Antidiarrheals (example, loperamide, diphenoxylate with atropine) for control of chronic diarrhea.
- •Antibiotics used for the treatment of CD (i.e., ciprofloxacin, metronidazole).
- •Azathioprine (AZA) or 6-mercaptopurine (6-MP) or methotrexate (MTX), provided the participant was receiving this medication during prior participation in MLN0002-
- •The participant's vaccinations are up to date as per inclusion criteria number 10 in MLN0002-
- •Exclusion Criteria:
- •Is female and is lactating or pregnant.
- •Has hypersensitivity or allergies to vedolizumab or any of its excipients.
- •Has withdrawn from Study MLN0002-
- •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.
- •Has a positive progressive multifocal leukoencephalopathy (PML) subjective symptom checklist prior to the administration of the first dose of study drug.
- •Currently requires major surgical intervention for UC or CD (example, bowel resection), or is anticipated to require major surgical intervention for UC or CD during the study.
- •Has other serious comorbidities that will limit his or her ability to complete the study.
研究组 & 干预措施
Vedolizumab Low Dose Group
Participants with UC or CD having baseline weight of >= 30 kg will receive vedolizumab 150 mg and participants with UC or CD having baseline weight of < 30 kg will receive vedolizumab 100 mg IV infusion, every 8 weeks until vedolizumab IV is commercially available for pediatric indication(s) in the participant's country or until other drug access programs become available (whichever comes first), the participant turns 18 years of age and can be transitioned to commercial drug (up to approximately 8 years).
干预措施: Vedolizumab (Drug)
Vedolizumab High Dose Group
Participants with UC or CD having baseline weight of greater than or equal to (>=) 30 kilogram (kg) will receive vedolizumab 300 mg and participants with UC or CD having baseline weight of less than (<) 30 kg will receive vedolizumab 200 mg, IV infusion, every 8 weeks until vedolizumab IV is commercially available for pediatric indication(s) in the participant's country or until other drug access programs become available (whichever comes first), the participant turns 18 years of age and can be transitioned to commercial drug (up to approximately 8 years).
干预措施: Vedolizumab (Drug)
结局指标
主要结局
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
时间窗: From first dose of study drug up to end of follow up (up to 6.8 years)
AE defined as any untoward medical occurrence in clinical investigation participants administered drug; it does not necessarily have to have causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug whether or not it was considered related to the drug. TEAE was defined as an AE whose date of onset occurred on or after the first dose of study drug, or an already-present AE that worsened in intensity or frequency following the treatment start, occurring from the first dose of study drug to the day of last dose of study drug.
次要结局
- Percentage of Participants With UC Who Achieved and Maintained Clinical Response Based on Complete Mayo Score(At Week 32)
- Percentage of Participants With CD Who Achieved and Maintained Clinical Response Based on Simple Endoscopic Score for Crohn's Disease (SES-CD) Score and Crohn's Disease Activity Index (CDAI) at Week 32(At Week 32)
- Time to Major Inflammatory Bowel Disease (IBD) - Related Events(Up to 6.8 years)
- Change From Baseline in IMPACT-III - Total Score(Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312)
- Change From Baseline in IMPACT-III - Bowel Symptoms Domain Score(Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312)
- Change From Baseline in IMPACT-III - Systemic Symptoms Domain Score(Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312)
- Change From Baseline in IMPACT-III - Social Functioning Domain Score(Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312)
- Change From Baseline in IMPACT-III - Body Image Domain Score(Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312)
- Change From Baseline in IMPACT-III - Treatment/Interventions Domain Score(Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312)
- Change From Baseline in IMPACT-III - Emotional Functioning Domain Score(Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, and 312)
- Height Velocity at Week 48 and Every 48 Weeks(At Weeks 48, 96, 144, 192, 240, 288, and 336)
- Change From Baseline in Height(Baseline, Weeks 48, 96, 144, 192, 240, 288, 336)
- Change From Baseline in Weight(Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, 312, and 336)
- Change From Baseline in Body Mass Index (BMI)(Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, 312, and 336)
- Percentage of Participants Who Achieved Tanner Stage V at or Before Age 16 (in Females) or Age 17 (in Males)(Up to 6.8 years)