A Prospective Randomized Double-Blind Study of PASER® in the Management of Patients Experiencing an Acute Flare of Crohn's Disease
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 2
- 试验地点
- 5
- 主要终点
- Reduction in the Modified Crohn's Disease Activity Index (mCDAI) Score of >70 Points by 4 Weeks Compared With Baseline
研究概览
简要总结
The purpose of this 4 week study is to determine whether PASER®, an approved delayed-release oral formulation of 4-aminosalicylic acid, in doses of 50 milligrams per kilogram three times daily for 2 weeks followed by 50 milligrams per kilogram twice daily for 2 weeks, will resolve an acute flare of ileocecal Crohn's disease.
详细描述
Eligible pediatric patients with acute flares of ileocecal Crohn's disease will be randomized to receive either PASER®, an approved delayed-release oral formulation of 4-aminosalicylic acid, in doses of 50 milligrams per kilogram three times daily for 2 weeks followed by 50 milligrams per kilogram twice daily for 2 weeks, or an identical-appearing placebo preparation.
Patients will be required to maintain a daily diary and to return at 2 weeks for blood and stool tests. At the four week mark, patients will return for clinical evaluation, global assessment of disease activity and change in disease activity, as well as additional laboratory tests.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age less than 18 years
- •Crohn's disease predominantly involving the ileum and/or cecum. The diagnosis must have been established by radiography, endoscopy and/or biopsy (at least 2 of the 3 modalities) with at least one confirmatory test having been performed no more than 36 months before entry. The diagnosis must have been confirmed by at least one gastroenterologist.
- •Harvey Bradshaw Index of at least 7
- •The onset of the acute flare should have been abrupt, declaring itself over 72 hours, and should have started no more than 4 weeks before study entry. Symptoms relating to the flare should not have diminished or started to improve prior to entry.
- •Written informed consent
排除标准
- •Concomitant corticosteroids, budesonide
- •Corticosteroids within 2 months
- •Cyclosporine, mycophenolate mofetil or experimental drugs during the last three months
- •Maintenance infliximab, or infliximab or other biologics in the preceding 3 months
- •If the severity of the flare has started to decrease spontaneously
- •Coexisting diagnosis of primary sclerosing cholangitis
- •Infectious diarrhea
- •Signs of intestinal obstruction or perforation
- •New fistulization as part of the acute flare or increased activity in chronic fistula(e) as part of the acute flare
- •Hypersensitivity to 4-ASA or any components of PASER®
- •Pregnancy or breast-feeding
- •Failure of a woman of child-bearing potential to agree to use adequate contraception for the 4 week period of the trial, if sexually active
- •Severe renal or hepatic disease (i.e., more than 3 times upper limit of normal) or a WBC < 3,000 during the preceding three months
研究组 & 干预措施
Active
4-Aminosalicylic acid extended release granules (as volume equivalent of active product), 50 mg/kg orally three times daily for two weeks followed by (as volume equivalent) 50 mg/kg orally two times daily for 2 weeks
干预措施: 4-Aminosalicylic acid extended release granules (Drug)
Placebo
Placebo granules identical in appearance to the active arm (as volume equivalent of active product), 0 mg/kg orally three times daily for two weeks followed by (as volume equivalent) 0 mg/kg orally two times daily for 2 weeks
干预措施: 4-Aminosalicylic acid extended release granules (Drug)
结局指标
主要结局
Reduction in the Modified Crohn's Disease Activity Index (mCDAI) Score of >70 Points by 4 Weeks Compared With Baseline
时间窗: 4 weeks
Reduction in the Modified Crohn's Disease Activity Index (mCDAI) score of \>70 points by 4 weeks after randomization compared with baseline
次要结局
- Rate of Response as Defined by a Reduction in HBI to Less Than 5 by 4 Weeks(4 weeks)
- Rate of Remission as Defined by the Decrease in PCDAI < 10 by 4 Weeks(4 weeks)
- Rate of Remission as Defined by the Decrease in HBI to Less Than 3 by 4 Weeks(4 weeks)
- Time to Response and/or Remission Including Time to Change in HBI, According to Elements of the Daily Patient Diary(up to 4 weeks)
- Rate of Remission(4 weeks)
- Change in Global Physician Assessment of Disease Activity From Baseline to Study Completion(4 weeks)
- Change in IMPACT-III From Baseline to 4 Weeks(4 weeks)
- Change From Baseline in the Patient's General Sense of Disease Activity as Recorded in the Individual Daily Diary(4 weeks)
- Absence of Night Time Stools, if They Were Present on Entry, and Time to Disappearance(up to 4 weeks)
- Time to Normalization of All Other Components in the Diary(up to 4 weeks)
- Rate of Response as Defined by the Decrease in PCDAI of 12.5 Points by 4 Weeks(4 weeks)
- Change in Hgb, ESR, CRP, Platelet Count, Calprotectin From Baseline and Time to Normalization(2 weeks and 4 weeks)