Randomized Controlled Double-blind Vs. Placebo Multicentre Study on the Safety and Effectiveness of Thalidomide in the Treatment of Refractory Crohn's Disease and Ulcerative Colitis.
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 84
- 试验地点
- 7
- 主要终点
- Clinical Remission
研究概览
简要总结
Several open-label studies reported thalidomide efficacy in inducing clinical remission and steroid tapering in refractory Inflammatory Bowel diseases (IBD), both in adults and in children.
This is a randomized placebo controlled (RCT) double blind study, to evaluate the efficacy of thalidomide in inducing clinical remission at 8 weeks in refractory IBD patients aged 2-20 years.
The primary hypotheses of the study is that thalidomide would be more effective than placebo in inducing clinical remission.
The RCT phase is followed by a open-label phase, to further evaluate efficacy and safety of thalidomide in thalidomide responders, with a total follow up of one year.
详细描述
Inflammatory bowel diseases (IBD) are particularly debilitating. They present chronic-recurrent symptoms which often seriously affects the patient's quality of life, acute complications which may need surgical interventions and long-term risks as evolution in cancer for ulcerative colitis.
Although onset is most common in the third decade of life, more than one third of IBD cases are diagnosed before age sixteen, and these younger patients, especially with Crohn's disease, are increasing in Western countries. Recent studies in the United Kingdom, Sweden and the United States report an incidence of 5-7 new cases of IBD every year per 100,000 subjects < 16 years of age, with an increasing trend over the last two decades.
IBD-related morbidity causes special concern in childhood-onset cases, since the disease can compromise height and weight development. This is even worsened in patients with poor disease control, which need steroid treatment in multiple courses and/or for a long period of time.
There are various drugs and treatments available for the management of IBD, but some patients, during the course of the illness, become resistant to conventional treatment, or develop serious adverse effects to immunosuppressive drugs.
A particular challenge is represented by those patients (especially children) who fail to respond to conventional treatment, or are steroid-dependent, or suffer from serious side effects from steroids use.
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
- •Children, adolescents and young adults aged 2 to 20 with chronic refractory moderate to severe inflammatory bowel disease, referred by the six pediatric gastroenterological centres participating in the study.
- •Definition of patient with refractory disease:
- •Patients with active disease despite steroid therapy (prednisone at a dose 2 mg/kg/die, maximum 60mg/day, or equivalent) for 8 weeks and/or an immunosuppressive of proven efficacy (azathioprine o 6-mercaptopurine for 4 months; methotrexate for 3 months; Infliximab at the dose of 5 mg/kg at week 0,2,6 weeks; cyclosporine at the dose of 2mg/kg/day for 4 weeks or 1 week at 1m/kg/day EV) or patients exhibiting intolerance to these drugs which prevent them from continuing treatment.
排除标准
- •Patients with ileostomy or colostomy.
- •Disease requiring immediate surgical intervention.
- •Severe ulcerative colitis or toxic megacolon.
- •Contraindications to using thalidomide (on-going pregnancy, neuropathy) .
- •Any of the following conditions: active infection, stool culture positive for enteric pathogens, tumors, HIV, transplanted organ, or non-controlled disease of the kidney, liver, endocrine system, heart, blood, nervous system or brain.
- •Patients being treated with other drugs as part of an experimental study.
- •Patients treated with infliximab in the previous eight weeks.
研究组 & 干预措施
2
Placebo
干预措施: placebo (Drug)
1
Thalidomide
干预措施: Thalidomide (Drug)
结局指标
主要结局
Clinical Remission
时间窗: 8°, 12°, 26, 52° weeks
次要结局
- Clinical response(4°, 8° weeks)
- Endoscopic remission(12°, 26, 52° weeks)
- Adverse effects(4°, 8°, 12°, 26, 52° weeks)
- Steroid dose reduction(8°, 12°, 26, 52° weeks)
- Nutritinal indicators(8°, 52°)
研究者
Alessandro Ventura
MD
IRCCS Burlo Garofolo