Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia: Part II of a Prospective, Randomized Controlled, Multi-Institutional Trial
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 400
- 试验地点
- 4
- 主要终点
- GCSF Response on transplant-free survival (NoK)
研究概览
简要总结
The Investigators propose to test the hypothesis that GCSF enhances the clinical outcome of biliary atresia in a multi-institutional Phase 2 trial to prospectively evaluate the safety and efficacy of GCSF in each of the 2 groups of newly diagnosed BA patients: KBA (i.e., Kasai-operated) or NoK (i.e., patients who did not undergo Kasai surgery). Subjects who participate in the trial will be followed for 2 years.
详细描述
This is a prospective, randomized, multi-institutional trial in KBA and NoK subjects to be conducted under a Food and Drug Administration approved Investigational New Drug application.
The KBA group is composed of just operated Kasai patients with intraoperative liver biopsy-confirmed BA. Their clinical characteristics have been described in the previously completed Phase 1 study under CR00005169 (ie. inclusion and exclusion criteria as described below)
The NoK group will be composed of newly diagnosed BA patients, including the following:
- surgical patients in whom the Kasai was not performed for intraoperative technical reasons or due to advanced liver disease, who also have no option for rescue liver transplantation.
- Unoperated patients whose family refuses surgery or who are not operative candidates
Having met the same inclusion and exclusion criteria as the Kasai KBS group,
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
- None
入排标准
- 年龄范围
- 14 Days 至 180 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Kasai GCSF
The Kasai GCSF group will receive the standard of care PLUS 3 consecutive daily doses of 10 ug/kg of GCSF to be administered subcutaneously by day 3 post Kasai surgery
干预措施: Filgrastim (Drug)
No Kasai GCSF
The No Kasai GCSF group will receive the standard of care PLUS 3 consecutive daily doses of 10 ug/kg of GCSF to be administered subcutaneously once the diagnosis of BA is established
干预措施: Filgrastim (Drug)
结局指标
主要结局
GCSF Response on transplant-free survival (NoK)
时间窗: 24 months
For NoK subjects: Changes at 6, 12, 18 and 24 months-transplant free survival
GCSF Response on Bile flow (KBA)
时间窗: 3 months
For KBA subjects: Bile flow as measured by the percentage of subjects with total bilirubin\< 2 mg/dL at 3 months post-Kasai.
次要结局
- GCSF response on liver function (NoK)(24 months)
- GCSF response on liver function and outcome (KBA)(24 months)