A Group-Wide Double-Blind Randomized Placebo-Controlled Trial of Palifermin to Prevent Chemotherapy and/or Radiotherapy Induced Oral Mucositis in Children Undergoing Autologous or Allogeneic Hematopoietic Stem Cell Transplantation
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 主要终点
- Incidence of WHO grade 3 or 4 oral mucositis
研究概览
简要总结
This randomized phase II trial is studying palifermin to see how well it works compared with a placebo in preventing oral mucositis caused by chemotherapy and/or radiation therapy in young patients undergoing stem cell transplant. Palifermin may help relieve or prevent oral mucositis caused by chemotherapy and radiation therapy in young patients undergoing stem cell transplant.
详细描述
PRIMARY OBJECTIVES:
I. To compare whether palifermin versus placebo administered to pediatric patients three days prior to conditioning and three days after autologous or allogeneic hematopoietic stem cell transplantation (HSCT) is associated with a reduction in the incidence of WHO grade 3 or 4 oral mucositis.
SECONDARY OBJECTIVES:
I. To evaluate the safety and tolerability of palifermin. II. To evaluate the long-term effects of palifermin on disease outcome and survival.
III. To compare the incidence, total dose, and duration of parenteral opioid analgesic use (morphine equivalents), and incidence and duration of total parenteral nutrition (TPN) administration in patients treated with these regimens.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 1 Year 至 16 Years（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
Arm I (palifermin)
Patients receive palifermin IV once daily for 3 days prior to chemotherapy and/or radiotherapy in the absence of unacceptable toxicity. Patients then receive palifermin IV on days 0, 1, and 2 after autologous or allogeneic hematopoietic stem cell transplantation.
干预措施: Palifermin (Drug)
Arm II (placebo)
Patients receive placebo IV once daily for 3 days prior to chemotherapy and/or radiotherapy in the absence of unacceptable toxicity. Patients then receive placebo IV on days 0, 1, and 2 after autologous or allogeneic hematopoietic stem cell transplantation.
干预措施: Placebo (Other)
结局指标
主要结局
Incidence of WHO grade 3 or 4 oral mucositis
时间窗: Up to day 32
The incidence of WHO grade 3 or 4 mucositis, the palifermin and placebo groups, will be compared using a generalized Cochran-Mantel-Haenszel method for general association as the primary analysis. In addition, this outcome will be examined using a logistic regression model; both approaches will account for the randomization strata. Potential confounders will be examined using multiple logistic regression models.
次要结局
- Duration of WHO grade 3 or 4 oral mucositis(Up to day 32)
- Daily OMAS scores(Up to day 32)
- Incidence of adverse events and laboratory abnormalities of palifermin according to using Common Terminology Criteria for Adverse Events (CTCAE) v 3.0(Up to 100 days post-HSCT)
- Daily OMDQ(Up to day 32)
- Incidence, total dose, and duration of parenteral opioid analgesic use (morphine equivalents)(Up to day 32)
- Long-term effects of palifermin on disease outcome and survival(Up to 10 years)
- Daily modified Walsh mucositis scores(Up to day 32)
- Daily pain categorical rating scales(Up to day 32)
- Incidence and duration of total parenteral nutrition administration(Up to day 32)
- Incidence of febrile neutropenia and invasive bacterial infections(Up to day 32)