The Efficacy of Oral Prednisone in Treating Langerhans Cell Histiocytosis of Bone in Childhood and Adolescence: A Multi-center, Open-label, Randomized-controlled, Phase II Study
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 118
- 试验地点
- 1
- 主要终点
- Progression-free survival
研究概览
简要总结
Langerhans cell histiocytosis (LCH) of bone is a benign-tumor-like osteolytic lesion in childhood and adolescence, which is characterized by the aberrant activation of antigen presenting cells. Rather than the multi-system involvements of LCH, no standard or widely-accepted therapeutic regimens were established for LCH of bone. In the previous clinical practice, several LCH patients obtained remarkable pain relief after taking prednisone. Therefore, the investigators aim to conducting a multi-center, open-labelled, randomized-controlled, Phase II study to investigate the efficacy and safety of oral prednisone in treating LCH of bone in children and adolescents. The enrolled patients will be randomly recruited to the following groups: (1) Oral prednisone [Test group); (2) Regular observation [Control group].
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- None
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
- •Pathological diagnosis of Langerhans cell histiocytosis (LCH) of bone;
- •Single-system involvement (skeletal system);
- •No need of surgical intervention;
- •Must be able to swallow tablets;
- •Signing informed consent form.
排除标准
- •Multi-system involvements (≥2 systems, including bone, liver, spleen, hematologic system, central nerve system);
- •Need of surgical intervention (e.g. pathological fracture and/or spinal cord compression)
- •Glucocorticoid allergy;
- •Immunodeficiency;
- •Severe infection;
- •Insulin dependent/independent Diabetes;
- •Having taken glucocorticoid in the past two weeks;
- •Not capable of swallowing tablets;
- •Without signed informed consent inform.
研究组 & 干预措施
Oral prednisone
Oral prednisone, 0.5mg/kg/day, first 5 days/month, 6months
干预措施: Prednisone (Drug)
结局指标
主要结局
Progression-free survival
时间窗: 2 years
Any evidence proving the disease progression
次要结局
- Local control rate(2 years)
- Adverse effect rate(2 years)
- Pain relief(2 years)
研究者
Jianru Xiao
Professor, Chief Surgeon
Shanghai Changzheng Hospital