Safety & Efficacy of Thalidomide in Children With Transfusion Dependent Thalassemia: a Quasi Randomized Control Trial in a Tertiary Care Hospital in Bangladesh
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- change in Hb level
研究概览
简要总结
Transfusion Dependent Thalassemia (TDT) is emerging as a global public health concern. Hemopoietic stem cell transplantation (HSCT) is the only curative treatment. But its adoption is limited due to lack of Human leukocyte antigen (HLA) matched donor, experienced centers and high initial cost. So, researches are going on in search of an effective, safe, easily available treatment option. Thalidomide a Fetal Hemoglobin (HbF) inducing drug shown to be effective in treatment of TDT patients in few case reports and small scale prospective and retrospective studies. However, most of these researches were done in adolescent and adult population. No randomized control trial was done to determine the safety and efficacy of Thalidomide in TDT children. So, this study will predict the safety and efficacy of Thalidomide in TDT children and will play an important role in planning a cost effective and affordable treatment option for TDT children.
This single centered non blinded quasi randomized clinical trial will be conducted at the Department of Pediatric Hematology and Oncology in Bangabandhu Sheikh Mujib Medical University (BSMMU), Bangladesh for one year of period. The objective of this study is to assess the safety and efficacy of Thalidomide in TDT children 30 transfusion dependent thalassemia children of 3-18 years old will be included.
This study will involve minimum physical risk to the patient. Written informed consent will be taken from parents or study subjects after brief explanation of the purpose and procedure. They will also be informed about the freedom to participate or not to participate at any time. Privacy and confidentiality will be safe guarded. History regarding age, sex, height, weight of these patients will be taken. Through physical examinations and laboratory investigations including complete blood count (CBC), Hb electrophoresis, serum Ferritin, serum creatinine, serum glutamic pyruvic transaminase (SGPT), serum lactate dehydrogenase (LDH) will be done.
Data will be collected in a predesigned questionnaire and will be kept confidential. Statistical analysis will be done using the statistical package for social science (SPSS) software .
详细描述
Study design: Quasi-randomized controlled tria
Period of Study: From March 2023 to February 2023
Place of study:
Department of Pediatric Hematology & Oncology, Bangabandhu Sheikh Mujib Medical University (BSMMU), Dhaka, Bangladesh.
Study Population:
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
- Single (Participant)
入排标准
- 年龄范围
- 3 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients diagnosed as transfusion dependent thalassemia
- •Age ranged from 3-18 years
- •Blood transfusion for more than 1 year.
排除标准
- •Active systemic illness,
- •Abnormal liver and kidney functions;
- •Severe cardiopulmonary or cerebrovascular diseases;
- •Recent fracture or recent major surgery
- •Use of drugs that might affect Hb levels 3 months before enrollment;
- •Any bleeding disorder .
研究组 & 干预措施
Thalidomide group
Thalidomide group
干预措施: Thalidomide Capsules (Drug)
Placebo group
Placebo group
干预措施: Placebo (Drug)
结局指标
主要结局
change in Hb level
时间窗: 12 weeks after treatment initiation.
gm/dl
次要结局
- blood transfusion frequency(3 months from initiation of treatment)
- change in Hb F(3 months after initiation of treatment)
- Change in s. Ferritin level(3 months after initiation of treatment)
- Adverse effect(3 months after initiation of treatment)
研究者
Dr. Md. Mehedi Hasan
Principal Investigator
Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh