EFFICACY AND SAFETY OF LOW DOSE THALIDOMIDE VERSUS HYDROXYUREA IN TRANSFUSION DEPENDENT THALASSEMIA PATIENTS: A RANDOMIZED CONTROLLED TRIAL
试验速览
- 阶段
- 2/3 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 90
- 试验地点
- 1
- 主要终点
- 1.Change in hemoglobin levels
研究概览
简要总结
Transfusion dependent thalassemia (TDT) patients constitutes a major disease burden of Indian subcontinent. Till now regular transfusion and adequate chelation forms the main backbone of treatment. There is a worldwide search for newer therapies to decrease the transfusion dependence in TDTs nowadays.
Out study is a randomized controlled trial comparing low dose thalidomide (50mg), hydroxyurea (500mg) and standard of care arm. Our study is based on the fact that low dose thalidomide and hydroxyurea have been found in multiple case reports, single arm retrospective trials individually and in combination to increase haemoglobin levels in thalassemia and decrease transfusion dependence with no serious side-effects causing hospitalisation or death.
It will be a prospective open label randomized controlled trial with blinded end-point study design. Eligible patients with be randomized to either hydroxyurea, thalidomide or standard of care arm. Patients will be regularly followed up at monthly intervals for observing rise in haemoglobin and side-effects if any. Sample size required per group is 27 patients. Estimated duration of trial is 3 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Permuted block randomization, fixed
- 盲法
- Open Label
入排标准
- 年龄范围
- 18.00 Year(s) 至 60.00 Year(s)（—）
- 性别
- All
入选标准
- •1.Treatment naive adult transfusion dependent thalassemia patients of either sex willing to give written informed consent.
- •2.Age more than 18 years.
排除标准
- •1.Those with active systemic comorbidity, with past personal or family history of thrombophilia and recent splenectomy (within 3 months).
- •2.Absolute neutrophil count (ANC) less than 2000/ mm3 in the 8 weeks before study entry or a history of chronic neutropenia, defined as an ANC less than 2000/mm
- •Platelet count less than 100,000/mm3 or greater than 1,000,000/mm3 in the 8 weeks before study entry.
- •4.Evidence of liver disease, as defined by one or more of the following conditions: Alanine aminotransferase (ALT) level greater than three times the upper limit of normal in the 8 weeks before study entry.
- •Serum albumin level less than 3 g/dL in the 8 weeks before study entry.
- •5.Creatinine level more than twice the upper limit of normal for age or greater than 1.5mg/dl.
- •6.Pregnant, planning to become pregnant, or breastfeeding.
- •7.HCV/HBV/HIV infection.
- •8.Currently being treated with any other experimental or fetal hemoglobin-modulating agent.
- •Current participation in any other studies of investigational drugs or devices.
- •Baseline peripheral neuropathy symptoms equivalent to grade 2 Therapy-induced peripheral neuropathy (TiPN) will be excluded.
结局指标
主要结局
1.Change in hemoglobin levels
时间窗: at 3 months and 6 months
次要结局
- 1.Average time to repeat transfusion over 6 months follow up.(2.Average volume of blood transfused over a period of 6 months compared to previous)