A Prospective Randomized Comparative Study of Efficacy and Safety of Combined Deferiprone (DFP) and Deferasirox Versus DFP and Desferrioxamine (DFO) Therapy in Diseases With Severe Iron Overload
试验速览
- 阶段
- 2 期
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- to assess efficacy of combining DFP and deferasirox compared to combined DFP and DFO in decreasing the serum ferritin level in conditions with severe chronic iron overload.
研究概览
简要总结
Interventional Allocation: Randomized Endpoint Classification: Safety/Efficacy Study of combined chelation therapy Masking: Open Label Primary Purpose: Treatment of transfusional iron overload
Primary Outcome Measures:
• The primary outcome measure is to assess efficacy in lowering serum ferritin level(the change in serum ferritin compared to baseline) with combining DFP and deferasirox compared to combined DFP and DFO in conditions with severe chronic iron overload; showing an up-trend of SF over previous 12 months on single chelator.
Secondary Outcome Measures:
• The secondary outcome measure is to determine the number of patients who will develop adverse events in order to assess safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP.
详细描述
Study population:
- Beta-thalassemia major patients; Patients with high iron stores Serum ferritin consistently > 2500 mcg/l and or increasing trend over previous 12 months Liver iron >14 mg/g dry weight- by R2 MRI
- Other causes of transfusional hemosiderosis
Estimated Enrollment: 30 patients in each arm Study Start Date: January 1st ; 2011 enrollment period 8 weeks Estimated Study Completion Date: End of February 1212
Arms Assigned Interventions
Arm 1: Patients will be treated with combined DFP and deferasirox. Drug: Ferriprox It will be given orally in the morning Other Name: DFP Initial dose 25/mg/kg in 3 divided doses (better tolerated if started and then built up over 4 weeks).
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
- 6 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects with transfusional iron overload secondary to thalassemia major , sickle cell disease showing up-trend in SF aged 6 Years or older, may participate after Approval of Ethical committee giving written informed consent.
- •Subjects must have a serum ferritin greater than >2500 ng/mL, a platelet count greater than 100,000/mm3, and a serum creatinine within the normal range.
- •A woman of childbearing potential must have a negative serum pregnancy test at screening. She must use a medically acceptable form of birth control during the study and for 1 month afterward.
- •The subjects must also have a level of understanding and willingness to cooperate with the confinement and procedures described in the consent form and scheduled by the study site. In addition, he/she must be able to provide voluntary written informed consent.
排除标准
- •Subjects with a past history of agranulocytosis, history of clinically significant gastrointestinal, renal, hepatic ALT > 10 times high normal, OR > 50% increase of serum creatinine from basal value, pulmonary or cardiovascular disease. Patients with a history of tuberculosis, epilepsy, psychosis, glaucoma or any other condition, which in the opinion of the investigators, would jeopardize the safety of the subject or impact the validity of the study results.
- •Subjects with HIV positive or have active HCV.
- •A history of serious immunologic hypersensitivity to any medication, such as anaphylaxis or angioedema.
- •Participation in a previous investigational drug study within the 30 days preceding screening..
- •Women who are pregnant, or breast-feeding.
- •Current alcohol or drug abuse.
- •An inability to adhere to the designated procedures and restrictions of this protocol.
- •Subjects receiving warfarin, digoxin, or anti-arrhythmic or anti-seizure medications.
- •Subjects with a known allergy to Exjade or DFP that prevents chronic administration.
研究组 & 干预措施
arm 1
30 Patients will be treated with combined DFP and deferasirox.
干预措施: DFP (ferriprox) and deferasirox (ICL 670) (Drug)
arm 2
Patients will be treated for 6 days with a combination of deferoxamine and DFP
干预措施: DFP, DFO (Drug)
结局指标
主要结局
to assess efficacy of combining DFP and deferasirox compared to combined DFP and DFO in decreasing the serum ferritin level in conditions with severe chronic iron overload.
时间窗: 12 months
• The primary outcome measure is to measure the change in serum ferritin level from baseline in the 2 combination therapy.
次要结局
- to determine the safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP.(12 months)
研究者
Mohsen Saleh Elalfy
professour of pediatrics
Ain Shams University