A Single-arm, Open-label Study of the Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 65
- 试验地点
- 45
- 主要终点
- Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12
研究概览
简要总结
This single-arm, open-label, multi-center study enrolled 65 patients from approximately 20 centers. All patients who met the study criteria and were taking, beginning or resuming treatment with Deferasirox were allowed. The study will began with a one month run-in phase, where all patients were instructed to take Deferasirox according to their physician's prescribing information.
详细描述
Following the run-in phase, patients entered a three month, assessment phase. During the assessment phase, patients were given five general options for taking Deferasirox including with or without meals, crushed and added to a soft food or mixed in a liquid of choice.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients with thalassemia major, sickle cell disease (SCD), low or intermediate 1 (INT 1) risk myelodysplastic syndrome (MDS) or other anemias and transfusional hemosiderosis.
- •Patients who were on, starting, or resuming treatment with Exjade.
- •Patients who were >2 years (i.e., 2 years of age or older).
- •Exclusion criteria:
- •Serum creatinine above the upper limit of normal (ULN) for age.
- •Alanine aminotransferase (ALT) >2.5 times the ULN.-High risk intermediate-2 or high risk MDS or acute leukemia.
排除标准
- 未提供
结局指标
主要结局
Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12
时间窗: Week 8 and Week 12
Palatability was assessed by participants based on a five-point Facial Hedonic scale defined as: dislike extremely; somewhat dislike; neither like or dislike; somewhat like; like extremely for the meal and method of administration. For participants under 5 years of age, the scale was completed by parent or caregiver.
次要结局
- Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption(Day 1 up to Week 16)
- Change From Baseline in Serum Ferritin at Week 16(Baseline, Week 16 (End of study))
- Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16(Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16)