Long-term Safety and Efficacy Study of Ferriprox® for the Treatment of Transfusional Iron Overload in Patients With Sickle Cell Disease or Other Anemias
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 发起方
- ApoPharma
- 入组人数
- 134
- 试验地点
- 13
- 主要终点
- Number of Patients With Adverse Events
研究概览
简要总结
This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions.
详细描述
Deferiprone (brand name Ferriprox®) is an iron chelator that is approved in the United States and over 60 other countries for the treatment of iron overload in patients with thalassemia, when other treatments are inadequate. This study has been designed to evaluate the long-term efficacy, safety, and tolerability of deferiprone to treat iron overload in patients who have sickle cell disease or other anemias.
Only patients who have completed an earlier study, LA38-0411, are eligible to enroll in this one.
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
- None
入排标准
- 年龄范围
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completed study LA38-0411
- •Females of childbearing potential must have a negative pregnancy test result at Visit
- •In addition, if applicable, they must:
- •Use an effective method of contraception according to local requirements, during the study and within 30 days following their last dose of study medication, OR
- •Have had a tubal ligation (supporting evidence required), OR
- •Have had a hysterectomy (supporting evidence required), OR
- •Participate in a non-heterosexual lifestyle, OR
- •Have a male sexual partner who has been sterilized (supporting evidence required)
- •Fertile heterosexual males and/or their partners must agree to use an effective method of contraception during the study and for 30 days following the last dose of study medication
- •All patients and/or their authorized legal representatives must provide signed and dated written informed consent prior to the first study intervention, and assent will be obtained from patients who are considered to be minors. Patients must be able to adhere to study restrictions, appointments, and evaluation schedules.
排除标准
- •Plan to participate in another clinical trial at any time from the day of enrollment until 30 days post-treatment in the current study
- •For only those patients who were treated with deferoxamine in study LA38-0411 (Group 2): Presence of any medical condition (including clinically significant laboratory abnormalities, such as ALT (alanine aminotransferase) ≥ 5 x ULN or creatinine ≥ 2 x ULN), psychological condition, or psychiatric condition which in the opinion of the investigator would cause participation in the study to be unwise.
- •Pregnant, breastfeeding, or planning to become pregnant during the study period.
- •Treatment failure after 1 year on deferiprone which in the investigator's judgment indicates the need for the patient to be started on a different iron chelator
研究组 & 干预措施
Group 1: Deferiprone 3 years
Patients in this group are those who were randomized to the deferiprone arm in study LA38-0411, and hence will receive deferiprone for a total of 3 years (1 year in the initial study plus 2 years in the extension study).
干预措施: Deferiprone (Drug)
Group 2: Deferiprone 2 years
Patients in this group are those who were randomized to the deferoxamine arm in study LA38-0411, and hence will receive deferiprone for 2 years (both of them in the extension study).
干预措施: Deferiprone (Drug)
结局指标
主要结局
Number of Patients With Adverse Events
时间窗: From the first day of the study until the last study visit (Week 104 or early termination)
Number of patients with at least one adverse event (AE) of any type; number of patients with at least one serious adverse event, and number of patients who withdrew from the study due to an AE
次要结局
- Change From Baseline in Liver Iron Concentration (LIC)(One year, two years, and three years after the start of deferiprone therapy)
- Change From Baseline in Cardiac MRI T2*(One year, two years, and three years after the start of deferiprone therapy)
- Change From Baseline in Serum Ferritin(One year, two years, and three years after the start of deferiprone therapy)