An Open-label, Single Treatment, Safety and Efficacy, Long-term Study of Deferiprone in Subjects With Friedreich's Ataxia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- ApoPharma
- 入组人数
- 36
- 试验地点
- 4
- 主要终点
- The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events.
研究概览
简要总结
The primary objective of this study is to evaluate the long-term safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA).
The secondary objective is to evaluate the long-term efficacy of deferiprone for the treatment of FRDA.
The tertiary objectives are to evaluate the effect of deferiprone on:
- cardiac function,
- quality of life, and
- functional status.
详细描述
This is a multi-centre, open-label, non-randomized, single treatment, safety and efficacy study. All subjects who completed the LA29-0207 study are eligible for participation. Participants will receive deferiprone oral solution at the same dose (20 or 40 mg/kg/day) that they were assigned for LA29-0207. The duration of treatment will be 52 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 7 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects who completed the ApoPharma study LA29-0207
- •Female subjects of childbearing potential must have a negative pregnancy test.
- •Male subjects must confirm that he and/or his female partner will use an effective method of contraception for the length of the trial and for 30 days following completion of the study or early termination.
- •Signed and witnessed written informed consent/assent, obtained prior to the first study intervention, as well as the ability to adhere to study restrictions, appointments and evaluation schedules.
排除标准
- •Serum Ferritin and Hemoglobin (Hb) levels are below the reference range for age and sex-matched controls.
- •Unable to complete T25FW AND with a score > 5 minutes in the 9HPT. Subjects who can complete T25FW or with a score ≤ 5 minutes in the 9HPT will be allowed to enrol).
- •Doubling of score on 9HPT or T25FW compared to their study baseline results in LA29-
- •History or evidence of neutropenia/agranulocytosis defined by a confirmed absolute neutrophil count (ANC) < 1.5 x 109/L or thrombocytopenia defined by a platelet count <150 x 109/L.
- •Occurrence of SAEs or any other AEs during the LA29-0207 study, which in the opinion of the investigator cause the patient's participation in the extension study to be inappropriate.
- •Unable to comply with requirements of the protocol.
- •Pregnant, breastfeeding or planning to become pregnant during the study period.
- •QTc interval >450ms.
- •Have been on antioxidants prior to start of study treatment.
研究组 & 干预措施
Dose 1
Deferiprone oral solution 20 mg/kg/day
干预措施: Deferiprone oral solution 100mg/mL (Drug)
Dose 2
Deferiprone oral solution 40 mg/kg/day
干预措施: Deferiprone oral solution 100 mg/mL (Drug)
结局指标
主要结局
The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events.
时间窗: 52 weeks
次要结局
- The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS.(52 weeks)