Long-term Safety and Efficacy Study of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- ApoPharma
- 入组人数
- 68
- 试验地点
- 4
- 主要终点
- Number of Participants With Adverse Events
研究概览
简要总结
Patients with PKAN will be treated with the iron chelator deferiprone for 18 months. Only patients who have completed the earlier study TIRCON2012V1 (NCT01741532), a double-blind placebo-controlled trial in which participants were randomized to receive either deferiprone or placebo for 18 months, are eligible to enroll.
详细描述
TIRCON2012V1-EXT is a multi-center, single-arm, open-label study. All patients who completed the earlier study TIRCON2012V1 (NCT01741532) are eligible to take part. In the initial study, patients were randomized in a 2:1 ratio to receive 18 months of treatment with either the iron chelator deferiprone or placebo, respectively. In this extension study, all participants will receive deferiprone for 18 months. Thus, depending on which product was received earlier, patients will be on deferiprone for a total of either 1.5 years or 3 years. As in the earlier study, assessments will be carried out every six months to look at the safety of the drug and to see if patients are showing any improvement in dystonia and other symptoms of PKAN.
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
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completed study TIRCON2012V1
排除标准
- •Withdrew from the study TIRCON2012V1 for reasons of safety
- •Plan to participate in another clinical trial at any time from the day of enrolment until 30 days post-treatment in the current study
研究组 & 干预措施
Deferiprone
All patients will receive deferiprone oral solution.
干预措施: Deferiprone oral solution (Drug)
结局指标
主要结局
Number of Participants With Adverse Events
时间窗: 18 months
Safety and tolerability were assessed based on changes in: frequency of adverse events (AEs), frequency of serious adverse events (SAEs), and discontinuation due to AEs. No statistical comparison between the groups was conducted as all participants received the same study product.
次要结局
- Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies(Baseline and Month 18 of each study)
- Proportion of Patients With Improved or Unchanged BAD Score(Month 18 of each study)
- Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies(Baseline and Month 18 of each study)
- Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies(Month 18 of each study)
- Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study(Baseline and Month 18 of each study)