EUCTR2016-001955-29-ES进行中（未招募）1 期
Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension
Retrophin, Inc.0 个研究点目标入组 82 人开始时间: 2017年3月30日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 82
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •- The patient has a diagnosis of PKAN as indicated by confirmed mutations in the pantothenate kinase 2 (PANK2) gene.
- •- The patient has a score of >= 6 on the Pantothenate Kinase-associated Neurodegeneration Activities of Daily Living (PKAN-ADL) scale.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 41
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 41
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •- The patient has required regular or intermittent invasive ventilatory support to maintain vital signs within 24 weeks prior to randomization.
- •- The patient has had a deep brain stimulation (DBS) device implanted within 6 months prior to screening.
- •- The patient is unable or unwilling to remain on their pre-study dose(s) of allowed concomitant PKAN maintenance medications and therapies for the double-blind period of the study.
- •- The patient has taken deferiprone within 30 days prior to screening.
研究者
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