A Randomized Open-label Phase 2a Study to Assess PXL770 After 12 Weeks of Treatment in Male Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- Poxel SA
- 入组人数
- 24
- 主要终点
- Pharmacokinetic (PK) parameters
研究概览
简要总结
A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL770 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN).
详细描述
A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL770 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN).
There are 3 study periods.
- Screening Visit: within a maximum of 4 weeks prior to the open-label Treatment Period
- Open-label Treatment Period: 12 weeks
- Follow-up Period: 2 weeks after the last intake of the treatment
During the treatment period, VLCFA will be assessed every 4 weeks, to evaluate the kinetics of the effect. NfL will be assessed after 8 and 12 weeks of treatment, and other exploratory biomarkers after 12 weeks of treatment. A follow up period will allow monitoring the subjects' safety as well as the duration of the effect on the 2 main biomarkers (VLCFA and NfL) at 2 and 4 weeks after the drug withdrawal.
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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male subjects with either a confirmed diagnosis of AMN by genetic testing (mutation in the ATP binding cassette subfamily D (ABCD1 gene)) or a family history of X-linked adrenoleukodystrophy (ALD) together with an elevation in VLCFA obtained from overnight fasting plasma sample at Screening Visit (V1).
- •Age: ≥ 18 to ≤ 65 years at informed consent signature.
- •Normal brain magnetic resonance imaging (MRI) or brain MRI showing non-specific abnormalities that can be observed in AMN subjects without signs of cerebral form of ALD (C-ALD). MRI must be performed within 6 months prior to V
- •If there is no available brain MRI within this period, a brain MRI must be performed before V2.
排除标准
- •Any progressive neurological disease other than AMN.
- •Arrested or progressing C-ALD as defined by cerebral lesions (except for non-specific abnormalities that can be observed in AMN subjects).
- •Prior receipt of an allogeneic hematopoietic stem cell transplant or gene therapy.
研究组 & 干预措施
PXL770 500 mg QD
干预措施: PXL770 (Drug)
PXL770 250 mg BID
干预措施: PXL770 (Drug)
结局指标
主要结局
Pharmacokinetic (PK) parameters
时间窗: 4 week
Peak plasma concentration (Cmax) for 500mg QD
Pk parameters
时间窗: 4 week
Area under the plasma concentration versus time curve (AUC)0-24 for 500mg QD
PK parameters
时间窗: 4 week
AUC0-8 for 250mg BID
次要结局
未报告次要终点