An Open-label Phase 2a Study to Assess the Pharmacokinetics and Pharmacodynamic of PXL065 Parameters 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
- 12
- 主要终点
- PharmacoKinetic
研究概览
简要总结
A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL065 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 PXL065 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN).
There will be a total of 3 study periods.
- Screening period of a maximum of 4 weeks prior to the open-label Treatment Period (Baseline Visit - V2). This period can be exceptionally extended for 2 weeks
- Open-label Treatment Period of 12 weeks
- Follow-up Period of 2 weeks after the last intake of the treatment (V5-End of Treatment Visit (EoT)).
During the treatment period, very long chain fatty acids (VLCFA) will be assessed every 4 weeks, to evaluate the kinetics of the PXL065 effect. Neurofilament light (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 continued monitoring the subjects' safety and evaluation of the kinetics of the 2 main biomarkers (VLCFA and NfL) at 2 weeks after the last intake of the treatment.
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
- 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
PXL065
PXL065 22.5 mg QD
干预措施: PXL065 (Drug)
结局指标
主要结局
PharmacoKinetic
时间窗: Week 4
Deuterated and protonated forms of (R)-pioglitazone and (S)-pioglitazone: AUC0-24 (AUCtau)
Pharmacokinetic
时间窗: Week 4
Deuterated and protonated forms of (R)-pioglitazone and (S)-pioglitazone: Cmax
次要结局
未报告次要终点