A Phase 2 Safety, Tolerability, and Proof-of-Concept Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP) (The Ignite Study)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 20
- 试验地点
- 10
- 主要终点
- Core Study Adverse Events
研究概览
简要总结
This is a multicenter, open-label study to assess the safety and tolerability of iluzanebart (also referred to as VGL101) in subjects with documentation of a gene mutation in the CSF1R gene for the treatment of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) and to evaluate the effects of iluzanebart on imaging and biomarkers of disease progression in subjects with ALSP. Participants will receive infusions of iluzanebart approximately every 4 weeks for 1 year. The study includes a 52-week, open-label Core Study, followed by a Long-Term Extension (LTE), which provides subjects who complete the original 52-week study (Core Study) with the option to continue treatment for up to an additional 2 years.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants who have documentation of a gene mutation in the CSF1R gene
- •Participants fulfill both (Parts A and B) of the following criteria:
- •The participant has more than 2 findings of clinical signs or symptoms in the following categories:
- •Cognitive impairment or psychiatric problem
- •Pyramidal signs on neurological examination
- •Extrapyramidal signs, such as rigidity.
- •MRI findings consistent with ALSP, specifically, bilateral cerebral white matter lesions with or without thinning of the corpus callosum, on the Screening MRI.
- •The participant must have a study partner (i.e., caregiver, family member, friend, etc.) who, in the investigator's judgment, has frequent and sufficient contact with the subject so as to be able to provide accurate information about the participant's health and cognitive and functional abilities. The study partner must be willing to sign a study partner ICF.
排除标准
- •The participant has any neurological disease that poses a risk to the participant or can produce cognitive, motor, or behavioral impairment similar to ALSP, including, but not limited to, brain tumor, hydrocephalus, Alzheimer's disease, frontotemporal dementia (FTD), ALS, stroke, Huntington disease, multiple sclerosis, Parkinson's disease, and Down syndrome.
- •Participant with any condition or situation that, in the opinion of the investigator or sponsor medical personnel, may place the subject at significant risk, confound the study results, or interfere significantly with the participant's participation in the study.
研究组 & 干预措施
VGL101
Solution administered via Intravenous Infusion (IV)
干预措施: VGL101 (Drug)
结局指标
主要结局
Core Study Adverse Events
时间窗: Through Week 52
To evaluate the safety and tolerability of iluzanebart for the treatment of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) by adverse events in the Core Study
Long-Term Extension Adverse Events
时间窗: Week 52 through Week 148
To evaluate the safety and tolerability of iluzanebart for the treatment of ALSP by adverse events in the Long-Term Extension
次要结局
- To evaluate the effects of iluzanebart on brain volume loss on MRI in subjects with ALSP in the Core Study(Baseline and Week 52)
- To assess the correlation between brain volume loss on MRI and clinical measures of disease progression in subjects with ALSP in the Core Study(Baseline and Week 52)
- To evaluate the effects of iluzanebart on clinical measures of disease progression in subjects with ALSP in the Core Study(Baseline and Week 52)
- To evaluate the effects of iluzanebart on fluid biomarkers of neurodegeneration in subjects with ALSP in the Core Study(Baseline and Week 52)
- To evaluate the pharmacokinetics of iluzanebart in subjects with ALSP in the Core Study(Through Week 52)
- To evaluate the effects of iluzanebart on brain volume loss on MRI in subjects with ALSP in the Long-Term Extension(Baseline and Weeks 76, 100, 124, and 148)
- To assess the correlation between brain volume loss on MRI and clinical measures of disease progression in subjects with ALSP in the Long-Term Extension(Baseline and Weeks 76, 100, 124, and 148)
- To evaluate the effects of iluzanebart on fluid biomarkers of neurodegeneration in subjects with ALSP in the Long-Term Extension(Baseline and Weeks 76, 100, 124, and 148)
- To evaluate the effects of iluzanebart on clinical measures of disease progression in subjects with ALSP in the Long-Term Extension(Baseline and Weeks 64, 76, 88, 100, 112, 124, 136, and 148)
- To evaluate the pharmacokinetics of iluzanebart in subjects with ALSP in the Long-Term Extension(Week 52 through Week 148)