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) - VGL101-01.201

Vigil Neuroscience Inc.0 个研究点目标入组 18 人开始时间: 2023年3月31日最近更新: 2024年7月22日

适应症

试验速览

阶段: 1 期
状态: 招募中
发起方
入组人数: 18

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional

入排标准

年龄范围: 18 至 65+（—）
性别: All

入选标准

•The subject is of either sex aged =18 years on the day the informed consent form (ICF) is signed., The subject is ambulatory with or without aids (cane, crutches, etc) or, if restricted to a wheelchair, can wheel himself/herself, transfer in and out of the wheelchair, and walk up to 5 meters with or without aid. Subjects who are not ambulatory may be eligible after consultation with the medical monitor., If the subject has a cognitive or mental impairment that can affect his/her ability to comply with the study requirements, the subject has a designated study partner. The study partner is able and willing to assist the subject in complying with the study requirements, can provide information during study visits, and is willing to sign a study partner ICF., The subject must have a study partner (ie, 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 subject’s health and cognitive and functional abilities. The study partner must be willing to sign a study partner ICF., If a sexually active women of childbearing potential (ie, women who have not achieved postmenopausal status, defined as cessation of regular menses for ?12 consecutive months with no alternative pathological or physiological cause, and have a serum follicle-stimulating hormone [FSH] level confirming the postmenopausal state) or who have not undergone a documented hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) or a man who has not been surgically sterilized by vasectomy, the subject agrees to use effective contraception during the study and for at least 3 months after the last dose of VGL101., The subject must provide written informed consent, including signing and dating the ICF prior to inclusion in the study, or have a study partner or legal guardian who provides written informed consent with subject assent prior to inclusion in the study if they are unable to provide their own informed consent., If a woman of childbearing potential, the subject has a negative serum pregnancy test at the Screening Visit and a negative urine pregnancy test within 24 hours before administration of the first dose of VGL101 at the Baseline Visit., The subject has a body mass index (BMI) between 17.5 and 38.0 kg/m2, inclusive, at the Screening Visit., The subject meets local guidelines related to COVID-19 testing before the Screening Visit., The subject is, in the investigator’s judgment, able to understand the nature of the study and to comply with the protocol requirements, including scheduled visits, blood and CSF sampling, and other study procedures, or has a study partner or legal guardian who can understand and assist the subject in complying with the protocol requirements for the duration of the study., The subject is willing and able to refrain from use of any medications or treatments that are not permitted by the protocol throughout the study period., The subject has received the approval of Sponsor medical personnel as to final suitability for the study., The subject has documentation of a gene mutation in the CSF1R gene before enrollment into the study. Historical documentation is sufficient to support eligibility for the study; a blood sample for confirmatory testing will be obtained at Baseline., The subject fulfills both (Parts a and b) of the following criteria: a. The subject has >2 findings of clinical signs or symptoms

排除标准

•The subject has any neurological disease that poses a risk to the subject 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., The subject has undergone HSCT or is planning to undergo HSCT during the conduct of this study., The subject is currently enrolled in another investigational drug or device study or has received an investigational product within 30 days or 5 half-lives prior to Screening for this study., The subject has a significant Axis I psychiatric disease as defined by Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (American Psychiatric Association, 2013) that in the opinion of the investigator could pose a risk to the subject or interfere with participation in the study. Presence of minor depression or treated, stable depressive disorder is acceptable., The subject has previously participated in a gene therapy study., The subject is involved, directly or indirectly, in the conduct or administration of this study as an investigator, sub-investigator, study coordinator, or other study staff member., The subject has a clinically significant alcohol or substance use disorder (other than caffeine or nicotine) as defined by Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (American Psychiatric Association, 2013) within 1 year before Screening., The subject has any concurrent diagnosis that may confound neuropsychological testing (eg, hearing impairment, visual impairment) in the opinion of the investigator., The subject has any concurrent diagnosis that may confound ambulation measurements (eg, amputee) in the opinion of the investigator., The subject has a hypersensitivity to anesthetic or derivatives that are used during CSF collection, a history of vertebral deformities, major lumbar back surgery, clinically significant back pain, clinically significant abnormal X-ray, ongoing skin infection or injury at the lumbar puncture injection site, radiological indication of increased intracranial pressure, or a bleeding disorder that, in the opinion of the investigator, would expose the subject to risk of injury or unsuccessful lumbar puncture., The subject is unable to undergo MRI (eg, has implants not compatible for MRI, claustrophobia, is unable to remain still so that a good quality scan can be obtained). Subjects who have renal impairment or contraindications for use of MRI may be enrolled. Contrast MRI scans should not be performed in these subjects; noncontrast scans are required, The subject is at significant risk of suicidal or violent behavior, in the opinion of the investigator. If a subject answers yes” to Question 4 or 5 on the C-SSRS, a risk assessment should be done by a qualified healthcare professional to assess whether it is safe for the subject to participate in the study., The subject has brain MRI findings, at Screening, of acute or subacute hemorrhage, macrohemorrhage, >4 microhemorrhages, or =1 superficial siderosis or other clinically significant imaging abnormalities, including, but not limited to, arteriovenous malformation, aneurism, and subdural hematoma that, in the opinion of the investigator, would pose a risk to the subject., The subject is female and is pregnant, planning pregnancy in the next 12 months, or breastfeeding., All groups of

研究者

发起方

Vigil Neuroscience Inc.

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