Intravitreal Enzyme Replacement Therapy to Prevent Retinal Disease Progression in Children With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Monitoring for the development of unacceptable toxicity.
研究概览
简要总结
This is a phase I/II randomized, masked, clinical trial to determine the safety and efficacy of intravitreal administration of cerliponase alfa.
详细描述
This is a Phase I/II study for 5 subjects receiving an intravitreal injection of cerliponase alfa under sedation into the proclaimed study eye(s) in a 4-week interval over 24 months. This study will be monitored by a Data Safety Monitoring Committee (DSMB). Each subject will participate in the ongoing study for an active period of 2 years. Subjects will then transfer to a bi-annual monitoring program where data will be collected from bi-annual standard of care visits for an additional 3 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
- 24 Months 至 72 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genotypic confirmation of classical CLN2 Batten's disease from a CLIA certified lab.
- •Enzyme level deficiency of tripeptidyl-peptidase
- •Minimum age requirement: 24 months of age at enrollment
- •Maximum age requirement: 72 months of age at enrollment
- •Currently receiving intraventricular cerliponase alfa
- •Willing to participate in the proposed study visits over the 2-year period
- •Minimum central retinal thickness (CRT) of 140μm based upon OCT assessment
- •Clear ocular media
- •No ocular pathology present to account for vision loss other than optic atrophy and pigmentary retinopathy that is felt to be due to the CLN2 disease process
排除标准
- •Any opacities in the clear ocular media including vitreous debris.
- •History of ocular trauma or prior ocular surgery.
- •Episode of generalized motor status epilepticus within four weeks before the First Dose visit
- •Severe infection (e.g., upper respiratory tract infection, pneumonia, pyelonephritis, or meningitis) within four weeks before the First Dose visit (enrollment may be postponed)
- •Those with a history of bleeding disorders.
- •History of or current chemotherapy, radiotherapy or other immunosuppression therapy within the past 30 days (corticosteroid treatment may be permitted at the discretion of the PI)
- •Has a medical condition, or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with the protocol required testing or procedures or compromise the subject's wellbeing, safety, or clinical interpretability
研究组 & 干预措施
Intervention
干预措施: Cerliponase Alfa (Drug)
结局指标
主要结局
Monitoring for the development of unacceptable toxicity.
时间窗: 2 years
Based on the development of unacceptable toxicity, defined as the occurrence of any Grade 3 or higher, unanticipated, treatment related toxicity.
次要结局
- Efficacy of intravitreal cerliponase alfa to stabilize fundoscopic features.(2 years)
- Efficacy of intravitreal cerliponase alfa to stabilize retinal architecture.(2 years)
研究者
David L Rogers, MD
Chief of the Department of Ophthalmology
Nationwide Children's Hospital