NCT04411654进行中（未招募）1 期
An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose LY3884961 in Infants With Type 2 Gaucher Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 7
- 试验地点
- 11
- 主要终点
- Immunogenicity of AAV9 and GCase in blood
研究概览
简要总结
J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 Gaucher disease (GD2). For each patient, the study will be approximately 5 years in duration. During the first 12 months after dosing, patients will be evaluated for the effects of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will be followed up for an additional 4 years to monitor safety and changes on selected biomarkers and clinical outcomes.
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
盲法说明
Blinded assessor used in secondary outcome measures
入排标准
- 年龄范围
- 0 Months 至 24 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Bi-allelic GBA1 mutations consistent with a diagnosis of GD2 confirmed by the central laboratory.
- •Clinical diagnosis of GD2
- •Parent/legal guardian is capable of providing signed informed consent; including compliance with the requirements and restrictions listed in the informed consent form (ICF) in this protocol.
- •Patient has a parent/legal guardian able to participate in the study as a source of information on the patient's health status and cognitive and functional abilities (including providing input into the rating scales).
排除标准
- •Significant CNS disease other than GD2 that may be a cause for the patient's symptoms or interfere with study objectives.
- •Achieved independent gait.
- •Severe peripheral symptoms of GD which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
- •Concomitant disease, condition, or treatment which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
- •Use of any substrate reduction therapy (SRT) for GD treatment.
- •Use of prohibited medications, herbals, or over-the-counter agents as listed in the protocol.
- •Any type of prior gene or cell therapy.
- •Use of systemic immunosuppressant or corticosteroid therapy other than protocol-specified immunosuppression.
- •Participation in another investigational drug or device study within the past 3 months.
- •Brain MRI (magnetic resonance imaging) and MRA (magnetic resonance angiography) showing clinically significant abnormality deemed a contraindication to intracisternal injection.
- •Clinically significant laboratory test result abnormalities assessed at screening.
- •Contraindications or intolerance to radiographic visualization methods (e.g. MRI, MRA, CT), and intolerance to contrast agents used for MRI or CT scans.
- •Contraindications to general anesthesia or sedation.
- •Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
Low Dose
Experimental
干预措施: LY3884961 (Genetic)
Low Dose
Experimental
干预措施: Methylprednisolone (Drug)
High Dose
Experimental
干预措施: Methylprednisolone (Drug)
High Dose
Experimental
干预措施: LY3884961 (Genetic)
Low Dose
Experimental
干预措施: Sirolimus (Drug)
Low Dose
Experimental
干预措施: Prednisone (Drug)
High Dose
Experimental
干预措施: Sirolimus (Drug)
High Dose
Experimental
干预措施: Prednisone (Drug)
结局指标
主要结局
Immunogenicity of AAV9 and GCase in blood
时间窗: Up to Year 2
Number of Adverse Events (AEs), Serious Adverse Events (SAEs), and Adverse Events leading to discontinuation
时间窗: Year 5
Immunogenicity of AAV9 and GCase in CSF
时间窗: Up to Year 1
次要结局
- Clinical Global Impressions (Improvement)(Months 6, 12 and up to Year 2)
- Change in adaptive behavior and functioning(Months 6 and 12 and up to Year 2)
- Time to death(Baseline until event or study completion, up to Year 5)
- Time to clinical event(Baseline until event or study completion, up to Year 5)
- Change in most troubling symptoms(Months 6, 12 and up to Year 2)
- Change in glycolipid levels in blood(Up to Year 5)
- Change in glycolipid levels in CSF(Up to Year 3)
- Individual Vector Shedding data(Up to Year 5)
- Change in cognitive function(Study Month 12 and up to Study Year 2)
- Change in behavioral symptoms(Months 6, 12 and up to Year 2)
- Change in GCase (glucocerebrosidase) enzyme activity levels in blood(Up to Year 5)
- Change in GCase enzyme activity levels in CSF (cerebrospinal fluid)(Up to Year 3)
- Change in Clinical Global Impressions (Severity)(Months 6, 12 and up to Year 2)
- Change in motor skills(Months 6, 12 and up to Year 2)
- Change in cognitive function(Months 6,12 and up to Year 2)
研究者
研究点 (11)
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