A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-121
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 27
- 试验地点
- 5
- 主要终点
- Incidences of Serious Adverse Events over time
研究概览
简要总结
RGX-121-5101 is an observational long-term follow-up study to the RGX-121-101 or RGX-121-1102 studies where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study will evaluate the long-term safety and efficacy of RGX-121.
详细描述
This is a prospective follow-up study to evaluate the long-term safety and efficacy after a single administration of RGX-121. Eligible participants are those who previously have enrolled in clinical study RGX-121-101 or RGX-121-1102 and received a single intracisternal (IC) or intracerebroventricular (ICV) infusion of RGX-121. Enrollment of each participant will occur the same day or after the participant has completed the end of study (EOS) visit or early termination visit (ET) from the previous (parent) study. Participants will be followed in this study cumulatively for up to 5 years after RGX-121 administration (inclusive of the parent study). No treatment will be administered under this observational protocol. The total study duration for each participant may vary depending on when they enroll in the current study following RGX-121 administration in the parent study.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •To be eligible, a participant must have previously received RGX-121 in a separate parent trial.
- •Participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent
排除标准
- •Participant has not received RGX-121 previously in a separate parent trial.
结局指标
主要结局
Incidences of Serious Adverse Events over time
时间窗: 3 year
Safety
Incidences of Adverse Events over time.
时间窗: 3 years
Safety
Long-term Safety of RGX-121
时间窗: 5 years inclusive of parent study
To evaluate the safety of RGX-121 serious and adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE Version 5.0).
次要结局
- Changes in neurodevelopmental parameters of cognitive function [Time Frame: 1 Month, 12 Months, 24 Months, 36 Months](3 years)
- Changes in neurodevelopmental parameters of adaptive behavior function [Time Frame: 1 Month, 12 Months, 24 Months, 36 Months](3 years)
- Biomarkers [Time frame: 1 Month, 12 Months, 24 Months, 36 Months](3 years)
- Evaluate the long-term effect of RGX-121 on neurodevelopmental parameters of cognitive, motor, behavioral, and adaptive function (Bayley Scales of Infant and Toddler Development, 3rd Edition)(5 years inclusive of parent study)
- Evaluate the long-term effect of RGX-121 on neurodevelopmental parameters of cognitive, motor, behavioral, and adaptive function (Kaufman Assessment Battery for Children, 2nd Edition)(5 years inclusive of parent study)
- Evaluate the long-term effect of RGX-121 on neurodevelopmental parameters of cognitive, motor, behavioral, and adaptive function (Mullen Scales of Early Learning)(5 years inclusive of parent study)
- Evaluate the long-term effect of RGX-121 on neurodevelopmental parameters of cognitive, motor, behavioral, and adaptive function (Vineland Adaptive Behavior Scales, 2nd Edition)(5 years inclusive of parent study)
- Evaluate the long-term impact of RGX-121 on Plasma Biomarkers (I2S)(5 years inclusive of parent study)
- Evaluate the long-term impact of RGX-121 on CSF Biomarkers (I2S)(5 years inclusive of parent study)
- Evaluate the long-term impact of RGX-121 on CSF Biomarkers (GAGs)(5 years inclusive of parent study)
- Long-term effect of RGX-121 on Urine Biomarker (GAGs)(5 years inclusive of parent study)