A Long-term Follow-up Study to Evaluate the Safety of RGX-111
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 7
- 试验地点
- 2
- 主要终点
- Evaluation of the long-term safety of RGX-111
研究概览
简要总结
RGX-111-5101 is a long-term follow up study that evaluates the long term safety of RGX-111 in participants who have received RGX-111 (a gene therapy intended to deliver a functional copy of the alpha-L-iduronidase gene (IDUA) to the central nervous system) in a separate parent study.
详细描述
This is a prospective, multicenter, observational, follow-up study to evaluate the long-term safety of RGX-111 after a single administration. Eligible participants are those who previously participated in a clinical study where they received a single IC or ICV administration of RGX-111. Enrollment of each participant in this study should occur on the same day or after the participant has completed the end of study (EOS) visit or early discontinuation visit (EDV) from a previous (parent) clinical study. Participants will be followed for up to five years after RGX-111 administration (inclusive of the parent study) or until RGX-111 is commercially available in the participant's country, whichever occurs first. No treatment will be directed under this observational protocol. The total study duration for each participant may vary depending on when s/he enrolls in the current study following RGX-111 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
- All
- 接受健康志愿者
- 否
入选标准
- •To be eligible, a participant must have previously received RGX-111 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
- 未提供
结局指标
主要结局
Evaluation of the long-term safety of RGX-111
时间窗: 5 years inclusive of parent study
Incidences of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) over time
次要结局
- Safety and Efficacy: Change from baseline in neurodevelopmental parameters(5 years inclusive of parent study)
