跳至主要内容
临床试验/NCT06103487
NCT06103487Enrolling By Invitation不适用

A Long-term Follow-up Study to Evaluate the Safety of RGX-111

REGENXBIO Inc.2 个研究点 分布在 2 个国家目标入组 7 人开始时间: 2023年7月24日最近更新:
适应症

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

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)

研究者

申办方类型
Industry
责任方
Sponsor

研究点 (2)

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