EUCTR2016-002328-10-IT进行中（未招募）1 期
A Phase I/II Open Label, Dose Escalation, Safety Study in Subjects with Mucopolysaccharidosis type VI (MPS VI) Using Adeno-Associated Viral Vector 8 to Deliver the human ARSB gene to Liver. - TIGEM1-MPSVI
FONDAZIONE TELETHO0 个研究点目标入组 10 人开始时间: 2021年6月17日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1.Subjects must have a documented biochemical and molecular diagnosis of MPS VI.
- •2.Subjects must be 4 years old or older.
- •3.Subjects should have received Enzyme Replacement Therapy (ERT) for at least 12 months before enrolment, and should continue to receive treatment until 7-14 days before IMP administration.
- •4.Documented informed consent; willingness to adhere to protocol and required long-term follow-up as evidenced by written informed consent.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 3
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.Subjects unable or unwilling to meet requirements of the study.
- •2.History of severe anaphylactoid reaction to Naglazyme in subjects receiving ERT that could affect the safety (severe reaction is meant to be an event with respiratory impairment that is life-threatening).
- •3.Serum AST or ALT above the upper limit of normal range at the baseline evaluations (Baseline 2, -5 days).
- •4. Detectable serum neutralizing antibodies (NAB) against AAV8 vector.
研究者
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