CTIS2022-501526-38-00招募中1 期
ong-Term Follow-Up (LTFU) for Gene Therapy of Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD - RP-L301-0222-LTF
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 64（—）
- 性别
- All
入选标准
- •Patient was enrolled in the parent Study RP-L301-0119, Patient received an autologous infusion of CD34+ enriched cells transduced ex vivo with a lentiviral vector carrying the coRPK gene, Patient is willing and able to adhere to the study visit schedule and other protocol requirements, Patient provided written informed consent and, as applicable, assent to participate in the current study in accordance with current regulatory requirements
排除标准
- •There are no exclusion criteria for the current study
研究者
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