跳至主要内容
临床试验/CTIS2024-512680-31-00
CTIS2024-512680-31-00招募中1 期

ong term safety follow up of patients enrolled in the phase I/II clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome (GTG002-07 and GTG003-08) - GNT-WAS-03

Genethon0 个研究点目标入组 10 人开始时间: 2024年6月28日最近更新:
适应症

试验速览

阶段
1 期
状态
招募中
发起方
Genethon
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

入排标准

年龄范围
0 至 64(—)
性别
Male

入选标准

  • Patients enrolled in the phase I/II studies and treated by a single infusion of autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector for WAS conducted in France and United Kingdom (GTG002.07 and GTG003. 08), Parents, guardians or patient signed informed consent.

排除标准

  • Parents, guardians, patients unwilling to return for the follow up study period.

研究者

发起方
Genethon

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