A phase I/II, non randomized, multicenter, open-label study of autologous CD34+ cells transduced with the G1XCGD Lentiviral vector in patients withX-Linked Chronic Granulomatous Disease - Phase I/II G1XCGD.01 study : an ex-vivo gene therapy for X-CGD patients

Genethon0 个研究点目标入组 11 人开始时间: 2012年7月19日最近更新: 2016年11月14日

试验速览

暂无简介。

•- Male X-CGD patients > 6 months of age
•-Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or significantly reduced biochemical activity of the NADPH-oxidase
•-At least one prior, ongoing or resistant severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy
•- No HLA-matched donor available after 3 months search unless the risk of waiting for a potential match or for performing an allogeneic transplant is considered unacceptable
•-No co-infection with Human Immunodeficiency Virus (HIV) or hepatitis B virus (HBsAg positive) or hepatitis C virus (HCV RNA positive)
•-Written informed consent for adult patient
•-Parental/guardian and where appropriate child’s signed consent/assent
•- If applicable, patients must agree to use appropriate medically approved contraception during the trial and for 1 month afterwards
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 8
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 2
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 0

•-10/10 HLA identical (A,B,C,DR,DQ) family or unrelated or cord blood donor unless there is deemed to be an unacceptable risk associated with an allogeneic procedure
•-Contraindication for leukapheresis (anaemia Hb <8g/dl, cardiovascular instability, severe coagulopathy)
•-Contraindication for administration of conditioning medication and any component of the Investigational Medicinal Product (IMP) preparation
•-Administration of gammainterferon within 30 days before the infusion of transduced autologous CD34+ cells
•-Participation in another experimental therapeutic protocol within 6 months prior to baseline and during the study period
•-Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study
•-Patient/Parent/Guardian unable or unwilling to comply with the protocol requirements

发起方