A phase I/II, non-randomized, monocentric, open-label study of autologous CD34+ cells transduced with the G1XCGD lentiviral vector in patients with X-Linked Chronic Granulomatous Disease. - An ex vivo gene therapy study for X-CGD patients

Genethon0 个研究点目标入组 5 人开始时间: 2015年6月26日最近更新: 2024年7月22日

适应症

试验速览

暂无简介。

•-Male X-CGD patients >23 months of age. Youngest patients (>1 month and = 23 months) may be enrolled at physician’s appreciation
•-Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or reduction > 70% of the biochemical activity of the NAHPD-oxidase
•-At least one ongoing or resistant or at high risk of relapse 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 (HBs Ag positive) or hepatitis C virus (HCV RNA positive)
•-Written informed consent for adult patient
•-Parental/guardian and where appropriate child’s signed consent/assent
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 4
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 1
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•-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 gamma interferon 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