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 - G1XCGD.02

Genethon0 个研究点目标入组 5 人开始时间: 2024年7月2日最近更新: 2024年8月21日

适应症

试验速览

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•a. Male X-CGD patients >23 months of age. Youngest patients (>1 month and = 23 months) may be enrolled at physician’s appreciation; in this case mobilization of peripheral HSC may be replaced by two bone marrow harvests., b. Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or reduction > 70% of the biochemical activity of the NAHPD-oxidase., c. At least one ongoing or resistant or at high risk of relapse severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy., d. 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., e. No co-infection with Human Immunodeficiency Virus (HIV) or hepatitis B virus (HBs Ag positive) or hepatitis C virus (anti-HCV Ab positive)., f. written informed consent for adult patient., g. Parental/guardian and where appropriate child’s signed consent/assent.

•a. 10/10 HLA identical (A, B, C, DR, DQ) family or unrelated., b. Contraindication for leukapheresis (anaemia Hb <8g/dl, cardiovascular instability, severe coagulopathy)., c. Contraindication for administration of conditioning medication and any component of the Investigational Medicinal Product (IMP) preparation., d. Administration of gamma interferon within 30 days before the infusion of transduced autologous CD34+ cells., e. Tested positive (definitive) for the presence of multiple types (2 or more) of anti-platelet antibodies, f. Tested positive (definitive) for the presence of anti-HLA (Class I & II) antibodies., g. Participation in another experimental therapeutic protocol within 6 months prior to baseline and during the study period., h. 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., i. Patient/Parent/Guardian unable or unwilling to comply with the protocol requirements

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