Phase I/II clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome

Genethon (France)0 个研究点目标入组 5 人开始时间: 2011年5月20日最近更新: 2019年5月5日

适应症

试验速览

•1. Males of all ages
•2. Severe WAS (clinical score 3 ? 5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
•3. Molecular confirmation by WAS gene DNA sequencing
•4. Unless desease severity indicates that one cannot wait for 3 months (score 5; refractory thrombocytopenia with platelets < 5000 with bleeding or severe autoimmunity)
•5. Lack of HLA-genotypically identical bone marrow after 3 month search
•6. Lack of a 10/10 or 9/10 antigen HLA-matched unrelated donor after 3 month search
•7. Lack of a HLA-matched cord blood after 3 month search
•8. Parental, guardian, patient signed informed consent/assessment
•9. Willing to return for follow-up during the 2 year study and lifelong for off study review
•10. Only for patients who have received previous allogenic haematopoietic stem cell transplant
•10.1. Failed allogenic haematopoietic stem cell transplant
•10.2. Contraindication to repeat allogenic transplantation

•1. Patient with HLA-genotypically identical bone marrow
•2. Patient with 10/10 or 9/10 antigen HLA-matched unrelated donor or with HLA-matched cord blood
•3. Contraindication to leukapheresis
•3.1. Anaemia (Hb < 8g/dl)
•3.2. Severe vascularitis
•3.3. Refractory thrompopenia
•3.3.1. Contraindication to bone marrow harvest
•3.3.2. Contraindication to administration of conditioning medication
•4. Human immunodeficiency virus (HIV) seropositive patient

发起方

Genethon (France)