A Long-term Follow-up Study for Subjects Previously Treated with Autologous ex vivo Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS)

Wiskott-Aldrich syndrome (WAS)

To characterize the long-term safety of the gene therapy treatment with Telethon003 as measured by: recording of adverse event (AE) and serious adverse event (SAE) related to gene therapy including: − insertional mutagenesis and oncogenesis (blood and solid malignancies); − transgene immunogenicity; − development of replication-competent lentiviruses (RCL);, To evaluate the overall survival (OS) as primary efficacy endpoint up to 15 years of follow-up post treatment with Telethon003

Event free survival, Annualized rate of severe infections compared with 1 year prior to gene therapy;, Annualized rate of moderate and severe bleeding episodes compared with 1 year prior to gene therapy, Eczema, Autoimmunity, Hematologic disorders (i.e. neutropenia and/or other cytopenias);, Malignancies, Use of IgRT, New or exacerbations of pre-existing neurological disorders, Variation in height and weight (for paediatric patients), Karnofsky/Lansky performance scales, Platelet count and MPV, Vector Copy Number (VCN), Insertion site analysis (ISA), PedsQL (Pediatric Quality of Life Inventory) health-related quality of life scales, EQ-5D, and SF-10 or SF-36 health-related quality of life questionnaires

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