A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human α-L-iduronidase gene for the treatment of patients affected by Mucopolysaccharidosis Type I, Hurler variant

Mucopolysaccharidosis type I Hurler

Overall survival from Advanced Therapy Investigational Medicinal Product (ATIMP) injection, Achievement of hematological engraftment less than or equal to day +45 from Advanced Therapy Investigational Medicinal Product (ATIMP) injection. Hematologic engraftment is defined as the first of 3 consecutive days with neutrophil count > 500/mm3 and platelets > 20,000/mm3 (in the absence of platelet transfusion for seven consecutive days)., Safety of the administration of autologous HSPC transduced with LVV-IDUA. This will be measured as: a) short-term tolerability (0-24 hours from ATIMP injection); b) absence of Replication Competent Lentivirus (RCL) (0-8 years); c) absence of malignancy or abnormal clonal proliferation due to insertional mutagenesis (0-8 years)., Overall safety and tolerability measured by Adverse Event (AE) recording., IDUA activity in blood (dried blood spot, DBS) (up to supraphysiologic levels) at 1-year posttreatment

Achievement of supraphysiologic IDUA activity in blood (DBS), IDUA activity in plasma, Engraftment of transduced cells >= 0.30 vector copy number (VCN)/genome, Normalization of urinary GAGs, Normalization of spleen and liver (for age), Growth velocity treatment, Anti-IDUA antibody immune response before and after infusion of IDUA LVV-transduced cells

No related papers found yet.

Papers published before 2008-02-04 may not appear yet. Historical indexing is in progress.

Italy