This phase I/II trial clinical trial will investigate safety and efficacy of the GLOBE lentiviral vector, which encodes the human β-globin under control of native promoter and control elements. Preclinical studies by the G. Ferrari group (TIGET, Ospedale San Raffale) have given extremely encouraging results for the GLOBE vector in mouse models of β-thalassaemia (see here) and in human patient-derived cells (see here). The study is open to patients aged 3 to 64 years with transfusion-dependent β-thalassaemia and is expected to enrol ten patients over a time period of two years. The detailed study description provides extensive inclusion and exclusion criteria for potential participants in this study.

More information: clinicaltrials.gov, ITHANET Clinical Trials