Beta-Thalassemia Clinical Trial
Official title:
A Phase I/II Study Evaluating Safety and Efficacy of Autologous Hematopoietic Stem Cells Genetically Modified With GLOBE Lentiviral Vector Encoding for the Human Beta-globin Gene for the Treatment of Patients Affected by Transfusion Dependent Beta-thalassemia
This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by transfusion dependent beta-thalassemia
Both adults and pediatric patients will be treated with genetically modified autologous
hematopoietic stem cells collected from mobilized peripheral blood (or bone marrow for
patients < 8 years in case mobilization will not be feasible) and transduced with GLOBE
lentiviral vector encoding for the human beta-globin gene.
This study will enroll 10 patients allocated in 3 groups, according to age and conditioning
regimen:
1. 3 adults (≥18 years) conditioned with treosulfan and thiotepa
2. 3 elderly children (8-17 years) conditioned with treosulfan and thiotepa
3. 4 younger children (3-7 years) conditioned with treosulfan and thiotepa Enrolment will
start in adult patients. Pediatric patients will be included once evidence of
preliminary safety and biological efficacy is shown in at least 2 adults.
Patients are included regardless of the beta globin gene mutation, provided an adequate
cardiac, renal, hepatic and pulmonary function is demonstrated. Patients with severe iron
overload are excluded as well as patients with active viral infections. Pediatric patients
can be enrolled only in absence of a human leukocyte antigen (HLA)-identical sibling or a
suitable 10/10 matched unrelated donor.
The treated patients will be followed for 2 years. After completion of the 2 years follow up,
patients will be enrolled in a long term follow up study and followed up for at least other
additional 6 years.
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