This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.
Diagnosis of Beta Thalathemia.
Age: ≥ 4 years.
Karnofsky: ≥ 80%.
Left ventricular ejection fraction (LVEF): > 50%; no obvious heart disease and pulmonary hypertension.
Diagnosis of active malignant disease (other than Bowen disease or cervical cancer); or has family history of cancer.
Myelopathy, tumor-related cytogenetic changes or other more severe blood diseases.
Has alcoholism experience within 6 months prior to enrollment.
History of epilepsy.
Single group
Gene-modified autologous hematopoeitic stem cells
Genetic1 infusion of 5x10\^6\~1x10\^7 per kilogram body weight gene-modified cells; or more infusions depending on the circumstances
Safety in patients using CTCAE version 4.0 standard to evaluate the level of adverse events
Physiological parameter (measuring cytokine response, fever, symptoms)
Tolerability of transplanted cells that are transduced ex vivo & transplanted in subjects with ß-thalassemia major conditioned with a reduced-intensity non-myeloablative preparative regimen.
Monitoring the following: The occurrence of insertional oncogenesis, which will be investigated by monitoring peripheral blood cell counts \& leukocyte clonality using PCR and sequencing analysis, and qPCR for vector copy number.
Treatment responses
Blood routine indexes will be recorded before and after treatment. Objective response, such as complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) will be assessed.
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This trial is not recruiting at the moment. You can still explore other options:
