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This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells transfusion in subjects with β-thalassemia major.
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Inclusion criteria
Exclusion criteria
Presence of clear contraindications for hematopoietic stem cell collection
Diagnosis of composite α thalassemia
A white blood cell (WBC) count <3×10^9/L, and/or platelet count <100×10^9/L not related to hypersplenism
Subjects with severe iron overload at the time of screening: severe iron overload of the liver showed by MRI, serum ferritin ≥ 5000 ng/mL, or moderate to severe iron overload of the heart
Any prior or current malignancy or myeloproliferative or significant immunodeficiency disorder
Meet the criteria for allo-HSCT and with an identified willing donor with a full HLA match
Prior receipt of gene therapy or allo-HSCT
Subjects with any severe active fungal, bacterial, viral, tuberculosis or other infection, including active hepatitis B (defined as serum HBV-DNA ≥2000 IU/ml), active hepatitis C virus, HCV) infection, human immunodeficiency virus (HIV) antibody-positive or active syphilis patients, etc.
Immediate family member (i.e. parent or siblings) with a known Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome and familial adenomatous polyposis)
Diagnosis of a significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study
History of major organ damage including:
Uncorrectable coagulation dysfunction or history of severe bleeding disorder
Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician
Known allergy to clinical trial drug (plerixafor or G-CSF or busulfan) or ingredient(DMSO etc.)
Participated in other clinical studies within 3 months prior to screening
Inoculated live vaccine within 6 weeks prior to screening
Pregnancy or breastfeeding women; Subjects or their sexual partners were unable to take medically recognized effective contraceptive measures during the 27-month study period
The subjects or their parents would not comply with the study procedures outlined in the protocol
The subjects received hydroxyurea or thalidomide or hypomethylating drugs within 3 months before hematopoietic stem cell collection
Patients considered to be ineligible for the study by the investigator for reasons other than the above
Primary purpose
Allocation
Interventional model
Masking
3 participants in 1 patient group
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Central trial contact
Zhen Gao, MD; Jun Shi, PhD
Data sourced from clinicaltrials.gov
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