Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients
Recruiting now · Not applicable
Conditions studied: Transfusion-dependent Beta-Thalassemia
In brief
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 transfusion-dependent β-thalassemia.
Key facts
- Study ID
- NCT06219239
- Run by
- Institute of Hematology & Blood Diseases Hospital, China
- People needed
- 3
- Starts
- 2024-01-04
- Expected to finish
- 2026-12-31
- Last updated by the study team
- 2025-08-05
Who can join
Age: 3 and older, up to 35. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Male or female age between 3-35 years
- Diagnosis of transfusion-dependent β-thalassemia and a history of at least 100 mL/kg/year of pRBCs or ≥8 transfusions of pRBCs per year for the prior 2 years
- Documented baseline, or pretransfusion, Hb level≤7 g/dL
- Karnofsky performance status ≥70 for subjects≥16 years of age; Lansky performance status of ≥70 for subjects<16 years of age
- Eligible to undergo auto-HSCT
- Willing and able to follow the research procedures and conditions, with good compliance
- Willing to receive at least the 2 years follow-up and maintain detailed medical records, including transfusion history
- Subject and/or legal guardians voluntarily participated in this clinical trial and signed the informed consent form, and can complete all follow-ups in accordance with the protocol requirements
You may not qualify if…
- 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:
- Liver function test suggest AST or ALT levels >3× upper limit of normal (ULN);
- Total serum bilirubin value >2.5×ULN;if combined with Gilbert syndrome, total bilirubin >3×ULN and direct bilirubin value >2.5×ULN;
- History of bridging fibrosis, cirrhosis;
- Left ventricular ejection fraction <45%;
- New York Heart Association (NYHA) class III or IV congestive heart failure;
- Severe arrhythmia requiring medical treatment;
- Uncontrolled hypertension or unstable angina pectoris;
- Myocardial infarction or bypass or stent surgery within 12 months before drug administration;
- Valvular disease with clinical significance;
- Baseline calculated eGFR<60mL/min/1.73m2;
- Pulmonary function: FEV1/FVC<60% and/or diffusion capacity of carbon monoxide (DLco) <60% of prediction;
- Evidence of clinically significant pulmonary hypertension requiring medical intervention.
- 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
Where it is running
- Institute of Hematology & Blood Diseases Hospital — Tianjin, Tianjin Municipality, China (enrolling)
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.