Status:
RECRUITING
Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients
Lead Sponsor:
Institute of Hematology & Blood Diseases Hospital, China
Collaborating Sponsors:
Kanglin Biotech
Conditions:
Transfusion-dependent Beta-Thalassemia
Eligibility:
All Genders
3-35 years
Phase:
NA
Brief Summary
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 au...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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
- 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
Key Trial Info
Start Date :
January 4 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2026
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT06219239
Start Date
January 4 2024
End Date
December 31 2026
Last Update
August 5 2025
Active Locations (1)
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1
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, China, 300020