Actively Recruiting
Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-08-05
3
Participants Needed
1
Research Sites
156 weeks
Total Duration
On this page
Sponsors
I
Institute of Hematology & Blood Diseases Hospital, China
Lead Sponsor
K
Kanglin Biotech
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged between 3 and 35 years
- Diagnosis of transfusion-dependent beta-thalassemia with a history of at least 100 mL/kg/year of packed red blood cells or 8 or more transfusions per year for the prior 2 years
- Documented baseline or pretransfusion hemoglobin level of 7 g/dL or less
- Karnofsky performance status of 70 or higher for subjects aged 16 years or older; Lansky performance status of 70 or higher for subjects younger than 16 years
- Eligible to undergo autologous hematopoietic stem cell transplant
- Willing and able to follow research procedures with good compliance
- Willing to receive at least 2 years of follow-up and maintain detailed medical records, including transfusion history
- Participant and/or legal guardians voluntarily agree and signed informed consent and can complete all follow-ups as required
You will not qualify if you...
- Clear contraindications for hematopoietic stem cell collection
- Diagnosis of composite alpha thalassemia
- White blood cell count less than 3 x 10^9/L and/or platelet count less than 100 x 10^9/L not due to hypersplenism
- Severe iron overload at screening shown by MRI, serum ferritin 5000 ng/mL or higher, or moderate to severe heart iron overload
- Any prior or current malignancy, myeloproliferative disorder, or significant immunodeficiency
- Eligible for allogeneic stem cell transplant with a willing full HLA match donor
- Prior gene therapy or allogeneic hematopoietic stem cell transplant
- Severe active infections including hepatitis B or C, HIV, active syphilis, or tuberculosis
- Immediate family member with known familial cancer syndrome
- Significant psychiatric disorder impairing study participation
- History of major organ damage including severe liver, heart, kidney, or lung conditions as specified
- Uncorrectable coagulation problems or history of severe bleeding
- Any other condition making the subject ineligible for transplant as determined by the physician
- Known allergy to trial drugs or ingredients such as plerixafor, G-CSF, busulfan, or DMSO
- Participation in other clinical trials within 3 months prior to screening
- Live vaccine received within 6 weeks prior to screening
- Pregnancy, breastfeeding, or inability to use effective contraception during the 27-month study
- Use of hydroxyurea, thalidomide, or hypomethylating drugs within 3 months before stem cell collection
- Other investigator-determined ineligibility reasons
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, China, 300020
Actively Recruiting
Research Team
J
Jun Shi, PhD
CONTACT
Z
Zhen Gao, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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