Actively Recruiting
Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia
Led by Shanghai BDgene Co., Ltd. · Updated on 2024-06-14
10
Participants Needed
1
Research Sites
164 weeks
Total Duration
On this page
Sponsors
S
Shanghai BDgene Co., Ltd.
Lead Sponsor
R
Ruijin Hospital
Collaborating Sponsor
AI-Summary
What this Trial Is About
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.
CONDITIONS
Official Title
Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 6 to 35 years
- Able to provide informed consent
- Diagnosed with transfusion-dependent beta-thalassemia of any genotype confirmed by hemoglobin analysis
- No alpha chain genetic abnormalities
- Stabilized and maintained on appropriate iron chelation therapy
- Requires at least 100 mL/kg/year of red blood cell transfusions
- Tumor gene panel shows no abnormalities related to acute leukemia or myeloid tumors
- Candidates for HLA semi-compatible hematopoietic stem cell transplantation
- Not eligible for allogeneic hematopoietic stem cell transplantation
- Cannot financially support treatment with erythrocyte maturation agent luspatercept
- Willing to follow study procedures
- Complete medical records including at least two years of blood transfusion history and follow-up
You will not qualify if you...
- Availability of fully HLA-matched hematopoietic cell donors unless recommended by Monitoring Committee
- Positive for HIV-1, HIV-2, HTLV-1, HTLV-2, or VSV-G antibodies
- Active bacterial, viral, fungal, or parasitic infection
- Contraindication to bone marrow extraction under anesthesia
- Any malignancy, myeloproliferative, or immunodeficiency disease or relevant medical history
- Peripheral blood white blood cell count less than 3 x 10^9/L or platelet count less than 120 x 10^9/L
- History of allogeneic transplantation
- Use of erythropoietin within 3 months before stem cell collection
- Immediate family history of known or suspected familial cancer syndromes
- Diagnosis of major mental illness causing serious disability to participate
- Active recurrent malaria
- Pregnant, postpartum nursing, or unable to use contraception
- History of major organ injury including severe liver, heart, kidney disease, severe iron overload, significant pulmonary hypertension
- Any other condition making the participant ineligible for stem cell transplantation
- Participation in another clinical study within 30 days before screening
- Expectation to become a parent during the 27-month study period
- Prior gene or cell therapy treatment
- Inability to comply with study procedures
- Hydroxyurea treatment within 3 months before stem cell collection
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Shanghai Ruijin Hospital
Shanghai, China, 200025
Actively Recruiting
Research Team
S
Sujiang Zhang, M.D.
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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