Actively Recruiting
Safety and Efficacy of Lentiviral Vector Transduction of Beta-globin Genetically Modified Autologous CD34+ Hematopoietic Stem Cells in Patients With Transfusion-dependent Beta-thalassemia
Led by Shanghai BDgene Co., Ltd. · Updated on 2024-06-14
10
Participants Needed
1
Research Sites
30 weeks
Total Duration
On this page
Sponsors
S
Shanghai BDgene Co., Ltd.
Lead Sponsor
S
Shanghai Children's Medical Center
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and engraftment effectiveness of a gene therapy called BD211 in children and adolescents aged 3 to 18 years with transfusion-dependent beta-thalassemia. This condition requires regular blood transfusions due to a genetic disorder affecting hemoglobin production. The study aims to assess if BD211 can help patients achieve transfusion independence while monitoring safety and treatment effects over 24 months. Participants receive a single intravenous infusion of BD211, which involves genetically modifying their own CD34+ hematopoietic stem cells with a lentiviral vector encoding the human betaA-T87Q-globin gene. The dose ranges from 5 to 10 million cells per kilogram of body weight. The treatment is given once, followed by a total follow-up period of 24 months to monitor safety and effectiveness. During the study, participants will undergo regular assessments to measure hemoglobin levels, transfusion independence, iron overload, growth and development, quality of life, and possible adverse events. Researchers will also evaluate neutrophil and platelet engraftment, transplant-related mortality, overall survival, and potential genetic changes related to the therapy. The study includes comprehensive medical evaluations and monitoring throughout the two-year follow-up period.
CONDITIONS
Brief Title
Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ages 3 to 18 years old
- Parents or legal guardians able to understand and provide informed consent; children aged 8 years and older encouraged to participate in consent
- Diagnosed with transfusion-dependent beta-thalassemia of any genotype confirmed by hemoglobin analysis
- No alpha chain genetic abnormalities
- Stabilized and maintained on an appropriate iron chelation regimen
- Requires at least 100 mL/kg/year of red blood cell transfusions
- Not eligible for allogeneic hematopoietic stem cell transplantation
- Unable to financially support luspatercept treatment
- Parents or guardians willing and able to follow study procedures
- Good organ function
- Complete medical records including at least two years of blood transfusion history and follow-up prior to screening
You will not qualify if you...
- Availability of voluntary, fully HLA-matched hematopoietic cell donors unless recommended for inclusion
- Positive for HIV-1, HIV-2, HTLV-1, HTLV-2, or VSV-G antibodies
- Active bacterial, viral, fungal, or parasitic infection
- Contraindications for bone marrow extraction under anesthesia
- History or presence of malignancy, myeloproliferative, or immunodeficient diseases
- 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 prior to stem cell collection
- Immediate family history of certain familial cancer syndromes
- Major mental illness impairing study participation
- Active recurrent malaria
- Autoimmune diseases complicating blood transfusions
- Major organ injuries including severe liver, heart, kidney disease or iron overload
- Untreated bleeding disorders
- Participation in another clinical study within 30 days before screening
- Allergy to the study drug or its components
- Prior gene or cell therapy treatments
- Inability to comply with study procedures
- Hydroxyurea treatment within 3 months prior to stem cell collection
- Diseases interfering with stem cell collection
- Other conditions deemed ineligible by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single dose
Participants receive a single intravenous dose of genetically modified autologous hematopoietic stem cells (BD211) designed to produce healthy β-globin in red blood cells.
1 treatment visit (in-person)
Duration - Up to 24 months
Participants are monitored for safety and efficacy outcomes including transfusion independence, blood parameters, growth, quality of life, and adverse events for up to 24 months after treatment.
Regular visits during follow-up period (exact number not specified)
Trial Site Locations
Total: 1 location
1
Shanghai Children's Medical Centre
Shanghai, China, 200127
Actively Recruiting
Research Team
C
Chen Jing, M.D.
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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