Actively Recruiting
A Phase 1 Clinical Trial of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Intravenous Infusion for Transfusion-dependent Beta-thalassemia Patients
Led by Shanghai BDgene Co., Ltd. · Updated on 2024-06-24
9
Participants Needed
3
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and effectiveness of a gene therapy treatment called BD211 for patients aged 3 to 35 years with transfusion-dependent beta-thalassemia. This condition requires regular blood transfusions, and the study aims to assess how well the gene-modified stem cells work and how safely they can be used. The study is a Phase 1, open-label trial sponsored by Shanghai BDgene Co., Ltd., focusing on patients who need frequent transfusions and have limited treatment options. The treatment involves a single intravenous infusion of BD211, which consists of autologous CD34+ hematopoietic stem cells that have been genetically modified with a lentiviral vector to produce healthy beta-globin. Participants receive one dose of at least 5 million cells per kilogram of body weight. The study includes an 18-month follow-up period to monitor safety endpoints and effectiveness, including the ability to reduce or eliminate the need for transfusions. During the study, participants undergo regular assessments to track neutrophil and platelet engraftment, transfusion independence, blood hemoglobin levels, ferritin levels, and the presence of the beta-globin protein in blood. Researchers also monitor for adverse events, survival rates, hospitalizations, and any signs of complications related to the gene therapy. Participants are expected to comply with scheduled visits, laboratory tests, and long-term follow-up assessments to help determine the treatment's overall safety and efficacy.
CONDITIONS
Brief Title
A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged 3 years (inclusive) to 18 years (exclusive) with no gender restrictions
- Parents or legal guardians have signed informed consent; children aged 8 and above are encouraged to provide written consent
- Transfusion-dependent beta-thalassemia patients requiring at least 100 mL/kg of packed red blood cells annually
- Eligible for allogeneic stem cell transplantation but without a donor or refusing transplantation
- Have had symptomatic treatment for at least 2 years with transfusion history records
- Stable condition with appropriate iron chelation treatment
- Good organ function status
- Good compliance and willingness to follow visit schedules, trial plans, and procedures
- Willing to participate in long-term follow-up research
You will not qualify if you...
- Having a fully HLA-matched stem cell donor and willing to receive transplantation unless reviewed by safety committee
- Positive for HIV, syphilis, HTLV, or VSV-G antibodies
- Positive for hepatitis B, hepatitis C, EBV, or CMV infections
- Severe active bacterial, viral, fungal, malarial, or parasitic infections
- History or presence of malignancy, myeloproliferative, or immunodeficiency disorders
- First-degree relatives with known or suspected hereditary cancer syndromes
- Autoimmune diseases causing transfusion difficulties
- Major organ diseases or abnormal lab tests including liver cirrhosis, heart disease with LVEF < 60%, kidney disease, endocrine disorders, severe iron overload, pulmonary hypertension
- Uncorrected bleeding disorders
- Severe psychiatric disorders
- Low white blood cell or platelet counts
- Hydroxyurea treatment within last 3 months before stem cell collection
- Use of erythropoiesis-stimulating agents within 3 months prior to stem cell collection
- History of allogeneic transplantation
- Previous gene or cell therapy
- Participation in another clinical trial within 30 days
- Contraindications to anesthesia or stem cell collection
- Allergy to investigational drug or excipients
- Any other conditions deemed unsuitable 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 dosing with follow-up to 18 months
Participants receive a single intravenous infusion of gene-modified autologous hematopoietic stem cells (BD211) designed to produce healthy β-globin in red blood cells.
Multiple visits for assessments during the 18-month follow-up period
Duration - Up to 18 months after treatment
Participants are monitored for safety, treatment effectiveness, and transfusion independence after receiving BD211.
Regular visits over 18 months for monitoring and assessments
Trial Site Locations
Total: 3 locations
1
Sun Yat-sen Memorial Hospital
Guangzhou, Guandong, China, 510120
Actively Recruiting
2
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China, 530021
Actively Recruiting
3
Shanghai Ruijin Hospital, Shanghai Jiaotong University
Shanghai, Shanghai City, China, 200025
Actively Recruiting
Research Team
F
fujun Li
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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