Actively Recruiting
A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major
Led by Shanghai BDgene Co., Ltd. · Updated on 2024-06-24
9
Participants Needed
3
Research Sites
151 weeks
Total Duration
On this page
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
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 provided informed consent; participants aged 8 and above are recommended to also provide written consent
- Diagnosed transfusion-dependent beta-thalassemia requiring at least 100 mL/kg of packed red blood cells annually, with genotypes b20/b20, b20/b2+, or b2+/b2+
- Eligible for allogeneic hematopoietic stem cell transplantation but without a donor or refusing transplantation
- Have received symptomatic treatment for at least 2 years with transfusion history records
- Stable condition with proper iron chelation treatment
- Good organ function
- Willingness to comply with visit schedules, trial plans, laboratory tests, and procedures
- Agree to participate in long-term follow-up research
You will not qualify if you...
- Has a fully HLA-matched hematopoietic stem cell donor and is willing to receive transplantation
- Positive for HIV-1/HIV-2 antibodies, Treponema pallidum antibodies, HTLV-1/HTLV-2 antibodies, or VSV-G
- Positive for Hepatitis B surface antigen or DNA, Hepatitis C antibody, Epstein-Barr Virus, or Cytomegalovirus
- Severe active infections (bacterial, viral, fungal, malarial, or parasitic)
- History or presence of malignant, myeloproliferative, or immunodeficiency disorders
- Direct relatives with hereditary cancer syndromes
- Autoimmune diseases causing transfusion difficulties
- Major organ diseases or abnormal lab tests including liver cirrhosis or dysfunction, heart disease or low ejection fraction, kidney disease or poor clearance, endocrine disorders, severe iron overload, significant pulmonary hypertension
- Uncorrected bleeding disorders
- Severe psychiatric disorders
- Low white blood cell count (<3x10^9/L) or low platelet count (<120x10^9/L)
- Received hydroxyurea or 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
- Allergic to investigational drug or excipients
- Other conditions deemed unsuitable by the investigator
AI-Screening
AI-Powered Screening
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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
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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