Actively Recruiting
Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia
Led by Shanghai BDgene Co., Ltd. · Updated on 2024-06-14
10
Participants Needed
1
Research Sites
158 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
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 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 or older encouraged to participate in consent process
- Diagnosed with transfusion-dependent β-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 packed red blood cells
- Not eligible for allogeneic hematopoietic stem cell transplantation
- Treatment with erythrocyte maturation agent luspatercept not financially supported
- Parents or legal 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 donor 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
- Contraindicated for bone marrow extraction under anesthesia
- Any malignancy, myeloproliferative, or immunodeficient disease or relevant medical history
- Peripheral blood white blood cell count below 3×10^9/L or platelet count below 120×10^9/L
- History of allo-transplantation
- Use of erythropoietin within 3 months prior to stem cell collection
- Immediate family history of known or suspected familial cancer syndromes
- Diagnosis of major mental illness causing serious disability for study participation
- Active recurrent malaria
- Autoimmune diseases complicating blood transfusions
- Major organ injury including liver disease with elevated transaminases, bridging fibrosis, cirrhosis, heart disease with ejection fraction less than 25%, kidney disease with creatinine clearance below 30%, severe iron overload, heart MRI T2* less than 10 ms, or significant pulmonary hypertension requiring intervention
- Uncured bleeding disorders
- Participation in another clinical study within 30 days prior to screening
- Allergy to study drug or its excipients
- Prior gene or cell therapy treatment
- Inability of participant or parents to comply with study procedures
- Hydroxyurea treatment within 3 months prior to stem cell collection
- Diseases interfering with stem cell collection
- Any other condition making participant ineligible for stem cell transplantation as determined by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Shanghai Children's Medical Centre
Shanghai, China, 200127
Actively Recruiting
Research Team
C
Chen Jing, 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
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here