Actively Recruiting
Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
Led by Shenzhen Hemogen · Updated on 2024-10-23
8
Participants Needed
3
Research Sites
133 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia. The study aims to evaluate the safety and efficacy of autologous lentiviral-transduced CD34+ human hematopoietic stem cells (hHSCs) using the gene therapy product HGI-001.
CONDITIONS
Official Title
Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 6 to 35 years and able to provide informed consent
- Diagnosed with severe transfusion-dependent beta-thalassemia without coexisting alpha-thalassemia, with valid test reports
- History of average transfusion volume over 100 mL/kg/year or more than 8 transfusions per year in the past 2 years
- Received full volume transfusions for at least 3 months before screening, maintaining hemoglobin at 9.0 g/dL or higher
- Serum ferritin less than 5000 µg/L and moderate or lower iron overload in heart and liver as shown by MRI
- Acceptable heart, liver, kidney, lung, and coagulation functions; stable condition; suitable for busulfan pre-treatment and stem cell transplantation
- Able to follow treatment and attend regular hospital visits for 2 years after reinfusion
You will not qualify if you...
- Having a fully HLA-matched donor
- Prior gene therapy, gene editing therapy, or allogeneic hematopoietic stem cell transplantation
- Uncorrected bleeding disorders
- Uncontrolled epilepsy or mental illness
- Use of specific drugs (Luspatercept, Hydroxyurea, Ruxolitinib, Thalidomide, Decitabine, Ara-c) within 3 months before enrollment
- Substance or alcohol abuse within 6 months before enrollment
- Pulmonary hypertension without effective treatment
- Positive anti-RBC antibodies
- Positive tests for Hepatitis B (with high HBV DNA), Hepatitis C, HIV, or Treponema pallidum (except from vaccination); abnormal viral DNA/RNA tests
- History of cancer, myeloproliferative, immunodeficiency, or autoimmune diseases
- First-degree relative with hereditary cancer history
- Severe infections
- Severe liver, kidney, or heart disease as defined by lab and imaging criteria
- Low white blood cell or platelet counts
- Diabetes, thyroid dysfunction, or other endocrine disorders
- Participation in other interventional clinical trials within 4 weeks
- Poor adherence or other conditions making participation unsuitable as judged by investigator
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 3 locations
1
Guangxi Medical University First Affiliated Hospital
Guangxi, China
Actively Recruiting
2
Shenzhen Children's Hospital
Shenzhen, China
Actively Recruiting
3
Shenzhen University General Hospital
Shenzhen, China
Actively Recruiting
Research Team
H
Haigang Sun
CONTACT
L
Linxia Qian
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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