Actively Recruiting
the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia
Led by Shenzhen Hemogen · Updated on 2024-11-29
3
Participants Needed
1
Research Sites
187 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients
CONDITIONS
Official Title
the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 18-35 years (inclusive) with informed consent provided by patient or legal guardian
- Definitive diagnosis of severe transfusion-dependent beta-thalassemia without genotype restriction and valid test report
- Average transfusion volume greater than 100 mL/kg/year or transfusion frequency over 8 times/year in the past 2 years, or confirmed diagnosis of transfusion-dependent beta-thalassemia
- At least 3 months of full volume transfusion prior to screening with hemoglobin maintained at 9.0 g/dL or higher
- Ferritin load less than 3000 mcg/L and moderate or less iron overload in heart and liver; records of iron chelation treatment within 3 months before screening
- Acceptable and stable organ functions (heart, liver, kidney, lung, coagulation) and suitability for busulfan pre-treatment and stem cell transplantation
- Ability to comply with follow-up and treatment schedules and attend hospital visits regularly for 2 years after reinfusion
You will not qualify if you...
- Presence of fully HLA-matched donors
- Previous allogeneic transplantation or other gene therapies
- Prior splenectomy
- Uncorrected bleeding disorders
- Uncontrolled epilepsy or mental illness
- Use of hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months prior to enrollment
- Psychoactive substance, drug, or alcohol abuse within 6 months before enrollment
- Pulmonary hypertension without effective treatment
- Persistent toxicity of grade 2 or higher from previous treatments
- Positive anti-RBC antibody screening
- Positive for hepatitis B surface antigen with high HBV DNA, hepatitis C antibody, HIV, or Treponema pallidum antibody (except vaccination-induced)
- History or presence of malignant tumors, myeloproliferative or immunodeficiency diseases
- Immediate family with or suspected familial cancer
- Severe bacterial, viral, fungal, or parasitic infections
- Severe liver, kidney, or heart disease as defined by specific lab and imaging criteria
- White blood cell count below 3 x 10^9/L or platelet count below 100 x 10^9/L
- Diabetes, abnormal thyroid function, or other endocrine disorders
- Participation in other interventional clinical trials within 4 weeks prior
- Poor adherence or other investigator-judged unsuitability for participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Shenzhen University General Hospital
Shenzhen, Guangdong, China
Actively Recruiting
Research Team
H
Haigang Sun
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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