Actively Recruiting
Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent Beta-Thalassemia (Child)
Led by Shenzhen Hemogen · Updated on 2024-11-29
3
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This trial investigates the safety and effectiveness of using beta-globin restored autologous hematopoietic stem cells in patients with transfusion-dependent beta-thalassemia major. The study focuses on patients aged 18 to 35 who require frequent blood transfusions due to this genetic blood disorder. Researchers aim to evaluate how this gene-modified stem cell therapy impacts disease outcomes and patient health over time. Participants will have their own hematopoietic stem cells collected and modified with the LentiHBBT87Q system to restore beta-globin production. Following a conditioning regimen, these modified cells will be reinfused back into the patient. The study is open-label and includes a two-year follow-up period to monitor how the treatment works and any effects it may have. Throughout the study, participants will undergo various assessments including monitoring for adverse events, survival rates, and changes in transfusion needs. Researchers will also evaluate hematology, bone marrow health, viral integration, iron levels in the heart and liver, and other markers related to treatment response. Participants are expected to attend regular visits over two years for these evaluations and to support adherence to the treatment and follow-up schedule.
CONDITIONS
Brief 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 to 35 years (inclusive) and able to provide informed consent
- Definitive diagnosis of severe transfusion-dependent beta-thalassemia major without genotype restriction
- Average transfusion volume greater than 100 mL/kg/year or transfusion frequency more than 8 times/year within 2 years before enrollment
- At least 3 months of full volume transfusion prior to screening with hemoglobin maintained at 9.0 g/dL or higher
- Ferritin level below 3000 µg/L with moderate or less iron overload in heart and liver
- Acceptable heart, liver, kidney, lung, and coagulation functions and suitable for busulfan pre-treatment and stem cell transplantation
- Willing and able to follow up regularly and attend hospital visits for examinations for 2 years after reinfusion
You will not qualify if you...
- Having a fully HLA-matched donor
- Previous allogeneic transplantation or other gene therapy treatments
- History of splenectomy
- Uncorrected bleeding disorders
- Uncontrolled epilepsy or mental illness
- Use of hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months before enrollment
- Substance abuse (drugs or alcohol) within 6 months before enrollment
- Pulmonary hypertension without effective intervention
- Persistent toxicity from previous treatments at or above grade 2
- Positive antibody screening for red blood cells
- Positive for hepatitis B surface antigen with active viral load, hepatitis C antibody, HIV, or syphilis antibody (except vaccination-related)
- History or presence of malignant tumors, myeloproliferative, or immunodeficiency diseases
- Immediate family history of familial cancer
- Severe infections (bacterial, viral, fungal, or parasitic)
- Severe liver, kidney, or heart disease as defined by specific laboratory and imaging criteria
- Low white blood cell or platelet counts below specified thresholds
- Diabetes, thyroid dysfunction, or other endocrine disorders
- Participation in other interventional studies within 4 weeks before this trial
- Poor adherence or other conditions judged unsuitable by the investigator
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single treatment event with follow-up starting immediately after reinfusion
Participants receive conditioning followed by reinfusion of their own hematopoietic stem cells modified to restore β-globin expression.
1 treatment visit (in-person)
Duration - Up to 24 months
Participants are followed for two years to monitor safety, treatment response, and health outcomes after reinfusion.
Regular hospital visits for examinations during 2 years after reinfusion
Trial Site Locations
Total: 1 location
1
Shenzhen University General Hospital
Shenzhen, Guangdong, China
Actively Recruiting
Research Team
H
Haigang Sun
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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