Actively Recruiting
An Open, Multi-center, Phase I Clinical Study on the Safety and Efficacy of HGI-001 Injection in Patients with Transfusion-Dependent Beta-Thalassemia
Led by Shenzhen Hemogen · Updated on 2024-10-23
8
Participants Needed
3
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This trial investigates the safety and effects of a gene therapy product called HGI-001 in patients with transfusion-dependent beta-thalassemia, a blood disorder requiring frequent blood transfusions. The study is a Phase 1, open-label, single-dose trial conducted across multiple centers. It focuses on using patients' own modified stem cells to restore beta-globin production, which is important for healthy red blood cells. Participants aged 6 to 35 years will have their own hematopoietic stem cells collected and genetically modified with the LentiHBBT87Q system to restore beta-globin expression. After a conditioning treatment, these modified cells are infused back into the patients. The trial involves a single arm where all subjects receive this gene therapy and are followed up for two years. Throughout the study, participants will be regularly monitored with tests including blood transfusion records, imaging scans to assess iron levels in the heart and liver, hematology and bone marrow analyses, and tracking of adverse events. Researchers will measure outcomes such as transfusion independence, survival, engraftment success, and changes in hemoglobin and iron levels. Safety and efficacy data will be collected over 24 months to evaluate the therapy's impact and long-term effects.
CONDITIONS
Brief 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 6-35 years (inclusive) and able to provide informed consent by the patient or legal guardian
- Definitively diagnosed with severe transfusion-dependent beta-thalassemia without genotype restriction (excluding coexisting alpha-thalassemia) with valid test reports
- Average transfusion volume greater than 100 mL/kg/year or transfusion frequency more than 8 times/year within 2 years prior to enrollment
- At least 3 months of full volume transfusion before screening, maintaining hemoglobin level at or above 9.0 g/dL
- Serum ferritin level less than 5000 micrograms per liter, with moderate or lower iron overload in heart and liver by MRI (liver MRI T2* > 1.4ms and cardiac MRI T2* > 10ms)
- Acceptable and stable organ functions (heart, liver, kidney, lung, coagulation) and suitable for busulfan conditioning and stem cell transplantation
- Ability to meet follow-up requirements and regularly return for examinations within 2 years after infusion
You will not qualify if you...
- Having a fully HLA-matched donor
- Previous gene therapy, gene editing therapy, or allogeneic hematopoietic stem cell transplantation
- Uncorrected bleeding disorders
- Uncontrolled epilepsy or mental illness
- Use of certain medications (Luspatercept, Hydroxyurea, Ruxolitinib, Thalidomide, Decitabine, Ara-c) within 3 months prior to enrollment
- Psychoactive substance, drug, or alcohol abuse within 6 months prior to enrollment
- Untreated pulmonary hypertension
- Positive for anti-RBC antibodies
- Positive for hepatitis B surface antigen with high HBV DNA, antibodies to hepatitis C, HIV, or Treponema pallidum, or abnormal viral tests
- History or presence of malignant tumors, myeloproliferative diseases, immunodeficiency, or autoimmune disorders
- First-degree relatives with hereditary cancer history or suspicion
- 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 less than 3 x 10^9/L or platelet count less than 100 x 10^9/L
- Diabetes, abnormal thyroid function, or other endocrine disorders
- Participation in other interventional clinical studies within 4 weeks prior to this trial
- Poor adherence or other conditions judged unsuitable by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
Participants receive busulfan conditioning followed by reinfusion of their own hematopoietic stem cells genetically modified to restore β-globin expression.
1 inpatient visit for conditioning and reinfusion
Duration - Up to 24 months
Participants are monitored for safety, efficacy, and transplant-related outcomes for up to 24 months after reinfusion.
Regular follow-up visits over 24 months
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
L
Linxia Qian
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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