Actively Recruiting
the Safety and Efficacy Evaluation of HGI-002 Injection in Patients With Transfusion-Dependent α-Thalassemia
Led by Shenzhen Hemogen · Updated on 2024-11-29
3
Participants Needed
1
Research Sites
220 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 α-globin Restored Autologous Hematopoietic Stem Cells in α-Thalassemia Major Patients
CONDITIONS
Official Title
the Safety and Efficacy Evaluation of HGI-002 Injection in Patients With Transfusion-Dependent α-Thalassemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 12 to 35 years and able to provide informed consent
- Definitive diagnosis of severe transfusion-dependent alpha-thalassemia without genotype restriction
- Average transfusion volume greater than 100 mL/kg/year or transfusion frequency over 8 times/year in the past 2 years
- At least 3 months of full volume transfusion before 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
- Acceptable and stable organ functions suitable for busulfan pre-treatment and stem cell transplantation
- Willing and able to follow up with regular hospital visits and examinations for 2 years after reinfusion
You will not qualify if you...
- Patients with fully HLA-matched donors
- Prior allogeneic transplantation or other gene therapies
- History of splenectomy
- Uncorrected bleeding disorders
- Uncontrolled epilepsy or mental illness
- Use of hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months before enrollment
- Psychoactive substance, drug, or alcohol abuse within 6 months before enrollment
- Pulmonary hypertension without effective intervention
- Persistent toxicity of grade 2 or higher from previous treatments
- Positive for anti-RBC antibodies
- Positive for hepatitis B surface antigen with high HBV DNA, hepatitis C antibody, HIV, or Treponema pallidum antibody (except vaccine-related)
- History or presence of malignant tumors, myeloproliferative or immunodeficiency diseases
- Immediate family history or suspicion of familial cancer
- Severe bacterial, viral, fungal, or parasitic infections
- Severe liver, kidney, or heart disease
- 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 before this trial
- Poor adherence or other conditions making participation unsuitable as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
PLA Joint Logistic Support Force No. 923 Hospital
Nanning, Guangxi, 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
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