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Tests treatment safety and results for β-thalassemia
Official title the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia
ClinicalTrials.gov ID: NCT05864170
What this study is testing
What is β-globin restored autologous hematopoietic stem cells?
β-globin restored autologous hematopoietic stem cells is an investigational medicine, being studied as a potential treatment for β-thalassemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 35
You may be able to join if
- Aged 18-35 years (inclusive), ICF can be provided by the patient and/or legal guardian;
- Definitively diagnosed with severe TDT without genotype restriction, and a valid test report can be provided;
- Average transfusion volume \> 100 mL/kg/year or transfusion frequency \> 8 times/year within 2 years prior to enrollment, or has been definitively...
- At least 3 months of full volume transfusion (verification of blood transfusion records can be provided) prior to screening, and Hb is maintained at...
- Ferritin load \< 3000 μg/L, cardiac and liver iron indicates moderate or lesser iron overload; records of iron chelation treatments within 3 months...
You likely can't join if
- Patients with fully HLA-matched donors;
- Received allogeneic transplantation, which needs to be weighed and evaluated by an expert committee; received other gene therapies;
- Have previously undergone splenectomy;
- Uncorrected bleeding disorder;
- Uncontrolled epilepsy and mental illness;
- Received hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months prior to enrollment;
See the full eligibility criteria
- Aged 18-35 years (inclusive), ICF can be provided by the patient and/or legal guardian;
- Definitively diagnosed with severe TDT without genotype restriction, and a valid test report can be provided;
- Average transfusion volume \> 100 mL/kg/year or transfusion frequency \> 8 times/year within 2 years prior to enrollment, or has been definitively diagnosed with TDT;
- At least 3 months of full volume transfusion (verification of blood transfusion records can be provided) prior to screening, and Hb is maintained at ≥ 9.0 g/dL;
- Ferritin load \< 3000 μg/L, cardiac and liver iron indicates moderate or lesser iron overload; records of iron chelation treatments within 3 months before screening (including prescription or receipt) can be provided;
- Acceptable organ functions (including heart, liver, kidney, lung and coagulation functions), stable disease condition, and suitable for busulfan pre-treatment and hematopoietic stem cell (HSC) transplantation as judged...
- Meets follow-up requirements, adheres to treatment arrangements, and is able to return to the hospital regularly to undergo various examinations within 2 years after reinfusion of HGI-001 injection.
- Patients with fully HLA-matched donors;
- Received allogeneic transplantation, which needs to be weighed and evaluated by an expert committee; received other gene therapies;
- Have previously undergone splenectomy;
- Uncorrected bleeding disorder;
- Uncontrolled epilepsy and mental illness;
- Received hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months prior to enrollment;
- Psychoactive substance abuse, drug or alcohol abuse within 6 months prior to enrollment;
- Patients with pulmonary hypertension who have not been given effective intervention;
- Persistent toxicity (≥ CTCAE grade 2) induced by previous treatment;
- Positive for anti-RBC antibodies in antibody screening;
- Positive for hepatitis B surface antigen (HBsAg) and HBV DNA copy number \> upper limit of normal (ULN) (HBV DNA test not required for patients negative for HBsAg), positive for hepatitis C virus (HCV) antibody...
- Has or has had malignant tumors or myeloproliferative disease or immunodeficiency disease;
- Immediate family member with or suspected of having a familial cancer (including but not limited to hereditary breast and ovarian cancers, nonpolyposis colorectal cancer, and adenomatous polyposis);
- Severe bacterial, viral, fungal or parasitic infection;
- Other illnesses which render the subject unsuitable for participation (e.g., severe liver, kidney or heart disease); Definition of severe liver and kidney disease: a. Aspartate aminotransferase (AST), alanine...
- WBC \< 3 × 109/L and/or PLT \< 100 × 109/L;
- Has diabetes, abnormal thyroid functions or other endocrine disorder;
- Participated in other treatment clinical studies within 4 weeks before the trial;
- Poor adherence or other conditions that renders the subject unsuitable for participation as judged by the investigator.
The study team makes the final eligibility decision.
Where it's taking place
- Shenzhen, Guangdong, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shenzhen, Guangdong, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.