NCT05864170 the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia
| NCT ID | NCT05864170 |
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Sponsor | Shenzhen Hemogen |
| Condition | β-thalassemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 3 participants |
| Start Date | 2022-05-27 |
| Primary Completion | 2025-12-30 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 3 participants in total. It began in 2022-05-27 with a primary completion date of 2025-12-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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
Eligibility Criteria
Inclusion Criteria: 1. Aged 18-35 years (inclusive), ICF can be provided by the patient and/or legal guardian; 2. Definitively diagnosed with severe TDT without genotype restriction, and a valid test report can be provided; 3. 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; 4. 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; 5. 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; 6. 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 by the investigator; 7. 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. Exclusion Criteria: 1. Patients with fully HLA-matched donors; 2. Received allogeneic transplantation, which needs to be weighed and evaluated by an expert committee; received other gene therapies; 3. Have previously undergone splenectomy; 4. Uncorrected bleeding disorder; 5. Uncontrolled epilepsy and mental illness; 6. Received hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months prior to enrollment; 7. Psychoactive substance abuse, drug or alcohol abuse within 6 months prior to enrollment; 8. Patients with pulmonary hypertension who have not been given effective intervention; 9. Persistent toxicity (≥ CTCAE grade 2) induced by previous treatment; 10. Positive for anti-RBC antibodies in antibody screening; 11. 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, positive human immunodeficiency virus (HIV), or positive for Treponema pallidum antibody (TP-Ab) (subjects who are positive for the antibody due to vaccination can be enrolled). In certain clinical environments/regions, subjects who are positive for other tests can also be excluded from the trial, such as, human lymphocytic virus-1 (HTLV-1) or -2 (HTLV-2), tuberculosis, and toxoplasmosis. 12. Has or has had malignant tumors or myeloproliferative disease or immunodeficiency disease; 13. 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); 14. Severe bacterial, viral, fungal or parasitic infection; 15. 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 aminotransferase (ALT), or total bilirubin \> 3 × ULN; b. Liver magnetic resonance imaging (MRI) indicates significant cirrhosis; c. Liver biopsy indicates cirrhosis, severe fibrosis or active hepatitis (liver biopsy is only performed when liver MRI indicates active hepatitis and significant fibrosis without evidence for cirrhosis); d. Creatinine clearance \< 30% of normal; 16. WBC \< 3 × 109/L and/or PLT \< 100 × 109/L; 17. Has diabetes, abnormal thyroid functions or other endocrine disorder; 18. Participated in other interventional clinical studies within 4 weeks before the trial; 19. Poor adherence or other conditions that renders the subject unsuitable for participation as judged by the investigator.
Contact & Investigator
Chao Liu, PHD
PRINCIPAL INVESTIGATOR
Shenzhen Hemogen
Frequently Asked Questions
Who can join the NCT05864170 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 35 Years, studying β-thalassemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05864170 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05864170 currently recruiting?
Yes, NCT05864170 is actively recruiting participants. Contact the research team at sunhaigang@genomics.cn for enrollment information.
Where is the NCT05864170 trial being conducted?
This trial is being conducted at Shenzhen, China.
Who is sponsoring the NCT05864170 clinical trial?
NCT05864170 is sponsored by Shenzhen Hemogen. The principal investigator is Chao Liu, PHD at Shenzhen Hemogen. The trial plans to enroll 3 participants.