RecruitingEarly Phase 1

the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia

China3 participantsStarted 2022-05-27

Plain-language 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

Who can participate

Age range

18 Years – 35 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion 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 by the investigator;
. 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

. Patients with fully HLA-matched donors;

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Overall response rate

Timeframe: 24 months

Incidence and severity of AEs

Timeframe: 0-24 months

Incidence of SAEs

Timeframe: 0-24 months

Transplantation-related fatal and disabling events within day 100 after transplantation

Timeframe: Day 100

Overall survival rate during the clinical trial

Timeframe: 0-24 months

HGI-001 injection-related replicating lentivirus test

Timeframe: 0-24 months

Change from baseline in Clonal variations containing specific viral integration sites

Timeframe: 0-24 months

Number of patients with abnormal hematology and bone marrow cytology within 24 months after reinfusion, and percent of patients with abnormal RBC proliferation

Trial details

NCT IDNCT05864170

SponsorShenzhen Hemogen

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-12-30

Contact for this trial

Haigang Sun

13823168465 sunhaigang@genomics.cn

View on ClinicalTrials.gov More β-thalassemia trials

Who can participate

Age range

18 Years – 35 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion 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 by the investigator;
. 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

. Patients with fully HLA-matched donors;

What they're measuring

Overall response rate

Timeframe: 24 months

Incidence and severity of AEs

Timeframe: 0-24 months

Incidence of SAEs

Timeframe: 0-24 months

Transplantation-related fatal and disabling events within day 100 after transplantation

Timeframe: Day 100

Overall survival rate during the clinical trial

Timeframe: 0-24 months

HGI-001 injection-related replicating lentivirus test

Timeframe: 0-24 months

Change from baseline in Clonal variations containing specific viral integration sites

Timeframe: 0-24 months

Number of patients with abnormal hematology and bone marrow cytology within 24 months after reinfusion, and percent of patients with abnormal RBC proliferation