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 range18 Years – 35 Years

SexALL

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;
✕. 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;

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

Timeframe: 0-24 months

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 range18 Years – 35 Years

SexALL

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;
✕. 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;

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

Timeframe: 0-24 months