An Early Phase 1 interventional study of α-globin restored autologous hematopoietic stem cells in α-thalassemia, sponsored by Shenzhen Hemogen. Recruiting at 1 site in China. Open to participants aged 12 Years to 35 Years. Per ClinicalTrials.gov, last updated 2024-11-29.
Sponsored by Shenzhen Hemogen · Early Phase 1, Interventional, and Treatment
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
250 studies on the registry are indexed under Thalassemia; 43 are open to participants now.
This study's planned enrollment of 3 is below the median of 33 across 170 interventional studies indexed under Thalassemia.
Browse Thalassemia studies →Shenzhen Hemogen is the lead sponsor of 4 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CTCAE grade
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Three transfusion-dependent α-thalassaemia subjects aged 12-35 years will be reinfused with α-globin restored autologous hematopoietic stem cells modified with LentiHBA T\>C
Biological: α-globin restored autologous hematopoietic stem cells
α-globin restored autologous hematopoietic stem cells modified with LentiHBA T\>C
Overall response rate
Percent of patients with average VCN \> 0.1 in peripheral blood mononuclear cells
Time frame: 0-24 months
Incidence and severity of AEs
The number and the percentage of adverse events related to transplantation will be summarized according to NCI CTCAE 5.0
Time frame: 0-24 months
incidence of SAEs
The number of SAE related to transplantation will be summarized according to NCI CTCAE 5.0
Time frame: 0-24 months
Transplantation-related fatal and disabling events within 100 d after transplantation
Transplantation-related fatal and disabling events
Time frame: Day 100
Overall survival rate during the clinical trial
Number of patients alive through the whole trial will be record
Time frame: 0-24 months
HGI-002 injection-related replicating lentivirus test
The percentage of RCL should be negative in the 24 months after transplant
Time frame: 0-24 months
Change from baseline in Clonal variations containing specific viral integration sites
Evaluation of the percentage of participants without abnormal clonal proliferation and polyclonal engraftment at baseline, 6, 12, 18 and 24 months after transplant. More than 1000 VIS retrieved from peripheral blood should be checked.
Time frame: 0-24 months
Number of patients with abnormal hematology cytology and bone marrow cytology within 24 months after reinfusion
Number of patients with abnormal hematology cytology and bone marrow cytology
Time frame: 0-24 months
Treatment response rate
Percent of patients with average VCN \> 0.1 in PBMCs
Time frame: 12 Months
Percent of subjects with successful HSC engraftment
Criteria for successful engraftment: Absolute neutrophil count \> 0.5 × 10 9 /L for 3 consecutive days; platelet count is maintained at \> 20 × 10 9 /L for 7 consecutive days without platelet transfusion
Time frame: 1 month
Change in transfusion volume or frequency
Change in average annual transfusion volume or frequency from baseline or change in percentage
Time frame: 0-24 Months
Transfusion improvement rate
Percent of subjects with ≥ 30% decrease in the average annual (0-12 months, 12-24 months) transfusion volume or frequency from baseline after reinfusion of HGI-002 injection
Time frame: 0-24 Months
Transfusion independence (TI) rate
Percent of subjects who do not require transfusion for at least 12 consecutive months after reinfusion of HGI-002 injection and have a weighted average Hb of ≥ 9.0 g/dL
Time frame: 0-24 Months
Transfusion-free survival
the time when a subject meets the TI criteria and maintains transfusion-free survival
Time frame: 0-24 Months
Changes in VCN
Vector copy number
Time frame: 0-24 Months
Changes in cardiac iron load after reinfusion of HGI-002 injection
T2 MRI
Time frame: 0-24 Months
Changes in liver iron load after reinfusion of HGI-002 injection
T2 MRI
Time frame: 0-24 Months
Changes in serum ferritin after reinfusion of HGI-002 injection
serum ferritin
Time frame: 0-24 Months
Changes use of iron chelation medications after reinfusion of HGI-002 injection
iron chelation medications
Time frame: 0-24 Months
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Shenzhen Hemogen