Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
ConditionΒ-thalassemia
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age6-35
SponsorShenzhen Hemogen
About this trial
This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia. The study aims to evaluate the safety and efficacy of autologous lentiviral-transduced CD34+ human hematopoietic stem cells (hHSCs) using the gene therapy product HGI-001.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Aged 6-35 years (inclusive), ICF can be provided by the patient and/or legal guardian;
Definitively diagnosed with severe TDT without genotype restriction (excluding patients with coexisting α-thalassemia), 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；
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;
Disqualifiers
Patients with fully HLA-matched donors;
Having previously received gene therapy, gene editing therapy, or allogeneic hematopoietic stem cell transplantation;
Uncorrected bleeding disorder;
Uncontrolled epilepsy and mental illness;
Trial design
Design model
Single group
Treatments tested in this trial
β-globin restored autologous hematopoietic stem cells
Other interventionEight transfusion-dependent β-thalassaemia subjects aged 6-35 years will be reinfused with β-globin restored autologous hematopoietic stem cells modified with LentiHBBT87Q
Treatment groups
8 Participants
are divided into 1 treatment groupGroup A: β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87QExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Transfusion Independence (TI) Rate
Percent of subjects who achieve transfusion independence, defined as not requiring transfusion for at least 12 consecutive months post-HGI-001 injection, with a weighted average Hb of ≥ 9.0 g/dL. Calculated from the point hemoglobin reaches ≥9 g/dL and no transfusions have occurred in the last 60 days.
Time frame
0-24 Months2
Incidence and Severity of Adverse Events (AEs) Related to Transplantation
Number and percentage of adverse events related to transplantation, summarized according to NCI CTCAE 5.0.
Time frame
0-24 Months3
Incidence of Serious Adverse Events (SAEs) Related to Transplantation
Number of serious adverse events related to transplantation, summarized according to NCI CTCAE 5.0.
Time frame
0-24 Months4
Overall Survival Rate During the Clinical Trial
Number of patients alive throughout the entire trial period.
Time frame
0-24 MonthsSecondary outcomes
1
Percentage of Subjects Achieving Transfusion Cessation for Over 6 Months Post-HGI-001 Injection
Percent of subjects who do not require transfusion for at least 6 consecutive months after reinfusion of HGI-001 injection
Time frame
0-24 Months2
Percentage of Subjects with Successful HSC Engraftment
Percentage of subjects meeting criteria for successful engraftment, defined as an absolute neutrophil count \> 0.5 × 10\^9/L for 3 consecutive days and a platelet count maintained at \> 20 × 10\^9/L for 7 consecutive days without platelet transfusion.
Time frame
1 month3
Percentage Change in Annual Transfusion Volume or Frequency
Percentage change in average annual transfusion volume or frequency from baseline.
Time frame
0-24 Months4
Transfusion Improvement Rate
Percentage of subjects with a decrease of 30% or more in the average annual transfusion volume or frequency (0-12 months, 12-24 months) from baseline after reinfusion of HGI-001 injection.
Time frame
0-24 MonthsSponsors and contacts
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Source ClinicalTrials.gov