Health TrialsbyHealthPanel

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

Conditionβ-thalassemia
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-35
SponsorShenzhen Hemogen

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

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;

Disqualifiers

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;

Trial design

Design model

Single group

Treatments tested in this trial

  • β-globin restored autologous hematopoietic stem cells

    Biological/Vaccine

    β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q

Treatment groups

3 Participants
are divided into 1 treatment group
Group A: ExperimentalExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Overall response rate

Percent of patients with average VCN \> 0.1 in peripheral blood mononuclear cells (PBMCs) and average expression of exogenous adult hemoglobin HbAT87Q \> 2.0 g/dL

Time frame
24 months
2

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
3

Incidence of SAEs

The number of SAE related to transplantation will be summarized according to NCI CTCAE 5.0

Time frame
0-24 months
4

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

Transplantation-related fatal and disabling events

Time frame
Day 100

Secondary outcomes

1

Treatment response rate

Percent of patients with average VCN \> 0.1 in PBMCs and average expression of exogenous adult hemoglobin HbAT87Q \> 2.0 g/dL after reinfusion of HGI-001 injection

Time frame
12 Months
2

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
3

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
4

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-001 injection

Time frame
0-24 Months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Shenzhen Hemogen

Lead sponsor

Source ClinicalTrials.gov
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