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
Qualifiers
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
Treatments tested in this trial
- β-globin restored autologous hematopoietic stem cells
Treatment groups
8 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov