This study is a single-arm, open-label, dose-escalation trial, planning to enroll 3-9 patients with transfusion-dependent β-thalassemia, aimed at assessing the safety and tolerability of a single-dose of YOLT-204 in patients with transfusion-dependent β-thalassemia; to preliminarily evaluate the impact of a single -dose of YOLT-204 on the levels of fetal hemoglobin in the plasma
Age between 18 to 35 years, gender not limited.
The patient has fully understood this study and voluntarily signed an informed consent form.
Clinically diagnosed as a patient with transfusion-dependent β-thalassemia, excluding the genotype: β0β0.
Karnofsky Performance Status (KPS) score of at least 70.
History of multiple drug allergies or a history of allergic reactions to oligonucleotides or lipid nanoparticles (LNPs).
Diagnosed with compound alpha-thalassemia.
Clinically significant active bacterial, viral, fungal, or parasitic infections at the time of screening, as judged by the investigator.
White blood cell count (WBC) <3×10^9/L and/or platelet count <100×10^9/L not due to hypersplenism, as judged by the investigator.
Single group
YOLT-204
DrugThe intervention group will receive YOLT-204 on day0
Treatment-Emergent Adverse Events
Number of Participants with Treatment-Emergent Adverse Events
Laboratory Test Findings
Number of Participants with Clinically Significant Clinical Laboratory Test Findings
Safety Measurements
Number of Participants with Clinically Significant Safety Measurements
3 months of sustained transfusion reduction
Analysis begins one month after treatment with YOLT-204, and the proportion of patients who achieve at least 3 months of sustained transfusion reduction (sustained TR3) is obtained.
6 months of sustained transfusion reduction
Analysis begins one month after treatment with YOLT-204, and the proportion of patients who achieve at least 6 months of sustained transfusion reduction (sustained TR6) is obtained.
6 months of transfusion independence
Analysis begins one month after treatment with YOLT-204, and the proportion of patients who achieve at least 6 months of transfusion independence (sustained TI6) is obtained.
The proportion of alleles with intended modifications
The proportion of alleles with intended modifications in peripheral blood leukocytes and bone marrow cells over time.
Fetal hemoglobin concentration
The change in fetal hemoglobin concentration over time after YOLT-204 infusion
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This trial is not recruiting at the moment. You can still explore other options:
