A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Treatment-Emergent Adverse Events
研究概览
简要总结
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
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 35 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •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.
- •Uncorrected bleeding disorders.
- •Received treatment with erythropoietin (EPO) within the three months prior to enrollment.
- •Severe iron overload, with serum ferritin levels ≥5000 ng/ml.
- •Positive for hepatitis B surface antigen (HBsAg), antibodies to hepatitis C virus, antibodies to human immunodeficiency virus (HIV), or specific antibodies to Treponema pallidum (syphilis).
- •History of hematopoietic stem cell transplantation, gene therapy, or gene editing therapy.
- •Participation in another clinical study and use of investigational drugs within 3 months prior to starting the study drug.
- •History or current presence of malignant tumors or myeloproliferative diseases or immunodeficiency diseases.
- •Presence of severe mental illness that prevents cooperation with treatment; significant pulmonary arterial hypertension requiring medical intervention; recent malaria; a history of hematological tumors in immediate family members.
- •Any past or current disease, treatment, or laboratory abnormality that may interfere with the study results, affect the patient's full participation in the study, or that the investigator deems unsuitable for participation in this clinical study.
研究组 & 干预措施
The intervention group will receive YOLT-204 on day0
干预措施: YOLT-204 (Drug)
结局指标
主要结局
Treatment-Emergent Adverse Events
时间窗: 52 weeks after dose
Number of Participants with Treatment-Emergent Adverse Events
Laboratory Test Findings
时间窗: 52 weeks after dose
Number of Participants with Clinically Significant Clinical Laboratory Test Findings
Safety Measurements
时间窗: 52 weeks after dose
Number of Participants with Clinically Significant Safety Measurements
3 months of sustained transfusion reduction
时间窗: 4 months after dose
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.
3 months of transfusion independence
时间窗: 4 months after dose
Analysis begins one month after treatment with YOLT-204, and the proportion of patients who achieve at least 3 months of transfusion independence (sustained TI3) is obtained.
次要结局
- 6 months of sustained transfusion reduction(7 months after dose)
- 6 months of transfusion independence(7 months after dose)
- The proportion of alleles with intended modifications(52 weeks after dose)
- Fetal hemoglobin concentration(52 weeks after dose)
- Total hemoglobin concentration(52 weeks after dose)