NCT06363760Enrolling By Invitation不适用
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
适应症
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 54
- 试验地点
- 18
- 主要终点
- Adverse events of special interest (AESI)
研究概览
简要总结
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
详细描述
This is a non-interventional, multicenter study evaluating the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion dependent b-thalassemia (TDT) who received EDIT-301 in parent studies EM-SCD-301-001 (NCT04853576) or EDIT-301-BThal-001 (NCT05444894). No investigational drug product will be administered in the LTFU study.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 12 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must have received an EDIT-301 infusion as part of a clinical study.
- •Participant or legal representative/guardian (if applicable) must sign and date the informed consent form (ICF) or assent, if applicable for this long-term follow-up study.
排除标准
- •Participant is still actively enrolled in an EDIT-301 treatment study and has not yet met eligibility for long term follow-up in this study.
结局指标
主要结局
Adverse events of special interest (AESI)
时间窗: up to 15 years post EDIT-301 infusion
Adverse events of interest (AEI)
时间窗: up to 15 years post EDIT-301 infusion
Adverse events (AEs) related to EDIT-301
时间窗: up to 15 years post EDIT-301 infusion
Serious adverse events (SAEs)
时间窗: up to 15 years post EDIT-301 infusion
All-cause mortality
时间窗: up to 15 years post EDIT-301 infusion
次要结局
- HbF concentration (g/dL) over time for TDT participants(up to 15 years post EDIT-301 infusion)
- Proportion of SCD participants with severe vaso-occlusive events (sVOEs) over time post-EDIT-301 infusion(up to 15 years post EDIT-301 infusion)
- Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion(up to 15 years post EDIT-301 infusion)
- SCD and TDT: Total hemoglobin (Hb) concentration (g/dL) over time(up to 15 years post EDIT-301 infusion)
- Complete Blood Count (CBC) red cell indices assay over time for SCD participants(up to 15 years post EDIT-301 infusion)
- Percentage of HbF over total non-transfused total Hb (non-transfused total Hb refers to the total g/dL of all Hb variants, excluding HbA) over time for TDT participants.(up to 15 years post EDIT-301 infusion)
- Frequency of SCD-related transfusions over time post-EDIT-301 infusion for SCD participants(up to 15 years post EDIT-301 infusion)
- The level of HbF production determined by fractionation of different forms of Hb (including but not limited to HbA, HbA2, HbC, HbD, HbE, and HbS) over time for SCD participants.(up to 15 years post EDIT-301 infusion)
- Proportion of TDT participants with sustained transfusion reduction (TR) over time(up to 15 years post EDIT-301 infusion)
- Frequency of TDT-related transfusions over time post-EDIT-301 infusion for TDT participants(up to 15 years post EDIT-301 infusion)
- SCD and TDT: Proportion of alleles with intended genetic modification present in peripheral blood nucleated cells and bone marrow derived CD34+ over time(up to 15 years post EDIT-301 infusion)
- HbF and HbS concentration (g/dL) over time for SCD participants(up to 15 years post EDIT-301 infusion)
- Proportion of TDT participants receiving iron chelation therapy over time.(up to 15 years post EDIT-301 infusion)
- Proportion of TDT participants with sustained transfusion independence (TI) over time(up to 15 years post EDIT-301 infusion)
- Average HbF (pg) per RBC over time for SCD participants(up to 15 years post EDIT-301 infusion)
- SCD and TDT: Percent of peripheral red blood cells (RBCs) containing HbF (F-cells) over time(up to 15 years post EDIT-301 infusion)
- Average HbF (pg) per F-cell over time for SCD participants(up to 15 years post EDIT-301 infusion)
- Iron overload of TDT participants(up to 15 years post EDIT-301 infusion)
研究者
研究点 (18)
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