A Safety and Effectiveness Registry Study of Patients With β-Thalassemia Treated With Betibeglogene Autotemcel (the Glostar Registry)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- bluebird bio
- 入组人数
- 150
- 试验地点
- 6
- 主要终点
- Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)
研究概览
简要总结
The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be treated with beti-cel in the post marketing setting at a center in the US that participates in the Registry.
- •Participant must sign an informed consent and/or assent prior to enrollment as required under applicable laws and regulations.
- •Participant must have signed an informed consent and/or assent permitting data to be shared with Center for International Blood and Marrow Transplant Research (CIBMTR).
- •Participant must be followed by a hematologist based in the US.
排除标准
- •There are no exclusion criteria for Registry participation.
结局指标
主要结局
Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)
时间窗: Through 15 years post-beti-cel infusion
The Sponsor considers the following events to be AEIs (which should be reported as a medically significant Serious Adverse Event \[SAE\]): * Any newly diagnosed malignancy * Neutrophil engraftment failure: defined as healthcare provider (HCP) decision to administer back-up cells or subsequent hematopoietic stem cell transplantation (HSCT) due to neutrophil recovery failure * Newly acquired human immunodeficiency virus-1 (HIV-1), HIV-2 infection and human t-lymphotropic virus (HTLV) infection * Any newly diagnosed autoimmune disorder * Any hepatic veno-occlusive disease (VOD) * Any clinically significant bleeding events.
次要结局
- Number of Participants with beti-cel related AEs(Through 15 years post-beti-cel infusion)
- Event-Free Survival(Through 15 years post-beti-cel infusion)
- Number of Participants with Serious Adverse Events (SAEs)(Through 15 years post-beti-cel infusion)
- Percentage of Participants Achieving Transfusion Independence(Through 15 years post-beti-cel infusion)