NCT05456880进行中（未招募）1 期
A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients With Sickle Cell Disease and Severe Vaso-Occlusive Crises (Beacon Trial)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 15
- 试验地点
- 19
- 主要终点
- Proportion of patients with successful neutrophil engraftment
研究概览
简要总结
This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD
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
- 12 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
BEAM-101
Experimental
BEAM-101 manufactured with autologous CD34+ hematopoietic stem cells collected by plerixafor mobilization and edited ex vivo. No maximum dose has been set for BEAM-101; all of the gene edited cells that pass release specifications will be administered to the patient. BEAM 101 will be administered as a single dose by IV infusion.
干预措施: BEAM-101 (Biological)
结局指标
主要结局
Proportion of patients with successful neutrophil engraftment
时间窗: BEAM-101 administration to month 24
Time to neutrophil engraftment
时间窗: BEAM-101 administration to month 24
Safety and tolerability assessments based on frequency, severity and seriousness of adverse events (AE's)
时间窗: BEAM-101 administration through month 24
Time to platelet engraftment
时间窗: BEAM-101 administration to month 24
Transplant-related mortality within 100 days after beam-101 treatment
时间窗: BEAM-101 administration to day 100
Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline
时间窗: 6 months to time of analysis as compared to baseline
次要结局
- Change in free Hgb over time(Month 3 post BEAM-101 treatment to month 24 as compared to baseline)
- Change in annualized duration of hospitalizations for VOCs(Month 6 post BEAM-101 treatment to month 24 as compared to baseline)
- Proportion of patients with HbF ≥30%, for at least 3 months(Month 6 post BEAM-101 treatment to month 24 as compared to baseline)
- Change in total bilirubin over time(Month 3 post BEAM-101 treatment to month 24 as compared to baseline)
- Proportion of patients experiencing at least 75% reduction in annualized rate of severe VOCs(Month 6 post BEAM-101 treatment to month 24 as compared to baseline)
- Proportion of patients experiencing no severe VOCs(6 months to time of analysis as compared to baseline)
- Change in lactate dehydrogenase (LDH) over time(Month 3 post BEAM-101 treatment to month 24 as compared to baseline)
- Change in reticulocyte count over time(Month 3 post BEAM-101 treatment to month 24 as compared to baseline)
- Change in total Hgb (g/dL) concentration over time(Baseline to month 24)
- Change in annualized number of hospitalizations for VOCs(Month 6 post BEAM-101 treatment to month 24 as compared to baseline)
- Change in RBC transfusions per month and per year for SCD-related indications(Month 2 post BEAM-101 treatment to month 24 as compared to baseline)
- Change in haptoglobin over time(Month 3 post BEAM-101 treatment to month 24 as compared to baseline)
研究者
研究点 (19)
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• The death was attributed to the busulfan-conditioning regimen, a known risk in myeloablative transplants, rather than the BEAM-101 therapy itself.
• Beam Therapeutics is developing an alternative approach, ESCAPE, using a CD117 monoclonal antibody to avoid the need for busulfan conditioning.
• Preclinical data in non-human primates showed that ESCAPE was well-tolerated and resulted in approximately 85% of red blood cells expressing HbF.last yearPatient Death in Beam Therapeutics' Sickle Cell Trial Linked to Conditioning Regimen• A patient in Beam Therapeutics' BEACON trial of BEAM-101, a base editing therapy for sickle cell disease (SCD), died four months post-infusion due to respiratory failure.
• Investigators determined the death was likely related to the busulfan conditioning regimen and not directly caused by BEAM-101, according to Beam Therapeutics.
• The FDA and the BEACON trial's Data Safety Monitoring Committee reviewed the death, allowing the trial to proceed without protocol changes.
• The event underscores the need for less toxic preconditioning options in cell and gene therapy, as current regimens are associated with significant toxic effects.last year