Lanoracopan to Prevent Acute GVHD Following Haploidentical Peripheral Blood Stem Cell Transplantation: A Prospective Multicenter Single-Arm Exploratory Study
Lanoracopan for Prevention of Acute Graft-Versus-Host Disease After Haploidentical Peripheral Blood Stem Cell Transplantation: A Prospective, Multicenter, Single-Arm Exploratory Study
This is a prospective, multicenter, open-label, single-arm exploratory study. Thirty adult patients with hematologic malignancies who are scheduled to receive haploidentical peripheral blood stem-cell transplantation with myeloablative conditioning will be enrolled.
All participants will receive standard anti-thymocyte globulin (ATG, total 7.5 mg/kg), standard graft-versus-host-disease (GVHD) prophylaxis (calcineurin inhibitor + mycophenolate mofetil + short-course methotrexate), plus lanoracopan 200 mg twice daily from day +1 to day +90 after stem-cell infusion. Lanoracopan is an approved complement alternative-pathway inhibitor being used off-label for GVHD prevention in this trial. Mandatory antibiotic prophylaxis against encapsulated bacterial pathogens will be given.
The primary goal is to evaluate the safety of lanoracopan in this transplant setting, including adverse events / serious adverse events, neutrophil and platelet engraftment, graft-failure rate, and infectious complications. The key secondary objective is to explore the cumulative incidence of grade II-IV acute GVHD at day 100 post-transplant. Additional secondary outcomes include chronic GVHD, TA-TMA, 1-year overall survival, disease-free survival, cumulative incidence of relapse, and non-relapse mortality. Exploratory biomarker assessments will be performed on plasma and serum samples.
Participants will be followed for 1 year after transplantation. No control group is included in this exploratory study.
研究概览
地位
研究类型
注册 (估计的)
阶段
- 阶段2
联系人和位置
学习联系方式
- 姓名：LIMIN LIU, MD
- 电话号码：+86-512-6778183
- 邮箱：Liminliu1006@163.com
参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria:
- Age 18-70 years, any gender.
- Diagnosed with hematologic malignancy according to WHO 2022 guidelines and eligible for allogeneic hematopoietic stem-cell transplantation.
- Scheduled to receive haploidentical peripheral blood stem-cell transplantation.
- Receive myeloablative conditioning regimen.
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
- Adequate major organ function: left-ventricular ejection fraction ≥50%; DLCO ≥50% predicted value; ALT/AST ≤3×ULN, total bilirubin ≤2×ULN; creatinine clearance ≥60 mL/min.
- Able to understand study requirements and provide written informed consent.
Exclusion Criteria:
- Prior allogeneic hematopoietic stem-cell transplantation.
- Active uncontrolled infection judged by investigator.
- Myocardial infarction or unstable angina within previous 6 months.
- Known hypersensitivity to lanoracopan or its excipients.
- Pregnant or breastfeeding female subjects.
- Concurrent participation in other investigational drug clinical trials with similar mechanism.
- Any other conditions that make the subject unsuitable for enrollment as judged by the investigator.
学习计划
研究是如何设计的？
设计细节
- 主要用途：预防
- 分配：不适用
- 介入模型：单组作业
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的：Lanoracopan Combined With ATG and Standard GVHD Prophylaxis
|
200 mg oral, twice daily, administered from post-transplant day +1 to day +90. Off-label use for prevention of acute graft-versus-host-disease. Total dose 7.5 mg/kg, administered intravenously from pre-transplant day -5 to day -2. Standard-dose calcineurin inhibitor (either cyclosporine or tacrolimus) as part of routine GVHD prophylaxis. Mycophenolate mofetil given per local center routine for GVHD prophylaxis. Short-course methotrexate for standard GVHD prophylaxis after allogeneic stem-cell transplantation. |
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Safety: Incidence and severity of adverse events (AEs) and serious adverse events (SAEs); neutrophil and platelet engraftment; graft-failure rate; infectious complications
大体时间：From study drug initiation (post-transplant day +1) up to 1 year after stem-cell transplantation
|
To evaluate the safety of lanoracopan for aGVHD prophylaxis. Assess the incidence and maximum grade of adverse events (AEs) and serious adverse events (SAEs) graded by CTCAE version 5.0; time to neutrophil engraftment, time to platelet engraftment; graft failure rate; incidence of infectious complications among participants. |
From study drug initiation (post-transplant day +1) up to 1 year after stem-cell transplantation
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Cumulative incidence of grade II-IV acute graft-versus-host disease (aGVHD)
大体时间：100 days after stem-cell transplantation
|
Key secondary endpoint. Cumulative incidence of grade II-IV aGVHD at day 100 after haploidentical peripheral blood stem-cell transplantation, estimated by cumulative incidence function with non-relapse mortality as competing event. |
100 days after stem-cell transplantation
|
|
Cumulative incidence of chronic graft-versus-host disease (cGVHD)
大体时间：Up to 1 year after stem-cell transplantation
|
Cumulative incidence of cGVHD within 1-year post-transplantation. |
Up to 1 year after stem-cell transplantation
|
|
One-year overall survival (OS), disease-free survival (DFS), cumulative incidence of relapse (CIR), non-relapse mortality (NRM)
大体时间：Up to 1 year after stem-cell transplantation
|
Evaluate 1-year OS, 1-year DFS, 1-year CIR and 1-year NRM after haploidentical peripheral blood stem-cell transplantation. |
Up to 1 year after stem-cell transplantation
|
合作者和调查者
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- 20261008093031021
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)？
IPD 计划说明
Individual participant-level data (IPD) will not be shared with external researchers. This is a single-arm exploratory study sponsored by a hospital. Aggregated study results will be published in peer-reviewed journals and presented at academic conferences, but raw individual-level participant data cannot be released due to participant privacy protection and institutional data management requirements.
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