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鲁索替尼治疗 II 级至 IV 级类固醇难治性急性移植物抗宿主病患者的疗效和安全性研究

2026年11月6日 更新者：Novartis Pharmaceuticals

鲁索替尼治疗中国 II-IV 级皮质类固醇难治性急性移植物抗宿主病患者的单臂、多中心研究

The purpose of this study is to assess the efficacy and safety of ruxolitinib therapy in Chinese adults and adolescents (≥ 12 years old) with Grade II-IV steroid-refractory acute graft versus host disease (SR-aGvHD).

研究概览

地位

主动，不招人

条件

干预/治疗

详细说明

Participants will start with a screening period to assess the eligibility; only participants who meet all the inclusion and none of the exclusion criteria will start study treatment from Day 1 to Week 24 or end of treatment. Following safety follow up visits, participants will receive the long-term follow-up until Month 12.

研究类型

介入性

注册 (实际的)

36

阶段

  • 第四阶段

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习地点

  • 中国
      • Beijing、中国、100034
        • Novartis Investigative Site
      • Beijing、中国、100070
        • Novartis Investigative Site
      • Taian、中国、271000
        • Novartis Investigative Site
      • Tianjin、中国、300020
        • Novartis Investigative Site
    • Guangdong
      • Guangzhou、Guangdong、中国、510515
        • Novartis Investigative Site
    • Henan
      • Zhengzhou、Henan、中国、450003
        • Novartis Investigative Site
    • Hubei
      • Wuhan、Hubei、中国、430030
        • Novartis Investigative Site
    • Shanxi
      • Xian、Shanxi、中国、710061
        • Novartis Investigative Site
    • Zhejiang
      • Hangzhou、Zhejiang、中国、310003
        • Novartis Investigative Site

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

12年 至 100年 (孩子、成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

描述

Key Inclusion criteria

  • Male or female Chinese participants aged 12 or older at the time of informed consent. Written informed consent from participant, parent or legal guardian.

  • Able to swallow tablets.

  • Have undergone alloSCT from any donor source (matched unrelated donor, sibling, haplo-identical) using bone marrow, peripheral blood stem cells, or cord blood.

  • Clinically diagnosed Grades II to IV acute GvHD as per standard criteria occurring after alloSCT requiring systemic immune suppressive therapy.

  • Evident myeloid and platelet engraftment (confirmed within 48 hours prior to study treatment (ruxolitinib) start):

  • Confirmed diagnosis of steroid refractory aGvHD defined as participants administered systemic corticosteroids (methylprednisolone at least 1 mg/kg/day [or equivalent prednisone dose at least 1.25 mg/kg/day]), given alone or combined with calcineurin inhibitors (CNI) and either:

    1. Progression based on organ assessment after at least 3 days compared to organ stage at the time of initiation of systemic corticosteroid +/- CNI for the treatment of Grade II to IV aGvHD. OR

    2. Failure to achieve at a minimum partial response based on organ assessment after 7 days compared to organ stage at the time of initiation of systemic corticosteroid +/-CNI for the treatment of Grade II to IV. OR

    3. Participants who fail corticosteroid taper defined as fulfilling either one of the following criteria:

      • Requirement for an increase in the corticosteroid dose to methylprednisolone ≥ 1 mg/kg/day (or equivalent prednisone dose ≥ 1.25 mg/kg/day). OR
      • Failure to taper the methylprednisolone dose to < 0.5 mg/kg/day (or equivalent prednisone dose <0.6 mg/kg/day) for a minimum of 7 days.

Key Exclusion criteria

  • Has received more than one systemic treatment for steroid refractory aGvHD. Participants who received JAK inhibitor therapy for any indication after initiation of current alloSCT conditioning.
  • Clinical presentation resembling de novo chronic GvHD or GvHD overlap syndrome with both acute and chronic GvHD features.
  • Failed prior alloSCT within the past 6 months. Presence of relapsed primary malignancy after the alloSCT was performed.
  • Presence of an active uncontrolled infection including significant bacterial, fungal, viral or parasitic infection requiring treatment.
  • SR-aGvHD occurring after non-scheduled donor lymphocyte infusion (DLI) administered for pre-emptive treatment of malignancy recurrence. Note: Participants who have received a scheduled DLI as part of their transplant procedure and not for management of malignancy relapse are eligible.
  • Presence of significant respiratory disease, severely impaired renal function, clinically significant or uncontrolled cardiac disease, unresolved cholestatic and liver disorders (not attributable to aGvHD). Disorders and/or current therapy with medications that interfere with coagulation or platelet function.

Other protocol-defined inclusion / exclusion criteria may apply

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 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：Ruxolitinib

Participants will receive ruxolitinib orally of 10 mg BID daily (given as two 5-mg tablets, approximately 12 hours apart).
药品：Ruxolitinib

Ruxolitinib is taken orally daily at 10 mg BID, given as two 5-mg tablets.

其他名称：
  • INC4​​24

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
Overall Response Rate (ORR) at Day 28 per Investigators
大体时间：Day 28

The ORR at Day 28 defined as the percentage of participants demonstrating a complete response (CR) or partial response (PR) without requirement for additional systemic therapies for an earlier progression, mixed response or nonresponse, according to standard criteria and assessed by investigators.
Day 28

次要结果测量

结果测量
措施说明
大体时间
Durable Overall response rate (ORR) at Day 56
大体时间：Day 56

Durable ORR at Day 56 is defined as the percentage of participants who achieve a complete response (CR) or partial response (PR) at Day 28 and maintain a CR or PR at Day 56.
Day 56
Duration of Response (DOR)
大体时间：From Week 1 to long term follow up Month 12

DOR is defined as the time from first response until aGvHD progression or the date of additional systemic therapies for aGvHD.
From Week 1 to long term follow up Month 12
Best overall response (BOR)
大体时间：From week 1 to Day 28

Percentage of participants who achieved overall response (complete response (CR) + Partial response (PR) at any time point up to and including Day 28 and before the start of additional systemic therapy for aGvHD.
From week 1 to Day 28
Overall survival (OS)
大体时间：From the date of start of study treatment to date of death, up to approx. 12 months

Overall survival (OS) is defined as the time from the date of start of study treatment to date of death due to any cause.
From the date of start of study treatment to date of death, up to approx. 12 months
Non-relapse mortality (NRM)
大体时间：From date of start of study treatment to date of death, up to approx. 12 months

Non-relapse mortality (NRM) is defined as the time from date of start of study treatment to date of death not preceded by hematologic disease relapse/progression.
From date of start of study treatment to date of death, up to approx. 12 months
Event-free survival (EFS)
大体时间：From the date of start of study treatment to the date of hematologic disease relapse/progression, graft failure, or death, up to approx. 12 months

Event-free survival (EFS) is defined as the time from the date of start of study treatment to the date of hematologic disease relapse/progression, graft failure, or death due to any cause.
From the date of start of study treatment to the date of hematologic disease relapse/progression, graft failure, or death, up to approx. 12 months
Failure-free survival (FFS)
大体时间：From the date of start of study treatment to date of hematologic disease relapse/progression, non-relapse mortality, or addition of new systemic aGvHD treatment, up to approx. 12 months

Failure-free survival (FFS) is defined as the time from the date of start of study treatment to date of hematologic disease relapse/progression, non-relapse mortality, or addition of new systemic aGvHD treatment.
From the date of start of study treatment to date of hematologic disease relapse/progression, non-relapse mortality, or addition of new systemic aGvHD treatment, up to approx. 12 months
Malignancy Relapse/Progression (MR)
大体时间：From date of start of study treatment to hematologic malignancy relapse/progression, up to approx. 12 months

Malignancy Relapse/Progression (MR) is defined as the time from date of start of study treatment to hematologic malignancy relapse/progression. Calculated for participants with underlying hematologic malignant disease.
From date of start of study treatment to hematologic malignancy relapse/progression, up to approx. 12 months
Reduction of daily corticosteroids dose
大体时间：Up to Day 56

This includes the assessment of systemic corticosteroid use and daily dose, and the percentage of participants successfully tapered off all systemic corticosteroids until Day 56, by time intervals and overall.
Up to Day 56
Cumulative incidence of chronic GvHD
大体时间：From Week 1 to long term follow up of month 12

Cumulative incidence of chronic GvHD (cGvHD) includes mild, moderate and severe occurrences.
From Week 1 to long term follow up of month 12

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Novartis Pharmaceuticals

调查人员

  • 研究主任：Novartis Pharmaceuticals、Novartis Pharmaceuticals

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2024年7月4日

初级完成 (实际的)

2026年9月3日

研究完成 (估计的)

2027年7月8日

研究注册日期

首次提交

2024年6月5日

首先提交符合 QC 标准的

2024年6月14日

首次发布 (实际的)

2024年6月17日

研究记录更新

最后更新发布 (实际的)

2026年11月9日

上次提交的符合 QC 标准的更新

2026年11月6日

最后验证

2026年11月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • CINC424C2416

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

药物和器械信息、研究文件

研究美国 FDA 监管的药品

不

研究美国 FDA 监管的设备产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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