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确定Asciminib在Ph+ CML-CP儿科患者中的疗效和安全性的研究

2026年11月9日 更新者：Novartis Pharmaceuticals

一项 II 期、多中心、开放标签、单臂研究，旨在评估 asciminib 在新诊断或既往接受过治疗的费城染色体阳性慢性期慢性粒细胞白血病（Ph+ CML-CP）儿童受试者中的安全性和有效性，无论是否伴有已知的 T315I 突变

The aim of this study is to support development of asciminib in the pediatric population (1 to < 18 years) with Ph+ CML-CP. The study will evaluate the efficacy and safety of asciminib in pediatric formulation (weigh-based dose, fed state) or adult formulation (fasted) in newly diagnosed and resistant or intolerant Ph+ CML-CP with or without T315I mutation.

研究概览

地位

招聘中

条件

干预/治疗

详细说明

This is a multi-center, open-label, single arm study of asciminib in pediatric participants aged

1 to <18 years old with Ph+ CML-CP newly diagnosed and previously treated with TKI treatment, with or without T315I mutation.

The study population will consist of three cohorts of Ph+ CML-CP pediatric participants:

  • Newly-diagnosed Ph+ CML-CP participants without known T315I mutation
  • Ph+ CML-CP participants resistant or intolerant to previous TKI without known T315I mutation
  • Ph+ CML-CP participants with known T315I mutation irrespective of prior TKI treatment

There is no fixed duration of study treatment for the participants. The study will end 5 years (240 weeks) after the last enrolled participants received their first dose of treatment in the study. The objective is to have enough follow up for safety, including growth and development and efficacy.

研究类型

介入性

注册 (估计的)

50

阶段

  • 阶段2

扩展访问

可用的 查看扩展访问记录。

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。

学习联系方式

研究联系人备份

  • 姓名：Novartis Pharmaceuticals
  • 电话号码：+41613241111

学习地点

  • 中国
      • Tianjin、中国、300020
        • 招聘中
        • Novartis Investigative Site
    • Henan
      • Zhengzhou、Henan、中国、450003
        • 招聘中
        • Novartis Investigative Site
  • 加拿大
    • Alberta
      • Edmonton、Alberta、加拿大、T6G 1C9
        • 招聘中
        • Novartis Investigative Site
    • Quebec
      • Montreal、Quebec、加拿大、H3T 1C5
        • 招聘中
        • Novartis Investigative Site
  • 匈牙利
    • Hajdu Bihar Megye
      • Debrecen、Hajdu Bihar Megye、匈牙利、4032
        • 招聘中
        • Novartis Investigative Site
  • 巴西
    • Rio de Janeiro
      • Rio de Janeiro、Rio de Janeiro、巴西、20211-030
        • 招聘中
        • Novartis Investigative Site
    • São Paulo
      • Barretos、São Paulo、巴西、14784-400
        • 招聘中
        • Novartis Investigative Site
      • São Paulo、São Paulo、巴西、04829-310
        • 招聘中
        • Novartis Investigative Site
  • 德国
      • Erlangen、德国、91054
        • 招聘中
        • Novartis Investigative Site
      • Hamburg、德国、20246
        • 招聘中
        • Novartis Investigative Site
    • Hesse
      • Frankfurt am Main、Hesse、德国、60590
        • 招聘中
        • Novartis Investigative Site
  • 意大利
    • PD
      • Padova、PD、意大利、35128
        • 招聘中
        • Novartis Investigative Site
    • RM
      • Roma、RM、意大利、00165
        • 招聘中
        • Novartis Investigative Site
  • 新加坡
      • Singapore、新加坡、229899
        • 招聘中
        • Novartis Investigative Site
  • 日本
      • Fukuoka、日本、8128582
        • 招聘中
        • Novartis Investigative Site
      • Saitama、日本、3308777
        • 招聘中
        • Novartis Investigative Site
    • Kanagawa
      • Yokohama、Kanagawa、日本、2328555
        • 招聘中
        • Novartis Investigative Site
  • 法国
      • Marseille、法国、13885
        • 招聘中
        • Novartis Investigative Site
      • Paris、法国、75019
        • 招聘中
        • Novartis Investigative Site
      • Poitiers、法国、86021
        • 招聘中
        • Novartis Investigative Site
      • Strasbourg、法国、67000
        • 招聘中
        • Novartis Investigative Site
  • 波兰
      • Gdansk、波兰、80-214
        • 招聘中
        • Novartis Investigative Site
      • Wroclaw、波兰、50-556
        • 招聘中
        • Novartis Investigative Site
  • 泰国
      • Bangkok、泰国、10330
        • 招聘中
        • Novartis Investigative Site
      • Bangkok、泰国、10700
        • 招聘中
        • Novartis Investigative Site
  • 澳大利亚
    • Queensland
      • Brisbane、Queensland、澳大利亚、4101
        • 招聘中
        • Novartis Investigative Site
    • South Australia
      • North Adelaide、South Australia、澳大利亚、5066
        • 招聘中
        • Novartis Investigative Site
  • 美国
    • Illinois
      • Chicago、Illinois、美国、60611
        • 招聘中
        • Ann and Robert H Lurie Childs Hosp
        • 首席研究员：
          • Sara Zarnegar-Lumley
        • 接触：
    • New Jersey
      • New Brunswick、New Jersey、美国、08901
        • 招聘中
        • Rutgers Cancer Institute of New Jersey
        • 首席研究员：
          • Richard DRACHTMAN
        • 接触：
    • New York
      • New York、New York、美国、10032
        • 招聘中
        • Columbia University Medical Center New York Presbyterian
        • 首席研究员：
          • Nobuko Hijiya
        • 接触：
    • Ohio
      • Cincinnati、Ohio、美国、45229-3039
        • 招聘中
        • Cinn Children Hosp Medical Center
        • 首席研究员：
          • Benjamin Mizukawa
        • 接触：
    • Washington
      • Seattle、Washington、美国、98105
  • 荷兰
      • Utrecht、荷兰、3584 CS
        • 招聘中
        • Novartis Investigative Site
  • 西班牙
      • Barcelona、西班牙、08035
        • 招聘中
        • Novartis Investigative Site
      • Madrid、西班牙、28046
        • 招聘中
        • Novartis Investigative Site
      • Madrid、西班牙、28009
        • 招聘中
        • Novartis Investigative Site
  • 越南
      • Hanoi、越南、100000
        • 招聘中
        • Novartis Investigative Site
  • 韩国
      • Seoul、韩国、03080
        • 招聘中
        • Novartis Investigative Site
      • Seoul、韩国、06591
        • 招聘中
        • 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

1年 至 18年 (孩子、成人)

接受健康志愿者

不

有资格学习的性别

全部

描述

Key Inclusion Criteria:

Participants eligible for inclusion in this study must meet all of the following criteria:

  1. Signed informed consent must be obtained prior to participation in the study.

  2. Male or female participants 1 and < 18 years of age at study enrollment

  3. Diagnosis of CML-CP (Apperley et al 2025) with cytogenetic confirmation of Philadelphia positive (Ph+) chromosome

  4. For participants with CML-CP newly diagnosed within 3 months of screening OR 5 For participants with CML - CP with high risk of developing resistance or intolerance to previous TKI:

    1. Unfavourable response to TKI is defined following the Apperley et al 2025 guidelines as:

      • At three months after the initiation of therapy: BCR::ABL1 ratio > 10% IS (if confirmed within 1-3 months)
      • At six months after the initiation of therapy: BCR::ABL1 ratio > 10% IS
      • At twelve months after initiation of therapy: BCR::ABL1 ratio > 1% IS
      • At any time loss of previous response
      • At any time emergent resistant BCR::ABL1 mutations or high-risk ACA from prior TKI treatment as per local test results

    2. Intolerance to TKI is defined as:

      • Non-hematologic intolerance: participants with grade 3 or 4 toxicity while on therapy (in which case the patient is eligible whether or not there was a dose reduction); or with persistent grade 2 toxicity unresponsive to optimal management including dose adjustments (unless dose reduction is not considered in the best interest of the patient if response is already suboptimal)
      • Hematologic intolerance: participants with grade 3 or 4 toxicity (absolute neutrophil count [ANC] or platelets) while on therapy that is recurrent after dose reduction to the lowest doses of the TKI

6. Evidence of typical BCR::ABL1 transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized RQ-PCR quantification.

7. Performance status: Karnofsky ≥ 50% for participants ≥ 16 years of age, and Lansky ≥ 50 for participants < 16 years of age at the time of screening.

Key Exclusion Criteria:

  1. Known second chronic phase (CP) of CML after previous progression to Accelerated Phase (AP)/Blast Phase (BP).
  2. Previous treatment with a hematopoietic stem-cell transplantation.
  3. Patient planned to undergo allogeneic hematopoietic stem cell transplantation
  4. Known presence of a BCR::ABL1 mutation with known resistance to study treatment in accordance with the most recent public version of international CML clinical guidelines (e.g. NCCN CML treatment guidelines v 1.2026 and Apperley et al 2025) any time prior to study entry

Other 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：Single arm study

This study will enroll pediatric patients (≥ 1 and < 18 years of age) with newly diagnosed or previously treated with Philadelphia positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) with or without known T315I mutation
药品：Asciminib single agent

Asciminib (labelled as ABL001) administered as 40 mg tablet (adult formulation) or as 1 mg film-coated granules mini-tablets (pediatric formulation)

其他名称：
  • ABL001

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
MMR at Week 48
大体时间：48 weeks

MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
48 weeks

次要结果测量

结果测量
措施说明
大体时间
疾病进展时间
大体时间：5年
5年
MMR at Week 96
大体时间：96 weeks

MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
96 weeks
MMR at and by scheduled timepoints
大体时间：Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96

MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96
Hematologic response at and by scheduled timepoints
大体时间：Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96

Complete hematologic response (CHR) is defined as all of the following present for ≥ 4 weeks (which means present at least at 2 visits 4-week apart, with no intermediate visit showing no CHR):

  • White blood cell(s) (WBC) count < 10 x 10^9/L
  • Platelet count < 450 x 10^9/L
  • Basophils < 5%
  • No blasts and promyelocytes in peripheral blood
  • Myelocytes + metamyelocytes < 5% in peripheral blood
  • No evidence of extramedullary disease, including spleen and liver
Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96
Cytogenetic response at and by scheduled timepoints
大体时间：5 years

Cytogenetic response is defined as follows:

  • Complete (CCyR) - 0% Ph+ metaphases
  • Partial (PCyR) - >0 to 35% Ph+ metaphases
  • Major* (MCyR) - 0 to 35% Ph+ metaphases
  • Minor (mCyR) - >35 to 65% Ph+ metaphases
  • Minimal - >65 to 95% Ph+ metaphases
  • None - >95 to 100% Ph+ metaphases Bone marrow aspirate for cytogenetic analyses will be performed at screening, and upon unfavorable response to treatment at the physician's discretion.
5 years
Time to response
大体时间：240 weeks after the last participant enrolled in the study received the first dose of treatment

Time to the first response is defined as: date of the first documented occurrence of the response or time from first study drug intake to first response - date of the first study treatment intake + 1.
240 weeks after the last participant enrolled in the study received the first dose of treatment
Duration of response (limited to the binary response endpoints)
大体时间：240 weeks after the last participant enrolled in the study received the first dose of treatment

Duration of response is defined as the time between the date of the first documented occurrence of the response and the earliest date of confirmed loss of the response, progression to accelerated phase/blast crisis (AP/BC) or CML-related death for participants in the respective Responder Set.
240 weeks after the last participant enrolled in the study received the first dose of treatment
Time to treatment failure (TTF)
大体时间：240 weeks after the last participant enrolled in the study received the first dose of treatment

TTF is defined as the time from the first study drug intake to the first/earliest documented date of any of the following events: unfavorable response to treatment, confirmed loss of MMR at any time while on study treatment and discontinuation from study treatment due to any reason.
240 weeks after the last participant enrolled in the study received the first dose of treatment
Event-free survival (EFS)
大体时间：240 weeks after the last participant enrolled in the study received the first dose of treatment

EFS is defined as the time from the first study drug intake to the earliest occurrence of the following events: unfavorable response to treatment, confirmed loss of MMR at any time while on study treatment, discontinuation from the study treatment due to an adverse event (AE) and death due to any cause.
240 weeks after the last participant enrolled in the study received the first dose of treatment
Overall survival (OS)
大体时间：240 weeks after the last participant enrolled in the study received the first dose of treatment

OS is defined as the time from the first study drug intake to the date of death from any cause during the study (including death observed during the survival follow-up period after discontinuation of study treatment).
240 weeks after the last participant enrolled in the study received the first dose of treatment
Growth: Height/length, weight, bone age measured by X-Ray
大体时间：240 weeks after the last participant enrolled in the study received the first dose of treatment

To assess growth and sexual maturation. An X-ray of the left hand and wrist to assess bone age will be performed at screening and every 48 weeks until end of treatment (EOT). This assessment will no longer be required for participants once bone maturity in post-puberty stage is confirmed by the last X-ray.
240 weeks after the last participant enrolled in the study received the first dose of treatment
Sexual maturation: Height/length, weight, bone age measured by Tanner staging
大体时间：240 weeks after the last participant enrolled in the study received the first dose of treatment

To assess growth and sexual maturation. Tanner staging will be based on external genitalia, breast and pubic hair examination. Tanner staging will be assessed for all study participants at screening. Participants who do not have a baseline Tanner Stage of 5 will continue to be assessed every 24 weeks until achievement of Tanner Stage 5 on both secondary sexual characteristics or EOT, whichever comes first. Once a participant reaches stage 5, examinations do not need to be conducted anymore. For all male participants, Tanner Stage 5 will be achieved once the participant has demonstrated both stage 5 genitalia development and stage 5 pubic hair development. For all female participants, Tanner Stage 5 will be achieved once the participant has demonstrated both stage 5 breast development and stage 5 pubic hair development.
240 weeks after the last participant enrolled in the study received the first dose of treatment
PK parameters of asciminib: AUClast
大体时间：Week 2, Day 1 at 1, 3 and 6 hours post dose

AUClast: The AUC from time zero to the last measurable concentration sampling time.
Week 2, Day 1 at 1, 3 and 6 hours post dose
PK parameters of asciminib: AUCtau
大体时间：Week 2, Day 1: pre dose (0 hour), Week 2: Day 1 at 1, 2, 3, and 6 hours post-dose

AUCtau: The AUC calculated to the end of a dosing interval (tau) at steady-state.
Week 2, Day 1: pre dose (0 hour), Week 2: Day 1 at 1, 2, 3, and 6 hours post-dose
PK parameter of asciminib: Cmax
大体时间：Week 2, Day 1 at 1, 3, and 6 hours post dose

Cmax is the maximum (peak) observed plasma drug concentration after single dose administration.
Week 2, Day 1 at 1, 3, and 6 hours post dose
PK parameter of asciminib: Tmax
大体时间：Week 2, Day 1 at 1, 3, and 6 hours post dose

Tmax is the time to reach maximum (peak) plasma drug concentration (h) after administration.
Week 2, Day 1 at 1, 3, and 6 hours post dose
PK parameter of asciminib: Ctrough
大体时间：Week 2, Day 1: pre-dose (0 hr), Week 24: pre-dose (0 hr), Week 48: Pre-dose (0 hr)

Ctrough refers to the lowest concentration a drug reaches in the bloodstream immediately before the next dose is administered.
Week 2, Day 1: pre-dose (0 hr), Week 24: pre-dose (0 hr), Week 48: Pre-dose (0 hr)

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)

2026年4月28日

初级完成 (估计的)

2033年2月23日

研究完成 (估计的)

2033年2月23日

研究注册日期

首次提交

2026年1月15日

首先提交符合 QC 标准的

2026年1月16日

首次发布 (实际的)

2026年1月21日

研究记录更新

最后更新发布 (实际的)

2026年11月10日

上次提交的符合 QC 标准的更新

2026年11月9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

  • CABL001I12202
  • 2025-522138-29 (注册表标识符：EU CTIS)
  • 2025 (美国 NIH 拨款/合同)

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

是的

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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