Asciminib-Based Induction Therapy for the Treatment of Newly Diagnosed Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia
A Phase 1 Study of Asciminib-Based Induction Therapy in Newly Diagnosed Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ ALL)
This phase I trial studies the side effects and best dose of asciminib when given together with intrathecal (IT) chemotherapy, dexamethasone, vincristine, and blinatumomab (asciminib-based induction therapy) in treating patients with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL). Asciminib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Chemotherapy drugs, such as methotrexate and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Dexamethasone is in a class of medications called corticosteroids. It is used to reduce inflammation and lower the body's immune response to help lessen the side effects of chemotherapy drugs. Vincristine is in a class of medications called vinca alkaloids. It works by stopping cancer cells from growing and dividing and may kill them. Blinatumomab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. A monoclonal antibody is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Giving asciminib-based induction therapy may be safe, tolerable, and/or effective in treating patients with newly diagnosed Ph+ ALL.
研究概览
地位
详细说明
PRIMARY OBJECTIVES:
I. Evaluate the safety of asciminib as the tyrosine kinase inhibitor (TKI) backbone in a chemotherapy minimizing induction strategy (dexamethasone, vincristine, IT chemotherapy, blinatumomab).
II. Determine the recommended phase 2 dose (RP2D) of asciminib induction therapy as the TKI backbone in a reduced intensity strategy for the treatment of Ph+ ALL.
SECONDARY OBJECTIVES:
I. To evaluate the safety and tolerability of an asciminib-based therapy for post-dose limiting toxicity (DLT) cycles.
II. To evaluate the complete molecular response (CMR) rate after 1 and 3 cycles.
III. To evaluate the complete response (CR)/CR with incomplete hematologic recovery (CRi) rate after 1 and 3 cycles.
IV. To evaluate 1-year overall survival and event-free survival rates. V. To evaluate the rate of transplant in patients achieving CR in cycle 1 (CR1).
VI. To evaluate the pharmacokinetics of asciminib in this combination. VII. To evaluate minimal residual disease negativity (MRD-) by clonoSEQ assay (clonoSEQ) after first and third cycles of induction.
EXPLORATORY OBJECTIVE:
I. Evaluate novel molecular minimal residual disease (MRD) status in plasma and cerebrospinal fluid (CSF).
OUTLINE: This is a dose-escalation study of asciminib in combination with triple IT chemotherapy, dexamethasone, vincristine, and blinatumomab.
Patients receive asciminib orally (PO) once daily (QD) or twice daily (BID) on days 1-28 of cycles 1 and 2 and days 1-42 of cycle 3, triple IT chemotherapy (methotrexate, cytarabine, corticosteroid) on days 1 and 14 of cycles 1 and 2 and days 1, 14, 28, and 42 of cycle 3, dexamethasone PO on days 1-4 and 11-14 of cycle 1, vincristine intravenously (IV) on days 1 and 14 of cycle 1, and blinatumomab IV on days 1-28 of cycle 2 and days 15-42 of cycle 3. Cycles repeat every 28 days (cycles 1 and 2) or 42 days (cycle 3) for up to 3 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo echocardiography (ECHO) during screening, blood and CSF sample collection on study, and bone marrow aspiration throughout the study.
After completion of study treatment, patients are followed up at 30 days and then every 3 months for up to 1 year post-treatment start.
研究类型
注册 (估计的)
阶段
- 阶段1
联系人和位置
学习地点
-
-
California
-
Duarte、California、美国、91010
- City of Hope Medical Center
-
首席研究员:
- Paul Koller
-
接触:
- Paul Koller
- 电话号码:626-218-2405
- 邮箱:pkoller@coh.org
-
-
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:
-
Documented informed consent of the participant and/or legally authorized representative
-
Newly diagnosed Ph+ or BCR-ABL1-positive ALL, as defined by the 2025 National Comprehensive Cancer Network (NCCN) guidelines
-
Age: ≥ 18 years
-
Eastern Cooperative Oncology Group (ECOG) ≤ 2
-
Total bilirubin ≤ 1.5 X upper limit of normal (ULN) (unless has Gilbert's disease)
-
Aspartate aminotransferase (AST) ≤ 3.0 x ULN
-
Alanine aminotransferase (ALT) ≤ 3.0 x ULN
-
Serum creatinine < 1.5 mg/dL
-
Left ventricular ejection fraction (LVEF) ≥ 50%
- Note: To be performed within 28 days prior to day 1 of protocol therapy
-
Normal QT interval corrected per Fridericia method (QTcF) on screening electrocardiogram, defined as QTcF of ≤ 480 msec
-
Oxygen (O2) saturation > 90% on room air
-
Seronegative for HIV antigen (Ag)/antibody (Ab) combo, hepatitis C virus (HCV), active hepatitis B virus (HBV) (surface antigen negative)
- If seropositive for HIV, HCV or HBV, nucleic acid quantitation must be performed. Viral load must be undetectable. HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
-
Meets other institutional and federal requirements for infectious disease titer requirements
- Note Infectious disease testing to be performed within 28 days prior to day 1 of protocol therapy
-
Women of childbearing potential (WOCBP): negative urine or serum pregnancy test
- If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
-
Agreement by females and males of childbearing potential to use a highly effective method of birth control or abstain from heterosexual activity for the course of the study through at least 120 days after the last dose of protocol therapy
- Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for > 1 year (women only)
Exclusion Criteria:
-
Prior treatment failure with asciminib
-
Prior/current treatment with any systemic anticancer therapy (including but not limited to any TKI) and/or radiotherapy for ALL, with the exception of an optional prephase therapy or chemotherapy induction (no more than one cycle), which should be discussed with the principal investigator (PI). Patients who have received no more than two weeks of chemotherapy (not including steroids) with or without other TKIs are considered minimally pretreated and still eligible
-
Currently taking drugs that are known to have a risk of causing prolonged corrected QT interval (QTc) or torsades depointes (TdP) (unless these can be changed to acceptable alternatives or discontinued)
-
Taking any medications or herbal supplements that are known to be strong inhibitors or strong inducers of cytochrome P450 (CYP)3A4 within at least 14 days before the first dose of study drug
-
Cardiac or cardiac repolarization abnormality, including any of the following:
- History within 6 months prior to starting study treatment of myocardial infarction (MI), or coronary artery bypass graft (CABG)
- Clinically significant cardiac arrhythmias (e.g., ventricular tachycardia), complete left bundle branch block, high-grade atrioventricular (AV) block (e.g., bifascicular block, Mobitz type II and third-degree AV block)
-
A history of cerebrovascular events such as stroke or transient ischemic attack
-
Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome. Treatment with any investigational products within 30 days before enrollment or 6 half-lives of the agent, whichever is shorter
-
History of pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis
-
Uncontrolled hypertriglyceridemia (triglycerides > 450 mg/dL)
-
Clinically significant uncontrolled illness
-
Active infection not responding to treatment at the time of starting therapy
-
Autoimmune disease with potential central nervous system (CNS) involvement
-
Other active malignancy. Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
-
Females only: Pregnant or breastfeeding
-
Any other condition that would, in the investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures
-
Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)
学习计划
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:Treatment (asciminib-based induction therapy)
Patients receive asciminib PO QD or BID on days 1-28 of cycles 1 and 2 and days 1-42 of cycle 3, triple IT chemotherapy on days 1 and 14 of cycles 1 and 2 and days 1, 14, 28, and 42 of cycle 3, dexamethasone PO on days 1-4 and 11-14 of cycle 1, vincristine IV on days 1 and 14 of cycle 1, and blinatumomab IV on days 1-28 of cycle 2 and days 15-42 of cycle 3. Cycles repeat every 28 days (cycles 1 and 2) or 42 days (cycle 3) for up to 3 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo ECHO during screening, blood and CSF sample collection on study, and bone marrow aspiration throughout the study. |
Given IV
其他名称:
Undergo ECHO
其他名称:
Given PO
其他名称:
Given IV
其他名称:
Undergo bone marrow aspiration Given IT
其他名称:
Undergo blood and CSF sample collection
其他名称:
Given PO
其他名称:
Given IT Given IT
其他名称:
|
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 |
|---|---|---|
|
Incidence of dose limiting toxicity (DLT)
大体时间:Up to 2 cycles (Cycle length = 28 days)
|
Toxicity will be graded according to the National Cancer Institute-Common Terminology Criteria for Adverse Events (CTCAE), version (v) 6.0. |
Up to 2 cycles (Cycle length = 28 days)
|
|
Maximum tolerated dose/recommended phase 2 dose
大体时间:Up to the 18.6 months
|
Will be established using a phase 1 queue 3+3 design. |
Up to the 18.6 months
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Incidence of adverse events (AEs)
大体时间:Up to 30 days after last dose of study drug
|
The number and percent of patients with AEs will be tabulated by system organ class and preferred term using the Medical Dictionary for Regulatory Activities preferred term dictionary. Observed toxicities will be summarized for the DLT period (cycles 1 and 2), as well as for each of the 3 cycles, in terms of type (organ affected or laboratory determination), severity, time of onset, duration, frequency, probable association with each study treatment and reversibility or outcome. Listings and narratives will be provided for deaths on study, DLTs/unacceptable toxicities, serious adverse events, and events leading to discontinuation of treatment. Descriptive summaries will also be generated for AE data assessed and graded according to the CTCAE v6.0 grading system. |
Up to 30 days after last dose of study drug
|
|
Complete molecular response
大体时间:At the completion of cycle 1 (Cycle length = 28 days) and cycle 3 (Cycle length = 42 days)
|
Will be assessed in accordance with the 2025 National Comprehensive Cancer Network (NCCN) guidelines at each dose level and disease assessment timepoint (cycles 1 and 3). Rates and 95% Clopper-Pearson binomial confidence interval (CI) will be calculated. |
At the completion of cycle 1 (Cycle length = 28 days) and cycle 3 (Cycle length = 42 days)
|
|
Complete response (CR)/CR with incomplete hematologic recovery
大体时间:At the completion of cycle 1 (Cycle length = 28 days) and cycle 3 (Cycle length = 42 days)
|
Will be assessed in accordance with the 2025 NCCN guidelines at each dose level and disease assessment timepoint (cycles 1 and 3). Rates and 95% Clopper-Pearson binomial CI will be calculated. |
At the completion of cycle 1 (Cycle length = 28 days) and cycle 3 (Cycle length = 42 days)
|
|
Minimal residual disease negativity
大体时间:At the completion of cycle 1 (Cycle length = 28 days) and cycle 3 (Cycle length = 42 days)
|
Will be assessed in accordance with the 2025 NCCN guidelines at each dose level and disease assessment timepoint (cycles 1 and 3). Rates and 95% Clopper-Pearson binomial CI will be calculated. |
At the completion of cycle 1 (Cycle length = 28 days) and cycle 3 (Cycle length = 42 days)
|
|
Rate of allogeneic hematopoietic cell transplantation
大体时间:Up to 1 cycle (Cycle length = 28 days)
|
Rates and 95% Clopper-Pearson binomial CI will be calculated. |
Up to 1 cycle (Cycle length = 28 days)
|
|
Overall survival (OS)
大体时间:From first study dose to death from any cause or last contact date, whichever comes first, assessed up to 1 year
|
Median OS and 1-year OS rate, will be estimated using the product-limit method of Kaplan-Meier product-limit method and the 95% C.I. of the survival curve will be calculated by the Brookmeyer-Crowley method with log-log transformation. |
From first study dose to death from any cause or last contact date, whichever comes first, assessed up to 1 year
|
|
Progression-free survival (PFS)
大体时间:Up to 1 year
|
Median PFS and 1-year PFS rate, will be estimated using the product-limit method of Kaplan-Meier product-limit method and the 95% C.I. of the survival curve will be calculated by the Brookmeyer-Crowley method with log-log transformation. |
Up to 1 year
|
|
Concentration of asciminib (Pharmacokinetics)
大体时间:Up to 98 days (Cycle 1 and 2 are 28 days each, Cycle 3 is 42 days)
|
Will be assessed over time by descriptive and graphical statistics. |
Up to 98 days (Cycle 1 and 2 are 28 days each, Cycle 3 is 42 days)
|
合作者和调查者
调查人员
- 首席研究员:Paul Koller、City of Hope Medical Center
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
- 荷尔蒙
- 激素、激素替代品和激素拮抗剂
- 硫化合物
- 有机化学品
- 杂环化合物,1形
- 杂环化合物
- 杂环化合物,2环
- 杂环化合物,融合环
- 调查技术
- 临床实验室技术
- 诊断技术和程序
- 诊断
- 核酸,核苷酸和核苷
- 碳氢化合物
- 碳氢化合物,循环
- 生物碱
- 碳氢化合物,芳香族
- 多环化合物
- 吲哚
- 胞苷
- 嘧啶核苷
- 嘧啶
- 怀孕
- 怀孕
- 类固醇
- 融合环化合物
- 类固醇,氟化
- 苯衍生物
- 磺酸
- 硫酸
- 核苷
- 翼
- 翼状
- 疗法
- VINCA生物碱
- Secologanin色素生物碱
- 吲哚生物碱
- 吲哚衍生物
- 吲哚王
- 阿拉伯核苷
- 氨基翅目
- 苯磺酸盐
- 芳基磺酸盐
- 芳基磺酸
- 地塞米松
- 甲氨蝶呤
- 阿糖胞苷
- 长春新碱
- 羟苯磺酸钙
- 标本处理
- blinatumomab
- 地塞米松21-磷酸盐
- N,N-二甲苯己基 - 异位醇-10-磺胺酰胺
- 铜
- 耳原则
- 乙酸地塞米松
- 肾上腺皮质激素
- asciminib
其他研究编号
- 25618
- P30CA033572 (美国 NIH 拨款/合同)
- NCI-2026-07296 (注册表标识符:CTRP (Clinical Trial Reporting Program))
- 25618 (其他标识符:City of Hope Medical Center)
药物和器械信息、研究文件
研究美国 FDA 监管的药品
研究美国 FDA 监管的设备产品
在美国制造并从美国出口的产品
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.