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SMAD4 Tailored Neoadjuvant Therapy for the Treatment of Resectable and Borderline Resectable Pancreatic Ductal Adenocarcinoma, SMART-PANC Trial

2026年7月31日 更新者:Northwestern University

SMART-PANC: SMAD4 Tailored Neoadjuvant Therapy for Pancreatic Cancer, A Phase ll Non-Randomized, Single Center Pilot Study

This phase II trial tests the impact of using SMAD4-mutant status to personalize chemotherapy in treating patients with pancreatic ductal adenocarcinoma (PDAC) that can be removed by surgery (resectable) or that may be between resectable and unresectable (borderline resectable) before undergoing surgery (neoadjuvant). PDAC is one of the most aggressive and fastest growing tumors. SMAD4, a tumor suppressor gene, acts like a brake on cell growth and helps to prevent tumor cells from growing. However, losing it makes the tumor more aggressive and often resistant to standard of care (SOC) treatments regimens, such as gemcitabine with nab-paclitaxel and fluorouracil, leucovorin, irinotecan, and oxaliplatin (FOLFIRINOX). Gemcitabine is a chemotherapy drug that blocks the cells from making deoxyribonucleic (DNA) and may kill tumor cells. Paclitaxel is in a class of medications called antimicrotubule agents. It stops tumor cells from growing and dividing and may kill them. Nab-paclitaxel is an albumin-stabilized nanoparticle formulation of paclitaxel which may have fewer side effects and work better than other forms of paclitaxel. Fluorouracil, a type of antimetabolite, stops cells from making DNA and it may kill tumor cells. Leucovorin is a form of folic acid. It is a type of chemoprotective agent and a type of chemosensitizing agent. Irinotecan is in a class of antineoplastic medications called topoisomerase I inhibitors. It blocks a certain enzyme needed for cell division and DNA repair and may kill tumor cells. Oxaliplatin is in a class of medications called platinum-containing antineoplastic agents. It damages the cell's DNA and may kill tumor cells. Instead of a one-sized fits all treatment approach with SOC therapies, matching therapy based on SMAD4 mutation status may use this specific genetic weakness against the tumor. This approach to neoadjuvant therapy may dramatically alter the path of treatment and improve outcomes, including complete resection rates, in patients with resectable or borderline resectable PDAC.

研究概览

详细说明

PRIMARY OBJECTIVE:

I. To determine the R0/R1 surgical resection rate among patients with SMAD4-mutant pancreatic cancer treated with gemcitabine/nab-paclitaxel (Cohort A).

SECONDARY OBJECTIVES:

I. To estimate the trial participation rate among patients approached for enrollment, defined as the proportion of patients who consent and enroll among those approached, with a target participation rate of ≥ 70%, and to monitor this rate continuously during accrual.

II. To estimate the R0/R1 surgical resection rate among SMAD4 wild-type patients treated with physician choice chemotherapy (Cohort B).

III. To determine pathologic response rates, among the subset of patients who reach surgical resection.

IV. To determine the progression free survival (PFS). V. To determine the overall survival (OS).

EXPLORATORY OBJECTIVES:

I. To estimate the proportion of R0 resections among patients with SMAD4-mutant pancreatic cancer treated with gemcitabine/nab-paclitaxel (Cohort A) who undergo surgical resection (R0/R1).

II. To estimate the proportion of R0 resections among patients with SMAD4 wild-type patients treated with physician choice chemotherapy (Cohort B) who undergo surgical resection (R0/R1).

III. To compare R0/R1 resection rates between Cohort A (SMAD4-mutant, gemcitabine/nab-paclitaxel) and Cohort B (SMAD4 wild-type, physician choice) in a descriptive, exploratory manner; the study is not powered for formal non-inferiority or superiority testing of this comparison.

IV. To determine the preoperative/neoadjuvant ca19-9 dynamics in both cohorts. V. To determine the preoperative/neoadjuvant circulating tumor DNA (ctDNA) dynamics in both cohorts.

VI. To assess reason for failure to reach surgical resection, categorized by clinical deterioration, patient withdrawal/refusal of surgery, metastatic progression, or local disease progression precluding surgery.

OUTLINE: Patients with SMAD4 alterations are assigned to Cohort A and patients without SMAD4 mutations are assigned to Cohort B.

COHORT A: Patients receive gemcitabine and nab-paclitaxel with or without radiation therapy per SOC. Treatment continues for 8 weeks in the absence of disease progression or unacceptable toxicity. After 8 week restaging, patients may continue to receive treatment for up to 16 or 24 weeks at the discretion of the multidisciplinary tumor board. Starting 21 days after last dose of chemotherapy, patients undergo surgical resection. Additionally, patients undergo blood sample collection and computed tomography (CT) or magnetic resonance imaging (MRI) as clinically indicated throughout the study.

COHORT B: Patients receive either physicians choice of either fluorouracil, leucovorin, irinotecan, and oxaliplatin or gemcitabine and nab-paclitaxel with or without radiation therapy per SOC. Treatment continues for 8 weeks in the absence of disease progression or unacceptable toxicity. After 8 week restaging, patients may continue to receive treatment for up to 16 or 24 weeks at the discretion of the multidisciplinary tumor board. Starting 21 days after last dose of chemotherapy, patients undergo surgical resection. Additionally, patients undergo blood sample collection and CT or MRI as clinically indicated throughout the study.

After completion of study treatment, patients are followed up at 30 days from surgical resection then every 3 months for up to 2 years from study enrollment.

研究类型

介入性

注册 (估计的)

125

阶段

  • 阶段2

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

学习地点

    • Illinois
      • Chicago、Illinois、美国、60611

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

  • 成人
  • 年长者

接受健康志愿者

不

描述

Inclusion Criteria:

  • Patients must have histologically confirmed pancreas ductal adenocarcinoma

    • Most recent cross-sectional imaging of the chest, abdomen and pelvis will be used to rule out distant metastases (this will have been completed within standard of care timelines which will typically fall within 90 days of registration)
  • Patients must have undergone next generation sequencing (NGS) testing on the pre-treatment tumor tissue and must have either SMAD4 mutant or SMAD4 wild-type mutation identified before consenting for this study

    • Note: Before consenting to this study, standard of care NGS procedure will be performed on pre-treatment tumor biopsies that are routinely collected through endoscopic biopsy specimens (in-house Oncomine Precision NGS). The results will be documented for this study from clinic notes
  • Patients must have resectable/borderline-resectable disease

    • Note: National Comprehensive Cancer Network (NCCN) definitions of resectability will be used
  • Patients must be treatment-naïve and clinically fit and eligible for either FOLFIRINOX or gemcitabine/nab-paclitaxel chemotherapy regimens (per treating physician's determination)
  • Patients must be ≥ 18 years of age
  • Patients must exhibit an Eastern Cooperative Oncology Group (ECOG) performance status of 0-1
  • Patients must have adequate organ and bone marrow function as determined by the treating physician to be considered to be clinically fit for the physician-determined chemotherapy regimen
  • Patients must agree to use adequate contraception: (e.g. hormonal or barrier method of birth control for a patient of child-bearing potential (POCBP), prior to study entry, and for the duration of study participation. Should a female patient, or a female partner of a patient become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician and study doctor immediately. Male patients with a female partner(s) of childbearing potential must agree to use highly effective contraceptive measures throughout the study starting with the screening visit through the time period indicated for standard of care drugs , after the last dose of study treatment is received. Males with pregnant partners must agree to use a condom; no additional method of contraception is required for the pregnant partner. Note: A POCBP is any patient (regardless of gender, sexual orientation, having undergone a tubal ligation, or remaining celibate by choice) with an egg-producing reproductive tract who meets the following criteria:

    • Has not undergone a hysterectomy or bilateral oophorectomy
    • Has had menses at any time in the preceding 12 consecutive months (and therefore has not been naturally postmenopausal for > 12 months)
  • POCBP must have a negative pregnancy test prior to registration on study
  • Patients must have the ability to understand and the willingness to sign a written informed consent document and comply with the study requirements

Exclusion Criteria:

  • Patients with the presence of any of the following genetic or molecular abnormalities:

    • Mismatch repair (MMR)-deficiency/Lynch syndrome
    • High-frequency microsatellite instability (MSI-H)
    • Homologous recombination deficiency (HRD)
  • Patients who are currently participating in another study and receiving a study drug
  • Patients who have not recovered from adverse events due to prior anti-cancer therapy (i.e., have residual toxicities > grade 1) with the exception of alopecia, neuropathy and other non-significant adverse events per National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version (v) 6.0
  • Patients with a prior or concurrent malignancy whose natural history or treatment has the potential to interfere with the safety or efficacy assessment of the investigational regimen (per treating physician's determination)
  • Patients who are pregnant or nursing
  • Patients who have an uncontrolled intercurrent illness (as determined by treating physician), including, but not limited to any of the following:

    • Hypertension that is not controlled on medication
    • Active infection requiring treatment
    • Symptomatic congestive heart failure
    • Unstable angina pectoris
    • Cardiac arrhythmia
  • Patient with presence of any concurrent medical or psychiatric condition/social situations which would make them inappropriate candidates for entry into this study or that would limit compliance with study requirements, or would compromise the patient's safety or study endpoints, in the treating investigator's judgment

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:非随机化
  • 介入模型:并行分配
  • 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:Cohort A (gemcitabine, nab-paclitaxel)
Patients receive gemcitabine and nab-paclitaxel with or without radiation therapy per SOC. Treatment continues for 8 weeks in the absence of disease progression or unacceptable toxicity. After 8 week restaging, patients may continue to receive treatment for up to 16 or 24 weeks at the discretion of the multidisciplinary tumor board. Starting 21 days after last dose of chemotherapy, patients undergo surgical resection. Additionally, patients undergo blood sample collection and CT or MRI as clinically indicated throughout the study.
进行核磁共振
其他名称:
  • 核磁共振
  • 磁共振
  • 磁共振成像扫描
  • 医学影像、磁共振/核磁共振
  • 先生
  • 磁共振成像
  • 核磁共振成像扫描
  • 核磁共振成像
  • 磁共振成像 (MRI)
  • 磁共振成像(程序)
  • 结构核磁共振
接受CT
其他名称:
  • CT
  • 猫
  • 电脑扫描
  • 计算机轴向断层扫描
  • 计算机断层扫描
  • CT扫描
  • 断层扫描
  • 计算机轴向断层扫描(程序)
  • 计算机断层扫描 (CT) 扫描
  • 诊断猫扫描
  • 诊断猫扫描服务类型
进行血液样本采集
其他名称:
  • 生物样本采集
  • 收集的生物样本
  • 标本采集
  • 样本收集
接受放射治疗
其他名称:
  • 癌症放疗
  • 能源类型
  • 照射
  • 辐照
  • 辐射
  • 放射治疗,NOS
  • 放疗
  • 转播
  • 治疗、放射
  • 能量类型
接受手术切除
其他名称:
  • 手术
  • 手术类型
  • 外科
  • 手术治疗
  • 手术干预
  • 外科手术
  • 手术,NOS
辅助研究
给予白蛋白结合型紫杉醇
其他名称:
  • ABI-007
  • Abraxane
  • 白蛋白结合型紫杉醇
  • 阿比007
  • 白蛋白稳定的纳米粒子紫杉醇
  • 纳米颗粒白蛋白结合紫杉醇
  • 纳米紫杉醇
  • 紫杉醇白蛋白
  • 紫杉醇白蛋白稳定的纳米颗粒制剂
  • 蛋白结合型紫杉醇
  • ABI007
  • 紫杉醇蛋白结合
  • 紫杉醇纳米颗粒白蛋白结合
  • 纳韦鲁克利夫
给定吉西他滨
其他名称:
  • dFdCyd
  • dFdC
  • 二氟脱氧胞苷
实验性的:Cohort B (gemcitabine, nab-paclitaxel or FOLFIRINOX)
Patients receive either physicians choice of either fluorouracil, leucovorin, irinotecan, and oxaliplatin or gemcitabine and nab-paclitaxel with or without radiation therapy per SOC. Treatment continues for 8 weeks in the absence of disease progression or unacceptable toxicity. After 8 week restaging, patients may continue to receive treatment for up to 16 or 24 weeks at the discretion of the multidisciplinary tumor board. Starting 21 days after last dose of chemotherapy, patients undergo surgical resection. Additionally, patients undergo blood sample collection and CT or MRI as clinically indicated throughout the study.
进行核磁共振
其他名称:
  • 核磁共振
  • 磁共振
  • 磁共振成像扫描
  • 医学影像、磁共振/核磁共振
  • 先生
  • 磁共振成像
  • 核磁共振成像扫描
  • 核磁共振成像
  • 磁共振成像 (MRI)
  • 磁共振成像(程序)
  • 结构核磁共振
接受CT
其他名称:
  • CT
  • 猫
  • 电脑扫描
  • 计算机轴向断层扫描
  • 计算机断层扫描
  • CT扫描
  • 断层扫描
  • 计算机轴向断层扫描(程序)
  • 计算机断层扫描 (CT) 扫描
  • 诊断猫扫描
  • 诊断猫扫描服务类型
进行血液样本采集
其他名称:
  • 生物样本采集
  • 收集的生物样本
  • 标本采集
  • 样本收集
接受放射治疗
其他名称:
  • 癌症放疗
  • 能源类型
  • 照射
  • 辐照
  • 辐射
  • 放射治疗,NOS
  • 放疗
  • 转播
  • 治疗、放射
  • 能量类型
接受手术切除
其他名称:
  • 手术
  • 手术类型
  • 外科
  • 手术治疗
  • 手术干预
  • 外科手术
  • 手术,NOS
辅助研究
给予白蛋白结合型紫杉醇
其他名称:
  • ABI-007
  • Abraxane
  • 白蛋白结合型紫杉醇
  • 阿比007
  • 白蛋白稳定的纳米粒子紫杉醇
  • 纳米颗粒白蛋白结合紫杉醇
  • 纳米紫杉醇
  • 紫杉醇白蛋白
  • 紫杉醇白蛋白稳定的纳米颗粒制剂
  • 蛋白结合型紫杉醇
  • ABI007
  • 紫杉醇蛋白结合
  • 紫杉醇纳米颗粒白蛋白结合
  • 纳韦鲁克利夫
给定吉西他滨
其他名称:
  • dFdCyd
  • dFdC
  • 二氟脱氧胞苷
给定奥沙利铂
其他名称:
  • 1-OHP
  • 大古丁
  • 达克普拉
  • 依洛沙丁
  • 艾恒
  • 爱恒
  • 二氨基环己烷草酸铂
  • 依洛沙汀
  • JM-83
  • 草酸铂
  • 零售价 54780
  • RP-54780
  • SR-96669
  • SR96669
  • 埃尔普拉特
  • 杰米83
  • JM83
  • RP54780
  • SR 96669
给定氟尿嘧啶
其他名称:
  • 5-氟嘧啶
  • 氟嘧啶
  • 5氟尿嘧啶
  • 5 氟尿嘧啶
  • 5傅
  • 5-Fluoro-2,4(1H, 3H)-pyrimidinedione
  • 5-氟尿嘧啶
  • 5福
  • 5FU
  • 精确站点
  • 卡拉克
  • 氟尿嘧啶
  • 氟黄素
  • 氟里尔
  • 氟芽素
  • 氟核糖
  • 罗 2-9757
  • Ro-2-9757
Given irinotecan
Given leucovorin
其他名称:
  • 亚叶酸

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
Proportion of patients who achieve R0/R1 surgical resection (Cohort A)
大体时间:Up to 30 days from surgical resection
Successful surgical resection is defined as macroscopically complete removal of the primary pancreatic tumor, classified as R0 or R1. Will follow the Simon's minimax two-stage design decision rule. The observed R0/R1 resection rate will be summarized descriptively and reported with an exact (Clopper-Pearson) binomial confidence interval.
Up to 30 days from surgical resection

次要结果测量

结果测量
措施说明
大体时间
Trial participation rate
大体时间:Up to 2 years
Will be defined as the proportion of patients who consent and enroll among all patients who are approached and eligible for trial participation. Will be summarized using proportions with exact confidence intervals. Feasibility monitoring will occur on an ongoing basis during accrual (e.g. monthly or after every 20 approached eligible patients, whichever occurs first) using cumulative participation rate.
Up to 2 years
Proportion of patients who achieve R0/R1 surgical resection rate (Cohort B)
大体时间:Up to 30 days after surgical resection
Among participants who undergo R0/R1 surgical resection, the proportion achieving R0 resection will be summarized.
Up to 30 days after surgical resection
Proportion of patients who achieve R0/R1 surgical resection rate (Cohort A)
大体时间:Up to 30 days after surgical resection
Among participants who undergo R0/R1 surgical resection, the proportion achieving R0 resection will be summarized.
Up to 30 days after surgical resection
Pathologic response rates
大体时间:Up to 30 days after surgical resection
Pathology reports will be reviewed to determine the College of American Pathologists (CAP) tumor regression score, categorized from 0 (complete response) to 3 (poor or no response). CAP tumor regression scores will be summarized descriptively as frequencies and percentages across categories. The distribution of reasons for failure to reach surgical resection will be summarized descriptively using frequencies and percentages. These summaries may be further stratified by baseline resectability status and SMAD4 mutation status to provide clinical context and inform future trial design.
Up to 30 days after surgical resection
Progression free survival (PFS)
大体时间:From enrollment to disease progression or clinical deterioration that precludes complete surgical resection, disease recurrence after surgery, or death from any cause, assessed up to 2 years
PFS will be analyzed using Kaplan-Meier methods. The median PFS and corresponding 95% confidence interval will be estimated.
From enrollment to disease progression or clinical deterioration that precludes complete surgical resection, disease recurrence after surgery, or death from any cause, assessed up to 2 years
Overall survival (OS)
大体时间:From enrollment to the date of any cause of death or to the date of last follow-up, assessed up to 2 years
OS will be analyzed using Kaplan-Meier methods. Median OS and corresponding 95% confidence intervals will be reported.
From enrollment to the date of any cause of death or to the date of last follow-up, assessed up to 2 years

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。

调查人员

  • 首席研究员:Brett L Ecker, MD、Northwestern University

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 (估计的)

2027年2月3日

初级完成 (估计的)

2030年2月3日

研究完成 (估计的)

2031年2月3日

研究注册日期

首次提交

2026年7月31日

首先提交符合 QC 标准的

2026年7月31日

首次发布 (实际的)

2026年8月6日

研究记录更新

最后更新发布 (实际的)

2026年8月6日

上次提交的符合 QC 标准的更新

2026年7月31日

最后验证

2026年7月1日

更多信息

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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