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

31. juli 2026 opdateret af: 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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

125

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Studiesteder

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

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

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: 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.
Gennemgå MR
Andre navne:
  • MR
  • Magnetisk resonans
  • Magnetisk resonansbilledscanning
  • Medicinsk billeddannelse, magnetisk resonans / kernemagnetisk resonans
  • HR
  • MR billeddannelse
  • MR-scanning
  • NMR billeddannelse
  • NMRI
  • Kernemagnetisk resonansbilleddannelse
  • Magnetisk resonansbilleddannelse (MRI)
  • sMRI
  • Magnetisk resonansbilleddannelse (procedure)
  • MRI'er
  • Strukturel MR
Gennemgå CT
Andre navne:
  • CT
  • KAT
  • CAT-scanning
  • Beregnet aksial tomografi
  • Computerstyret aksial tomografi
  • Computerstyret tomografi
  • CT-scanning
  • tomografi
  • Computerstyret aksial tomografi (procedure)
  • Computerstyret tomografi (CT) scanning
  • Diagnostisk CAT -scanning
  • Diagnostic CAT Scan Service Type
Gennemgå blodprøvetagning
Andre navne:
  • Biologisk prøvesamling
  • Bioprøve indsamlet
  • Prøvesamling
  • Prøvekollektion
Gennemgå strålebehandling
Andre navne:
  • Kræftstrålebehandling
  • ENERGY_TYPE
  • Bestråle
  • Bestrålet
  • Bestråling
  • Stråling
  • Stråleterapi, NOS
  • Radioterapeutika
  • Strålebehandling
  • RT
  • Terapi, Stråling
  • Energitype
Gennemgå kirurgisk resektion
Andre navne:
  • Operation
  • Kirurgi
  • Operationstype
  • Kirurgisk
  • Kirurgisk indgreb
  • Kirurgiske indgreb
  • Kirurgiske procedurer
  • Type kirurgi
  • Kirurgi, NOS
Hjælpestudier
Givet nab-paclitaxel
Andre navne:
  • ABI-007
  • Abraxane
  • Albumin-bundet Paclitaxel
  • ABI 007
  • Albumin-stabiliseret nanopartikel Paclitaxel
  • Nanopartikel Albumin-bundet Paclitaxel
  • Nanopartikel Paclitaxel
  • Paclitaxel Albumin
  • paclitaxel albumin-stabiliseret nanopartikelformulering
  • Proteinbundet Paclitaxel
  • ABI007
  • Paclitaxel proteinbundet
  • Paclitaxel Nanopartikel Albumin-bundet
  • Naveruclif
Givet gemcitabin
Andre navne:
  • dFdCyd
  • dFdC
  • Difluordeoxycytidin
Eksperimentel: 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.
Gennemgå MR
Andre navne:
  • MR
  • Magnetisk resonans
  • Magnetisk resonansbilledscanning
  • Medicinsk billeddannelse, magnetisk resonans / kernemagnetisk resonans
  • HR
  • MR billeddannelse
  • MR-scanning
  • NMR billeddannelse
  • NMRI
  • Kernemagnetisk resonansbilleddannelse
  • Magnetisk resonansbilleddannelse (MRI)
  • sMRI
  • Magnetisk resonansbilleddannelse (procedure)
  • MRI'er
  • Strukturel MR
Gennemgå CT
Andre navne:
  • CT
  • KAT
  • CAT-scanning
  • Beregnet aksial tomografi
  • Computerstyret aksial tomografi
  • Computerstyret tomografi
  • CT-scanning
  • tomografi
  • Computerstyret aksial tomografi (procedure)
  • Computerstyret tomografi (CT) scanning
  • Diagnostisk CAT -scanning
  • Diagnostic CAT Scan Service Type
Gennemgå blodprøvetagning
Andre navne:
  • Biologisk prøvesamling
  • Bioprøve indsamlet
  • Prøvesamling
  • Prøvekollektion
Gennemgå strålebehandling
Andre navne:
  • Kræftstrålebehandling
  • ENERGY_TYPE
  • Bestråle
  • Bestrålet
  • Bestråling
  • Stråling
  • Stråleterapi, NOS
  • Radioterapeutika
  • Strålebehandling
  • RT
  • Terapi, Stråling
  • Energitype
Gennemgå kirurgisk resektion
Andre navne:
  • Operation
  • Kirurgi
  • Operationstype
  • Kirurgisk
  • Kirurgisk indgreb
  • Kirurgiske indgreb
  • Kirurgiske procedurer
  • Type kirurgi
  • Kirurgi, NOS
Hjælpestudier
Givet nab-paclitaxel
Andre navne:
  • ABI-007
  • Abraxane
  • Albumin-bundet Paclitaxel
  • ABI 007
  • Albumin-stabiliseret nanopartikel Paclitaxel
  • Nanopartikel Albumin-bundet Paclitaxel
  • Nanopartikel Paclitaxel
  • Paclitaxel Albumin
  • paclitaxel albumin-stabiliseret nanopartikelformulering
  • Proteinbundet Paclitaxel
  • ABI007
  • Paclitaxel proteinbundet
  • Paclitaxel Nanopartikel Albumin-bundet
  • Naveruclif
Givet gemcitabin
Andre navne:
  • dFdCyd
  • dFdC
  • Difluordeoxycytidin
Givet oxaliplatin
Andre navne:
  • 1-OHP
  • Dakotin
  • Dacplat
  • Eloxatin
  • Ai Heng
  • Aiheng
  • Diaminocyclohexan Oxalatoplatin
  • JM-83
  • Oxalatoplatin
  • RP 54780
  • RP-54780
  • SR-96669
  • SR96669
  • Elplat
  • JM 83
  • JM83
  • RP54780
  • SR 96669
Givet fluorouracil
Andre navne:
  • 5-Fluracil
  • Fluracil
  • 5 Fluorouracil
  • 5 Fluorouracilum
  • 5 FU
  • 5-fluor-2,4(1H,3H)-pyrimidindion
  • 5-Fluorouracil
  • 5-Fu
  • 5FU
  • AccuSite
  • Carac
  • Fluor Uracil
  • Fluouracil
  • Flurablastin
  • Fluracedyl
  • Fluril
  • Fluroblastin
  • Ribofluor
  • Ro 2-9757
  • Ro-2-9757
Given irinotecan
Given leucovorin
Andre navne:
  • Folinsyre

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Proportion of patients who achieve R0/R1 surgical resection (Cohort A)
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Trial participation rate
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Samarbejdspartnere

Efterforskere

  • Ledende efterforsker: Brett L Ecker, MD, Northwestern University

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

3. februar 2027

Primær færdiggørelse (Anslået)

3. februar 2030

Studieafslutning (Anslået)

3. februar 2031

Datoer for studieregistrering

Først indsendt

31. juli 2026

Først indsendt, der opfyldte QC-kriterier

31. juli 2026

Først opslået (Faktiske)

6. august 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

6. august 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

31. juli 2026

Sidst verificeret

1. juli 2026

Mere information

Begreber relateret til denne undersøgelse

Andre undersøgelses-id-numre

  • NU 26I01 (Anden identifikator: Northwestern University)
  • P30CA060553 (U.S. NIH-bevilling/kontrakt)
  • NCI-2026-05293 (Registry Identifier: CTRP (Clinical Trial Reporting Program))
  • STU00226144

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .