SMAD4 Tailored Neoadjuvant Therapy for the Treatment of Resectable and Borderline Resectable Pancreatic Ductal Adenocarcinoma, SMART-PANC Trial
SMART-PANC: SMAD4 Tailored Neoadjuvant Therapy for Pancreatic Cancer, A Phase ll Non-Randomized, Single Center Pilot Study
Descripción general del estudio
Estado
Estado
Condiciones
Condiciones
Intervención / Tratamiento
Intervención / Tratamiento
- Procedimiento: Imagen de resonancia magnética
- Procedimiento: Tomografía computarizada
- Procedimiento: Colección de muestras biológicas
- Radiación: Radioterapia
- Procedimiento: Procedimiento quirúrgico
- Otro: Revisión de Historia Clínica Electrónica
- Droga: Nab-paclitaxel
- Droga: Gemcitabina
- Droga: Oxaliplatino
- Droga: Fluorouracil
- Droga: Irinotecan
- Droga: Leucovorin
Descripción detallada
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.
Tipo de estudio
Tipo de estudio
Inscripción (Estimado)
Inscripción
Fase
Fase
- Fase 2
Contactos y Ubicaciones
Estudio Contacto
Estudio Contacto
- Nombre: Study Coordinator
- Número de teléfono: 3126951301
- Correo electrónico: cancer@northwestern.edu
Ubicaciones de estudio
-
-
Illinois
-
Chicago, Illinois, Estados Unidos, 60611
- Northwestern University
-
Contacto:
- Brett L. Ecker, MD
- Correo electrónico: Brett.Ecker@northwestern.edu
-
Investigador principal:
- Brett L. Ecker, MD
-
-
Criterios de participación
Criterio de elegibilidad
Criterio de elegibilidad
Edades elegibles para estudiar
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Descripción
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
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: No aleatorizado
- Modelo Intervencionista: Asignación paralela
- Enmascaramiento: Ninguno (etiqueta abierta)
Número de brazos
Armas e Intervenciones
Grupo de participantes/brazoGrupo de participantes/brazo |
Intervención / TratamientoIntervención / Tratamiento |
|---|---|
|
Experimental: 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.
|
Someterse a una resonancia magnética
Otros nombres:
Someterse a una tomografía computarizada
Otros nombres:
Someterse a la recolección de muestras de sangre
Otros nombres:
Someterse a radioterapia
Otros nombres:
Someterse a una resección quirúrgica
Otros nombres:
Estudios complementarios
Dado nab-paclitaxel
Otros nombres:
Dada Gemcitabine
Otros nombres:
|
|
Experimental: 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.
|
Someterse a una resonancia magnética
Otros nombres:
Someterse a una tomografía computarizada
Otros nombres:
Someterse a la recolección de muestras de sangre
Otros nombres:
Someterse a radioterapia
Otros nombres:
Someterse a una resección quirúrgica
Otros nombres:
Estudios complementarios
Dado nab-paclitaxel
Otros nombres:
Dada Gemcitabine
Otros nombres:
Oxaliplatino dado
Otros nombres:
Dado fluorouracilo
Otros nombres:
Given irinotecan
Given leucovorin
Otros nombres:
|
¿Qué mide el estudio?
Medidas de resultado primarias
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Proportion of patients who achieve R0/R1 surgical resection (Cohort A)
Periodo de tiempo: 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
|
Medidas de resultado secundarias
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Trial participation rate
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
|
Colaboradores e Investigadores
Patrocinador
Patrocinador
Colaboradores
Colaboradores
Investigadores
Investigadores
- Investigador principal: Brett L Ecker, MD, Northwestern University
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Estimado)
Inicio del estudio
Finalización primaria (Estimado)
Finalización primaria
Finalización del estudio (Estimado)
Finalización del estudio
Fechas de registro del estudio
Enviado por primera vez
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Publicado por primera vez
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización publicada
Última actualización enviada que cumplió con los criterios de control de calidad
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
- Aminoácidos, péptidos y proteínas
- Proteínas
- Químicos orgánicos
- Compuestos heterocíclicos, 1 anillo
- Compuestos heterocíclicos
- Compuestos heterocíclicos, 2 anillos
- Compuestos heterocíclicos, anillo fusionado
- Técnicas de investigación
- Terapéutica
- Técnicas de laboratorio clínico
- Técnicas y procedimientos de diagnóstico
- Diagnóstico
- Hidrocarburos
- Cicloparafinas
- Hidrocarburos, alicíclicos
- Hidrocarburos, cíclico
- Terpenos
- Fenómeno físico
- Campeptecina
- Alcaloides
- Enzimas y coenzimas
- Complejos de coordinación
- Taxaides
- Ciclodecanos
- Diterpenos
- Desoxicitidina
- Citidina
- Nucleósidos de pirimidina
- Pirimidinas
- Economía y organizaciones de la atención médica
- Técnicas de química, analítica
- Análisis de espectro
- Formiltetrahidrofolatos
- Tetrahidrofolatos
- Ácido fólico
- Pterins
- Pteridinas
- Uracílico
- Pirimidinonas
- Coenzimas
- Albúmina
- Paclitaxel
- Ciencias económicas
- Oxaliplatino
- Irinotecán
- Paclitaxel unido a albúmina
- Gemcitabina
- Fluorouracilo
- Leucovorina
- Radioterapia
- Radiación
- Manejo de muestras
- Espectroscopía de resonancia magnética
- Procedimientos quirúrgicos, operativo
- Paclitaxel de 130 nm en albúmina
- deshidroftorafur
- Impuestos
Otros números de identificación del estudio
Otros números de identificación del estudio
- NU 26I01 (Otro identificador: Northwestern University)
- P30CA060553 (Subvención/contrato del NIH de EE. UU.)
- NCI-2026-05293 (Identificador de registro: CTRP (Clinical Trial Reporting Program))
- STU00226144
Información sobre medicamentos y dispositivos, documentos del estudio
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Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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