Adenosquamous Carcinoma of the PancreasPhase 2NCT04896073
Superenhancer Inhibitor Minnelide in Advanced Refractory Adenosquamous Carcinoma of the Pancreas (ASCP)
A Phase 2 study testing Minnelide, ECG and CT/MRI in people with Adenosquamous Carcinoma of the Pancreas, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Background: Pancreatic cancer is one of the most lethal types of cancer. American Society for Clinical Pathology (ASCP) is a highly aggressive type of pancreatic cancer. It is very rare. Researchers want to see if a drug called Minnelide can be used to treat ASCP. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Adenosquamous Carcinoma of the Pancreas
- What is being tested?
- Minnelide, ECG and CT/MRI
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Mar 2022to 30 Mar 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Histological or cytological diagnosis of adenosquamous carcinoma of the pancreas (ASCP) or high suspicion for ASCP as confirmed by National Institutes of Health (NIH) Laboratory of Pathology. ASCP will be defined as >=30% malignant squamous component in background of typical pancreatic ductal adenocarcinoma (PDA). If malignant squamous component is identified in the sample, but the pathologist is unable to determine whether it is >= 30%, then the participant will be considered to have high suspicion for ASCP.
- Note: To meet this criterion, participant must be able to submit a suitable archival tumor specimen (primary or metastatic site) for review or currently have tumor in a location deemed low risk for core biopsy so that suitable tissue can be acquired for confirmation of diagnosis. Note that cytopathology specimens are not considered suitable for definitive diagnosis of ASCP but can be used to determine high suspicion for ASCP.
- Participants with metastatic, recurrent or locally advanced unresectable disease and progression or intolerance to at least 1 prior systemic treatment regimen in the advanced disease setting.
- Disease measurable by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria.
- Progressive disease as evidenced by increasing tumor size on radiologic assessment, increasing serum tumor marker (on last 2 measurements taken at least 1 week apart), increasing ascites, and/or worsening tumor-related symptoms such as weight loss, pain, gastrointestinal (GI) upset.
- Age >18 years.
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- Eastern Cooperative Oncology Group (ECOG) performance status <2 (Karnofsky >60%).
- Be willing and able to provide written informed consent for the trial.
- Participants must have adequate organ and marrow function as defined below:
- absolute neutrophil count (ANC) >= 1,500/microL
- platelets >= 100,000/microL
- hemoglobin >= 9.0 g/dL or >= 5.6 mmol/La*
- Creatinine <= 1.5 x ULN
- OR
- measured or calculated bcreatinine clearance (Glomerular filtration rate (GFR) can also be used in place of creatinine or CrCl) >= 45 mL/min for participant with creatinine levels >1.5 x institutional upper limit of normal (ULN)
- total bilirubin <= 1.5 x ULN OR direct bilirubin <= ULN for participants with total bilirubin levels >1.5 x ULN
- Aspartate aminotransferase (AST)/serum glutamic-oxaloacetic transaminase (SGOT) and alanine aminotransferase (ALT)/serum glutamate pyruvate transaminase (SGPT) <= 2.5 x ULN (<= 5 x ULN for participants with liver metastases)
- International normalized ratio (INR) OR
- prothrombin time (PT)
- activated partial thromboplastin time (aPTT):
- <= 1.5 x ULN unless participant is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants
- ALT (SGPT) = alanine aminotransferase (serum glutamic pyruvic transaminase); AST (SGOT) = aspartate aminotransferase (serum glutamic oxaloacetic transaminase); GFR = glomerular filtration rate; ULN=upper limit of normal.
- a Criteria must be met without erythropoietin dependency and without packed red blood cell (pRBC)
- transfusion within last 2 weeks.
- b Creatinine clearance (CrCl) should be calculated per institutional standard.
- Note: This table includes eligibility-defining laboratory value requirements for treatment; laboratory value requirements should be adapted according to local regulations and guidelines for the administration of specific chemotherapies.
- -The effects of Minnelide on the developing human fetus are unknown. For this reason, women of child-bearing potential (WOCBP) and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 6 months (women) and 3 months (men) after the last dose of trial treatment. Male participants must also refrain from donating sperm during this period. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
Who cannot join
- Has uncontrolled vomiting or medical condition which inhibits oral ingestion or digestion because the study treatment is administered orally.
- Pregnant and/or women who are breast feeding are excluded from this study because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with Minnelide.
- Is currently participating and receiving trial therapy or has participated in a trial of an investigational agent/therapy or used an investigational device within 3 weeks of the first planned treatment on this study.
- Has had prior chemotherapy, targeted small molecule therapy, or radiation therapy within 2 weeks prior to Cycle 1/Day 1
- Requires use of ondansetron or another prohibited medication. Note that other 5-hydroxytryptamine 3 (5-HT3) inhibitors are NOT prohibited.
- Has received major surgery within the last 4 weeks, minor endoscopic procedure such as biliary stenting within the last 2 weeks, or percutaneous procedure such as hepatic biopsy or celiac plexus block within 24 hours of planned treatment start date. Note: participant must have recovered adequately from the toxicity and/or complications from the intervention prior to starting therapy.
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- Has a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer.
- Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis.
- Participants with previously treated brain metastases may participate if:
- follow-up brain imaging after central nervous system (CNS)-directed therapy shows no evidence of progression at >= 4 weeks since treatment, AND b) participant has stability of baseline neurologic symptoms without receiving immunosuppressive-doses of systemic corticosteroid (physiologic replacement doses are permitted) x7 days or increases in other supportive medications that treat neurologic symptoms such as antiepileptics x14 days. Participants with carcinomatous meningitis are excluded regardless of clinical stability.
- Has an active infection requiring systemic therapy.
- Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the participant s participation for the full duration of the trial, or is not in the best interest of the participant to participate, in the opinion of the treating investigator.
- Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
- Has known uncontrolled or poorly controlled human immunodeficiency virus (HIV) infection. HIV is considered uncontrolled or poorly controlled if an HIV-infected individual is not taking highly active anti-retroviral therapy or has a detectable viral load within the previous 6 months.
- Has active hepatitis B virus (HBV) or hepatitis C virus (HCV) or is currently under treatment for HBV or HCV. Active HBV or HCV does not include previously cleared HBV or HCV or successfully cured HBV or HCV through treatment
- Has received a live vaccine within 30 days of planned start of trial therapy.
- Note: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however intranasal influenza vaccines (e.g., Flu-Mist (Registered Trademark)) are live attenuated vaccines and are not allowed.
- -History of allergic reactions attributed to compounds of similar chemical or biologic composition to Minnelide
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: A Phase II Trial of the Superenhancer Inhibitor Minnelide in Advanced Refractory Adenosquamous Carcinoma of the Pancreas (ASCP)
Background:
Pancreatic cancer is one of the most lethal types of cancer. American Society for Clinical Pathology (ASCP) is a highly aggressive type of pancreatic cancer. It is very rare. Researchers want to see if a drug called Minnelide can be used to treat ASCP.
Read more
Objective:
To see if Minnelide is an effective treatment for ASCP.
Eligibility:
Adults ages 18 and older with ASCP whose cancer did not respond to previous treatments.
Design:
Participants will be screened with:
Medical history
Physical exam
Blood and urine samples
Evaluation of ability to do daily activities
Electrocardiogram to test heart function
Body and/or brain scans. For these, participants will lie in a machine that takes pictures of the body. They may have a contrast agent injected into a vein.
Tumor sample. If one is not available, participants will have a tumor biopsy. The biopsy will be taken with a small needle put through the skin into the tumor.
Treatment will be given in 28-day cycles, for up to 12 cycles. There is a 7-day resting period between cycles. Participants will take Minnelide by mouth every day for 21 days of each cycle. They will keep a medicine diary.
Participants will have at least 1 study visit every cycle. They will review their medicine diary. They will repeat some screening tests.
Participants may have optional tumor biopsies. Some participants may need to take birth control during the study and for up to 6 months after treatment.
Participants will have an end-of-treatment visit 4 weeks after they stop taking the study drug. They will repeat some screening tests.
Read the detailed description
Background:
- Adenosquamous carcinoma of the pancreas (ASCP) is a highly aggressive variant of pancreatic ductal adenocarcinoma (PDA), the most common type of pancreas cancer.
- ASCP is estimated to account for 0.5-4% of the 55,000 people who are diagnosed with pancreatic cancer in the United States (U.S.) each year, making it a very rare tumor type.
- No prospective clinical trials specific to ASCP have ever been performed.
- Preclinical data in ASCP models indicate that an activated superenhancer network drives epigenetic changes which cause the prognostically unfavorable squamous differentiation.
- Genomic analysis of ASCP tumors identifies frequent amplification of MYC.
- Minnelide is a small molecule anti-superenhancer drug that inhibits MYC.
- The recommended dose of Minnelide has previously been established through clinical testing for other indications.
Primary Objective:
-To determine the single agent antitumor activity (disease control rate) of the anti-superenhancer agent Minnelide in participants with advanced, previously treated ASCP
Eligibility:
- Age >= 18 years
- Histologically confirmed ASCP or high suspicion for ASCP based on histologic analysis for squamous markers
- Participants with metastatic or locally advanced unresectable disease and progression on at least 1 prior treatment regimen
Design:
- This is a phase II single cohort clinical trial with one arm.
- The number of evaluable participants needed for the primary endpoint is 25; maximum accrual set at 55 participants (accounting for screen failures and inevaluable participants).
- initial participants will receive Minnelide at 2 mg/day by mouth (PO) on Days 1-21 of a 28-day cycle.
- Later participants will receive a higher dose of 2.5 mg/day PO on the same schedule.
- Treatment will be continued for up to 12 cycles (1 year) in the absence of disease progression or unacceptable toxicity.
- Treatment response will be assessed by imaging every 2 cycles (8 weeks).
- Optional tumor biopsies will be requested mid-cycle 1 and at time of progression.
- A disease control rate of >= 40% in this highly refractory population would constitute a positive study. Up to 12 participants will be treated initially. If 3 of the 12 participants have a response, then up to 13 additional participants will be entered to determine the true response rate.
- Conditions
- Adenosquamous Carcinoma of the Pancreas
- Being tested
- Minnelide, ECG, CT/MRI, Tumor Biopsy
- Keywords
- Pancreatic Ductal Adenocarcinoma, epigenetic changes, MYC, triptolide, HSP70
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MinnelideDrug
Also called 14-O-phosphonooxymethyltriptolide-disodium-salt and Minnelide-001
Administered orally (2 mg) once daily for 21 days of 28-day cycles for 12 cycles.
ECGDiagnostic test
Also called Electrocardiogram
Screening and end of treatment visit.
CT/MRIDiagnostic test
Also called Computed tomography/Magnetic resonance imaging
Screening. Subsequent cycles Day 1 (≤3days) and Day 15 (±2 days) (every other cycle). End of treatment visit.
Tumor BiopsyProcedure or surgery
Also called Tumor Bx
Optional. Baseline Cycle 1, Day 1 and Cycle 1, Day 15. End of treatment visit or progressive disease.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1, Cohort 1, Minnelide in Advanced Adenosquamous Carcinoma of the Pancreas (ASCP) | Experimental | Minnelide 2mg Days 1-21 of 28-day cycle (x12) Receives: Drug: Minnelide; Diagnostic Test: ECG; Diagnostic Test: CT/MRI; Procedure: Tumor Biopsy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Evaluable Participants Who Experience Clinical Benefit (Disease Control Rate = Complete Response (CR)+Partial Response (PR)+Stable Disease x16 Weeks) Reported Along With a 95% Confidence Interval
To determine the single agent antitumor activity (disease control rate = CR + PR + stable disease x16 weeks) of the anti-superenhancer agent Minnelide in participants with advanced, previously treated adenosquamous carcinoma of the pancreas (ASCP) using Response Evaluation Criteria in Solid Tumors (RECIST) criteria. Complete Response is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in short axis to <10 mm. Partial Response is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum of diameters. Stable Disease is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD), taking as reference the smallest sum of diameters while on study. Progressive Disease is least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study.
Measured: 16 weeks
Secondary outcomes (5)
Number of Participants Who Experience a Non-serious Grade 2 or Higher Toxicity, by Grade and Type of Toxicity Assessed by the Common Terminology Criteria for Adverse Events (CTCAE)
Non-serious adverse events of Minnelide were assessed by the Common Terminology Criteria for Adverse Events (CTCAE). A non-serious adverse event is any untoward medical occurrence. Grade 2 is moderate. Grade 3 is severe. Grade 4 is life-threatening. Grade 5 is death related to adverse events.
Measured: Non-serious adverse events related to Minnelide that occur from start of treatment to 30 days after last treatment
Number of Participants Who Experience a Serious Grade 2 or Higher Toxicity, by Grade and Type of Toxicity Assessed by the Common Terminology Criteria for Adverse Events (CTCAE)
Serious adverse events of Minnelite were assessed by the Common Terminology Criteria for Adverse Events (CTCAE). A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. Grade 2 is moderate. Grade 3 is severe. Grade 4 is life-threatening. Grade 5 is death related to adverse events.
Measured: Serious adverse events related to Minnelide that occur from start of treatment to 30 days after last treatment
Progression Free Survival (PFS)
PFS is defined as the duration of time from start of treatment to time of progression or death, whichever occurs first. PFS will be determined using the Kaplan-Meier method and reported along with a 95% confidence interval for the median. Progression was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) criteria. Progressive Disease is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study. The appearance of one or more new lesions is also considered progressions.
Measured: start of treatment to time of progression or death, a median of 1.76 months
Overall Survival (OS)
OS is defined as the length of time from the start of treatment until death. OS will be determined by using the Kaplan-Meier method and reported along with a 95% confidence interval for the median.
Measured: time from the start of treatment until death, a median of 4.91 months
Objective Response Rate (ORR)
Objective response rate (ORR) will be calculated as the percentage of participants with Complete Response (CR) or Partial Response (PR) as defined by Response Evaluation Criteria in Solid Tumors (RECIST)1.1 criteria. Complete Response is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in short axis to <10 mm. Partial Response is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum of diameters.
Measured: From start of treatment until radiological progression response or off study; the median is approximately 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2021
- Study start7 Mar 2022
- Primary completion30 Mar 2025
- Study completion30 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Adenosquamous Carcinoma of the PancreasStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Principal investigator, Chen Zhao, National Cancer Institute (NCI)
- Investigators
- Chen Zhao, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04896073
- Sponsor's protocol ID
- 10000254
- Other IDs
- 000254-C
- Status last verified
- Jul 2026
- First submitted
- 20 May 2021
- Last updated
- 14 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All individual participant data (IPD) recorded in the medical record will be shared with intramural investigators upon request. In addition, all large-scale genomic sequencing data will be shared with subscribers to the database of Genotypes and Phenotypes (dbGaP).
Publications
Skorupan N, Ahmad MI, Steinberg SM, Trepel JB, Cridebring D, Han H, Von Hoff DD, Alewine C. A phase II trial of the super-enhancer inhibitor Minnelide in advanced refractory adenosquamous carcinoma of the pancreas. Future Oncol. 2022 Jun;18(20):2475-2481. doi: 10.2217/fon-2021-1609. Epub 2022 May 10.
PubMed 35535581
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of the Superenhancer Inhibitor Minnelide in Advanced Refractory Adenosquamous Carcinoma of the Pancreas (ASCP)
- ClinicalTrials.gov ID
- NCT04896073
- Acronym / sponsor ID
- 10000254
- Conditions
- Adenosquamous Carcinoma of the Pancreas
- Keywords
- Pancreatic Ductal Adenocarcinoma, epigenetic changes, MYC, triptolide, HSP70
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 7 Mar 2022 (actual)
- Primary completion
- 30 Mar 2025 (actual)
- Study completion
- 30 Mar 2025 (actual)
- First posted
- 21 May 2021 (actual)
- Results
- Posted 7 Oct 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
Pancreatic cancer is one of the most lethal types of cancer. American Society for Clinical Pathology (ASCP) is a highly aggressive type of pancreatic cancer. It is very rare. Researchers want to see if a drug called Minnelide can be used to treat ASCP.
Objective:
To see if Minnelide is an effective treatment for ASCP.
Eligibility:
Adults ages 18 and older with ASCP whose cancer did not respond to previous treatments.
Design:
Participants will be screened with:
Medical history
Physical exam
Blood and urine samples
Evaluation of ability to do daily activities
Electrocardiogram to test heart function
Body and/or brain scans. For these, participants will lie in a machine that takes pictures of the body. They may have a contrast agent injected into a vein.
Tumor sample. If one is not available, participants will have a tumor biopsy. The biopsy will be taken with a small needle put through the skin into the tumor.
Treatment will be given in 28-day cycles, for up to 12 cycles. There is a 7-day resting period between cycles. Participants will take Minnelide by mouth every day for 21 days of each cycle. They will keep a medicine diary.
Participants will have at least 1 study visit every cycle. They will review their medicine diary. They will repeat some screening tests.
Participants may have optional tumor biopsies. Some participants may need to take birth control during the study and for up to 6 months after treatment.
Participants will have an end-of-treatment visit 4 weeks after they stop taking the study drug. They will repeat some screening tests.
Detailed description
Background:
- Adenosquamous carcinoma of the pancreas (ASCP) is a highly aggressive variant of pancreatic ductal adenocarcinoma (PDA), the most common type of pancreas cancer.
- ASCP is estimated to account for 0.5-4% of the 55,000 people who are diagnosed with pancreatic cancer in the United States (U.S.) each year, making it a very rare tumor type.
- No prospective clinical trials specific to ASCP have ever been performed.
- Preclinical data in ASCP models indicate that an activated superenhancer network drives epigenetic changes which cause the prognostically unfavorable squamous differentiation.
- Genomic analysis of ASCP tumors identifies frequent amplification of MYC.
- Minnelide is a small molecule anti-superenhancer drug that inhibits MYC.
- The recommended dose of Minnelide has previously been established through clinical testing for other indications.
Primary Objective:
-To determine the single agent antitumor activity (disease control rate) of the anti-superenhancer agent Minnelide in participants with advanced, previously treated ASCP
Eligibility:
- Age >= 18 years
- Histologically confirmed ASCP or high suspicion for ASCP based on histologic analysis for squamous markers
- Participants with metastatic or locally advanced unresectable disease and progression on at least 1 prior treatment regimen
Design:
- This is a phase II single cohort clinical trial with one arm.
- The number of evaluable participants needed for the primary endpoint is 25; maximum accrual set at 55 participants (accounting for screen failures and inevaluable participants).
- initial participants will receive Minnelide at 2 mg/day by mouth (PO) on Days 1-21 of a 28-day cycle.
- Later participants will receive a higher dose of 2.5 mg/day PO on the same schedule.
- Treatment will be continued for up to 12 cycles (1 year) in the absence of disease progression or unacceptable toxicity.
- Treatment response will be assessed by imaging every 2 cycles (8 weeks).
- Optional tumor biopsies will be requested mid-cycle 1 and at time of progression.
- A disease control rate of >= 40% in this highly refractory population would constitute a positive study. Up to 12 participants will be treated initially. If 3 of the 12 participants have a response, then up to 13 additional participants will be entered to determine the true response rate.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm 1, Cohort 1, Minnelide in Advanced Adenosquamous Carcinoma of the Pancreas (ASCP) | Experimental | Drug: Minnelide, Diagnostic Test: ECG, Diagnostic Test: CT/MRI, Procedure: Tumor BiopsyMinnelide 2mg Days 1-21 of 28-day cycle (x12) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (27 listed)
- Histological or cytological diagnosis of adenosquamous carcinoma of the pancreas (ASCP) or high suspicion for ASCP as confirmed by National Institutes of Health (NIH) Laboratory of Pathology. ASCP will be defined as >=30% malignant squamous component in background of typical pancreatic ductal adenocarcinoma (PDA). If malignant squamous component is identified in the sample, but the pathologist is unable to determine whether it is >= 30%, then the participant will be considered to have high suspicion for ASCP.
- Note: To meet this criterion, participant must be able to submit a suitable archival tumor specimen (primary or metastatic site) for review or currently have tumor in a location deemed low risk for core biopsy so that suitable tissue can be acquired for confirmation of diagnosis. Note that cytopathology specimens are not considered suitable for definitive diagnosis of ASCP but can be used to determine high suspicion for ASCP.
- Participants with metastatic, recurrent or locally advanced unresectable disease and progression or intolerance to at least 1 prior systemic treatment regimen in the advanced disease setting.
- Disease measurable by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria.
- Progressive disease as evidenced by increasing tumor size on radiologic assessment, increasing serum tumor marker (on last 2 measurements taken at least 1 week apart), increasing ascites, and/or worsening tumor-related symptoms such as weight loss, pain, gastrointestinal (GI) upset.
Key exclusion criteria (18 listed)
- Has uncontrolled vomiting or medical condition which inhibits oral ingestion or digestion because the study treatment is administered orally.
- Pregnant and/or women who are breast feeding are excluded from this study because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with Minnelide.
- Is currently participating and receiving trial therapy or has participated in a trial of an investigational agent/therapy or used an investigational device within 3 weeks of the first planned treatment on this study.
- Has had prior chemotherapy, targeted small molecule therapy, or radiation therapy within 2 weeks prior to Cycle 1/Day 1
- Requires use of ondansetron or another prohibited medication. Note that other 5-hydroxytryptamine 3 (5-HT3) inhibitors are NOT prohibited.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MinnelideAlso: 14-O-phosphonooxymethyltriptolide-disodium-salt and Minnelide-001Administered orally (2 mg) once daily for 21 days of 28-day cycles for 12 cycles. | Drug | Arm 1, Cohort 1, Minnelide in Advanced Adenosquamous Carcinoma of the Pancreas (ASCP) |
| ECGAlso: ElectrocardiogramScreening and end of treatment visit. | Diagnostic test | Arm 1, Cohort 1, Minnelide in Advanced Adenosquamous Carcinoma of the Pancreas (ASCP) |
| CT/MRIAlso: Computed tomography/Magnetic resonance imagingScreening. Subsequent cycles Day 1 (≤3days) and Day 15 (±2 days) (every other cycle). End of treatment visit. | Diagnostic test | Arm 1, Cohort 1, Minnelide in Advanced Adenosquamous Carcinoma of the Pancreas (ASCP) |
| Tumor BiopsyAlso: Tumor BxOptional. Baseline Cycle 1, Day 1 and Cycle 1, Day 15. End of treatment visit or progressive disease. | Procedure or surgery | Arm 1, Cohort 1, Minnelide in Advanced Adenosquamous Carcinoma of the Pancreas (ASCP) |
Primary outcome
Proportion of Evaluable Participants Who Experience Clinical Benefit (Disease Control Rate = Complete Response (CR)+Partial Response (PR)+Stable Disease x16 Weeks) Reported Along With a 95% Confidence Interval
Time frame: 16 weeks
To determine the single agent antitumor activity (disease control rate = CR + PR + stable disease x16 weeks) of the anti-superenhancer agent Minnelide in participants with advanced, previously treated adenosquamous carcinoma of the pancreas (ASCP) using Response Evaluation Criteria in Solid Tumors (RECIST) criteria. Complete Response is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in short axis to <10 mm. Partial Response is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum of diameters. Stable Disease is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD), taking as reference the smallest sum of diameters while on study. Progressive Disease is least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study.
5 secondary outcomes
Number of Participants Who Experience a Non-serious Grade 2 or Higher Toxicity, by Grade and Type of Toxicity Assessed by the Common Terminology Criteria for Adverse Events (CTCAE)
Time frame: Non-serious adverse events related to Minnelide that occur from start of treatment to 30 days after last treatment
Number of Participants Who Experience a Serious Grade 2 or Higher Toxicity, by Grade and Type of Toxicity Assessed by the Common Terminology Criteria for Adverse Events (CTCAE)
Time frame: Serious adverse events related to Minnelide that occur from start of treatment to 30 days after last treatment
Progression Free Survival (PFS)
Time frame: start of treatment to time of progression or death, a median of 1.76 months
Overall Survival (OS)
Time frame: time from the start of treatment until death, a median of 4.91 months
Objective Response Rate (ORR)
Time frame: From start of treatment until radiological progression response or off study; the median is approximately 6 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 35535581Skorupan N, Ahmad MI, Steinberg SM, Trepel JB, Cridebring D, Han H, Von Hoff DD, Alewine C. A phase II trial of the super-enhancer inhibitor Minnelide in advanced refractory adenosquamous carcinoma of the pancreas. Future Oncol. 2022 Jun;18(20):2475-2481. doi: 10.2217/fon-2021-1609. Epub 2022 May 10.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. All individual participant data (IPD) recorded in the medical record will be shared with intramural investigators upon request. In addition, all large-scale genomic sequencing data will be shared with subscribers to the database of Genotypes and Phenotypes (dbGaP).
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Pancreatic Cancer, Pancreatic Cancer Metastatic
RecruitingPhase 3
Study of Zoldonrasib (RMC-9805) Plus Daraxonrasib (RMC-6236) Versus Gemcitabine and Nab-Paclitaxel as First-Line Treatment in Metastatic KRAS G12D-Mutated Pancreatic Adenocarcinoma
Pancreatic Cancer, Pancreatic Cancer Metastatic
RecruitingPhase 2
APOLLO: A Randomized Phase II Double-Blind Study of Olaparib Versus Placebo Following Curative Intent Therapy in Patients With Resected Pancreatic Cancer and a Pathogenic BRCA1, BRCA2 or PALB2 Mutation
Pancreatic Acinar Cell Carcinoma, Pancreatic Adenosquamous Carcinoma
Active, not recruitingPhase 2/3
The PLATINUM Trial: Optimizing Chemotherapy for the Second-Line Treatment of Metastatic BRCA1/2 or PALB2-Associated Metastatic Pancreatic Cancer
Pancreatic Acinar Cell Carcinoma, Pancreatic Adenocarcinoma
RecruitingPhase 3
Study of Daraxonrasib and Daraxonrasib + GnP as First-line Treatment in Patients With Metastatic Pancreatic Adenocarcinoma
Pancreatic Cancer, Pancreatic Cancer Metastatic
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Chen Zhao, M.D. principal investigator
National Cancer Institute (NCI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Minnelide, ECG and CT/MRI in people with Adenosquamous Carcinoma of the Pancreas, run by National Cancer Institute (NCI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 27 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Oct 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of Evaluable Participants Who Experience Clinical Benefit (Disease Control Rate = Complete Response (CR)+Partial Response (PR)+Stable Disease x16 Weeks) Reported Along With a 95% Confidence Interval (time frame: 16 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04896073, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Aug 2026.