Pancreatic cancer is one of the most lethal types of cancer. 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.
To see if Minnelide is an effective treatment for ASCP.
Adults ages 18 and older with ASCP whose cancer did not respond to previous treatments.
Participants will be screened with:
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.
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 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.
-To determine the single agent antitumor activity (disease control rate) of the
anti-superenhancer agent Minnelide in participants with advanced, previously treated ASCP
Age >= 18 years
Histologically confirmed ASCP
Participants with metastatic or locally advanced unresectable disease and progression on
at least 1 prior treatment regimen
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
All participants will receive Minnelide at 2 mg/day PO on Days 1-21 of a 28 day cycle.
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 treated be 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.
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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