Superenhancer Inhibitor Minnelide in Advanced Refractory Adenosquamous Carcinoma of the Pancreas (ASCP)
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|ClinicalTrials.gov Identifier: NCT04896073|
Recruitment Status : Recruiting
First Posted : May 21, 2021
Last Update Posted : July 6, 2022
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.
|Condition or disease||Intervention/treatment||Phase|
|Adenosquamous Carcinoma of the Pancreas||Drug: Minnelide||Phase 2|
- 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 participants).
- 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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||55 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Trial of the Superenhancer Inhibitor Minnelide in Advanced Refractory Adenosquamous Carcinoma of the Pancreas (ASCP)|
|Actual Study Start Date :||March 7, 2022|
|Estimated Primary Completion Date :||September 1, 2026|
|Estimated Study Completion Date :||September 1, 2026|
Minnelide 2mg Days 1-21 of 28 day cycle (x12)
Administered orally (2 mg) once daily for 21 days of 28-day cycles for 12 cycles.
- disease control rate [ Time Frame: 16 weeks ]To determine the single agent antitumor activity (disease control rate = CR+PR+stable x16 weeks) of the anti-superenhancer agent Minnelide in participants with advanced, previously treated ASCP
- safety and tolerability of Minnelide [ Time Frame: duration of treatment ]AEs and SAEs of Minnelite
- progression free survival (PFS) [ Time Frame: start of treatment to 30 days after last treatment ]frequency and grade of AEs and SAEs
- Overall Survival (OS) [ Time Frame: through 1 year after last Minnelide treatment in the last subject who completes treatment ]The length of time from the start of treatment that patients diagnosed with the disease are still alive.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04896073
|Contact: NCI Medical Oncology Referral Office||(240) email@example.com|
|Contact: Christine C Alewine, M.D.||(240) firstname.lastname@example.org|
|United States, Maryland|
|National Institutes of Health Clinical Center||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office 888-624-1937|
|Principal Investigator:||Christine C Alewine, M.D.||National Cancer Institute (NCI)|