A study to assess the biochemical and immunomodulatory effects of BXCL701 in pancreatic
This is a Phase 0 or "window of opportunity" study where paired specimen analysis, taken
before and after drug exposure, will permit the evaluation of target modulation and
assessment of immune effector cell infiltration into the tumor and the generation of relevant
In this study, BXCL701 will be administered at a dose of 0.3 mg, twice daily for a total
daily dose of 0.6mg (the previously defined maximum tolerated dose [MTD] of the drug), to all
patients for a short period of 14 days. This study is designed to assess the biochemical and
immunomodulatory effects of BXCL701 in pancreatic cancer.
1. Has untreated (eg, no prior investigational therapies, chemotherapy, or radiation
therapy), locally advanced or metastatic adenocarcinoma of the head, neck, uncinate
process, or tail of the pancreas with a local or metastatic lesion that is amenable to
biopsy before and after treatment. (Whenever possible, the before and after treatment
biopsies should be from the same lesion.)
2. Is able and willing to undergo tumor biopsy before and after treatment. (A
pretreatment biopsy may not be needed if tissue is available from a biopsy conducted
within 28 days prior to screening that is adequate for the study assessments.)
3. Has Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
4. Is 18 to 75 years of age, inclusive
5. Has adequate organ function within 28 days of treatment initiation
6. For participants with exposure to prior agents associated with decreased left
ventricular ejection fraction (LVEF) (e.g. anthracyclines), or if clinically
warranted, a documented LVEF > 45% using a standard echocardiogram (ECHO) or
multigated acquisition (MUGA) scan test at Screening or within 60 days prior to Cycle
1 Day 1. ECHO or MUGA testing for other participants without relevant medical history
or clinical symptoms can be performed if feasible.
7. Has oxygen saturation ≥ 92% on room air.
8. Is able to take an oral medication.
9. Has signed an Informed Consent Form (ICF) prior to the initiation of any
study-specific procedures or treatment.
10. Is willing and able to adhere to the study visit schedule and other protocol
11. Women of childbearing potential (WOCBP) must have a negative pregnancy test at
baseline. A woman must be menopausal for at least 12 months before she is considered
not to be of reproductive potential.
12. Male and female patients of reproductive potential must agree to use an effective
contraceptive method during participation in this study and for 6 months following the
1. A female who is pregnant or breast-feeding.
2. Has other concurrent malignancies except for basal and squamous cell cancers of the
skin and in-situ cervical cancer.
3. Has uncontrolled epilepsy, central nervous system diseases, or a history of mental
disorder that is severe enough to hinder the ability of the patient to provide
informed consent or that may influence the patient's compliance with the protocol in
the judgments of the investigator.
4. Has an upper gastrointestinal obstruction, abnormal physiological function, or
malabsorption syndrome that may affect the absorption of study medication.
5. Has required chronic corticosteroids, defined as > 10 mg/day of prednisone or
equivalent, or immunosuppressive therapy within the past 3 months. Patient requires
treatment with DPP4 inhibitors (e.g. gliptins).
6. Has a premalignant hematologic disorder, eg, myelodysplastic syndrome.
7. Has a severe organ dysfunction or disease that might prevent completion of the
treatment regimen, eg, cardiopulmonary diseases (New York Heart Association [NYHA] ≥
Class III, arrhythmia Lown III/IV, global respiratory insufficiency); ascites; acute
pancreatitis; bleeding diathesis, coagulopathy, or need for full dose anticoagulation.
8. Has a chronic infectious disease, especially immune deficiency syndromes, eg, human
immunodeficiency virus (HIV) infection, active tuberculosis within 12 months prior to
potential study participation.
9. Has a history of severe neurologic disorders, eg, cerebrovascular ischemia within the
10. Has a history of prior deep venous thrombosis or pulmonary embolism within the past
11. Has serious medical, psychological, familial, sociological, or geographical conditions
or circumstances potentially hampering compliance with the study protocol and
12. QT interval corrected for heart rate using Bazett's formula (QTcB) > 440 msec at
13. Patients with history of symptomatic orthostatic hypotension within 3 months prior to
enrollment. Orthostatic hypotension is defined as a drop in systolic blood pressure
(BP) of ≥ 20 mmHg or diastolic BP of ≥ 10 mmHg with assumption of an upright posture