Cancer of PancreasEarly Phase 1NCT04123574
A Pilot Study of BXCL701 in Patients With Pancreatic Cancer
A Early Phase 1 study testing Talabostat Mesylate in people with Cancer of Pancreas and Cancer of the Pancreas, run by BioXcel Therapeutics Inc at 2 sites in United States.
Trial summary
A study to assess the biochemical and immunomodulatory effects of BXCL701 in pancreatic cancer. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Cancer of Pancreas, Cancer of the Pancreas, Neoplasms, Pancreatic
- What is being tested?
- Talabostat Mesylate
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 2 sitesUnited States
- Study dates
- 15 Oct 2019to 6 Dec 2021
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.)
- 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.)
- Has Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
- Is 18 to 75 years of age, inclusive
- Has adequate organ function within 28 days of treatment initiation
- 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.
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- Has oxygen saturation ≥ 92% on room air.
- Is able to take an oral medication.
- Has signed an Informed Consent Form (ICF) prior to the initiation of any study-specific procedures or treatment.
- Is willing and able to adhere to the study visit schedule and other protocol requirements.
- 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.
- 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 study.
Who cannot join
- A female who is pregnant or breast-feeding.
- Has other concurrent malignancies except for basal and squamous cell cancers of the skin and in-situ cervical cancer.
- 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.
- Has an upper gastrointestinal obstruction, abnormal physiological function, or malabsorption syndrome that may affect the absorption of study medication.
- 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).
- Has a premalignant hematologic disorder, eg, myelodysplastic syndrome.
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- 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.
- 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 or suspected/active SARS-CoV-2 (Covid-19) infection.
- Has a history of severe neurologic disorders, eg, cerebrovascular ischemia within the past year.
- Has a history of prior deep venous thrombosis or pulmonary embolism within the past year.
- Has serious medical, psychological, familial, sociological, or geographical conditions or circumstances potentially hampering compliance with the study protocol and follow-up.
- QT interval corrected for heart rate using Bazett's formula (QTcB) > 440 msec at Screening.
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
BioXcel Clinical Research Site
Boston, Massachusetts, 02215
BioXcel Clinical Research Site
New York, New York, 10021
About this study
Official title: A Pilot Proof of Mechanism Study of BXCL701, a Small Molecule Inhibitor of Dipeptidyl Peptidases (DPPs), in Patients With Pancreatic Cancer
A study to assess the biochemical and immunomodulatory effects of BXCL701 in pancreatic cancer.
Read the detailed description
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 immune cytokines.
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.
- Conditions
- Cancer of Pancreas, Cancer of the Pancreas, Neoplasms, Pancreatic, Pancreas Cancer, Pancreatic Cancer
- Being tested
- Talabostat Mesylate
- Keywords
- Pancreas, Cancer, Neoplasms, Pancreatic
- Medical subject headings
- Pancreatic Neoplasms, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Talabostat MesylateDrug
Also called BXCL701
BXCL701 tablets dosage strengths include 0.2mg and 0.05mg tablets for oral administration.
Patients are to self-administer the prescribed number of BXCL701 tablets for a total daily dose of 0.6 mg. BXCL701 should not be taken on an empty stomach.
Daily blood pressure monitoring will be performed during the dosing period. Administration of at least 1L of intravenous (IV) fluids is required on Day 1.
On days when pharmacokinetic (PK) assessments are being performed, BXCL701 should be administered at the study center and should be administered at (approximately) the same time of day on each treatment day.
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | BXCL701 will be administered for one week at a dose of 0.2 mg, twice daily (BID). If BXCL701 is well-tolerated after the first week of treatment, the dose will be increased to 0.3mg BID for a total daily dose of 0.6mg to all patients for the second week of treatment. Receives: Drug: Talabostat Mesylate |
What is being measured
The primary outcome is the main question the study is designed to answer.
To characterize the quantitative and qualitative effects of BXCL701 on relevant immune effector cytokines and various immunological effector cells that are consistent with its known mechanism of action.
To measure how BXCL701 effects the tumor by measuring the rate of tumor cell death or the reduction of tumor cell growth. This will be measured by scans and blood work.
Measured: Up to 37 days post treatment
Secondary outcomes (3)
Evaluate the tolerability of exposure to BXCL701: National Cancer Institute Common Terminology Criteria
assessed using the National Cancer Institute Common Terminology Criteria for Adverse Events
Measured: Up to 37 days post treatment
Evaluate the effect of exposure to BCXL701 on cancer cell death
Measure the rate of cancer cell death measured by histological staining methods of post-treatment biopsied tissue.
Measured: Up to 37days post treatment
Genomic analysis before and after treatment.
Genomic analysis is the identification, measurement or comparison of genomic features such as DNA sequence, structural variation, gene expression, or regulatory and functional element annotation at a genomic scale.
Measured: Up to 37 days post treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Oct 2019
- Study start15 Oct 2019 (estimated)
- Primary completion6 Dec 2021 (estimated)
- Study completion6 Dec 2021 (estimated)
Why it stopped: No longer relevant to field
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cancer of PancreasStudies of the same condition, open and under way.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioXcel Therapeutics Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- IQVIA Biotech
- Responsible party
- Sponsor
- Investigators
- BioXcel MM, MD, Study chair, BioXcel Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04123574
- Sponsor's protocol ID
- BXCL701-001
- Status last verified
- Aug 2026
- First submitted
- 30 Apr 2019
- Last updated
- 11 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Proof of Mechanism Study of BXCL701, a Small Molecule Inhibitor of Dipeptidyl Peptidases (DPPs), in Patients With Pancreatic Cancer
- ClinicalTrials.gov ID
- NCT04123574
- Acronym / sponsor ID
- BXCL701-001
- Conditions
- Cancer of Pancreas, Cancer of the Pancreas, Neoplasms, Pancreatic, Pancreas Cancer, Pancreatic Cancer
- Keywords
- Pancreas, Cancer, Neoplasms, Pancreatic
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: No longer relevant to field
- Enrolment
- 0 participants (actual)
- Lead sponsor
- BioXcel Therapeutics IncIndustry (pharma, biotech, devices)
- Collaborators
- IQVIA Biotech
- Study start
- 15 Oct 2019 (estimated)
- Primary completion
- 6 Dec 2021 (estimated)
- Study completion
- 6 Dec 2021 (estimated)
- First posted
- 11 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A study to assess the biochemical and immunomodulatory effects of BXCL701 in pancreatic cancer.
Detailed description
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 immune cytokines.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Drug: Talabostat MesylateBXCL701 will be administered for one week at a dose of 0.2 mg, twice daily (BID). If BXCL701 is well-tolerated after the first week of treatment, the dose will be increased to 0.3mg BID for a total daily dose of 0.6mg to all patients for the second week of treatment. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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.)
- 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.)
- Has Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
- Is 18 to 75 years of age, inclusive
- Has adequate organ function within 28 days of treatment initiation
Key exclusion criteria (13 listed)
- A female who is pregnant or breast-feeding.
- Has other concurrent malignancies except for basal and squamous cell cancers of the skin and in-situ cervical cancer.
- 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.
- Has an upper gastrointestinal obstruction, abnormal physiological function, or malabsorption syndrome that may affect the absorption of study medication.
- 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).
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 |
|---|---|---|
| Talabostat MesylateAlso: BXCL701BXCL701 tablets dosage strengths include 0.2mg and 0.05mg tablets for oral administration. Patients are to self-administer the prescribed number of BXCL701 tablets for a total daily dose of 0.6 mg. BXCL701 should not be taken on an empty stomach. Daily blood pressure monitoring will be performed during the dosing period. Administration of at least 1L of intravenous (IV) fluids is required on Day 1. On days when pharmacokinetic (PK) assessments are being performed, BXCL701 should be administered at the study center and should be administered at (approximately) the same time of day on each treatment day. | Drug | Single Arm |
Primary outcome
To characterize the quantitative and qualitative effects of BXCL701 on relevant immune effector cytokines and various immunological effector cells that are consistent with its known mechanism of action.
Time frame: Up to 37 days post treatment
To measure how BXCL701 effects the tumor by measuring the rate of tumor cell death or the reduction of tumor cell growth. This will be measured by scans and blood work.
3 secondary outcomes
Evaluate the tolerability of exposure to BXCL701: National Cancer Institute Common Terminology Criteria
Time frame: Up to 37 days post treatment
Evaluate the effect of exposure to BCXL701 on cancer cell death
Time frame: Up to 37days post treatment
Genomic analysis before and after treatment.
Time frame: Up to 37 days post treatment
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2026.
- Individual participant dataSharing planned: No
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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.
BioXcel MM, MD study chair
BioXcel Therapeutics
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Talabostat Mesylate in people with Cancer of Pancreas and Cancer of the Pancreas, run by BioXcel Therapeutics Inc at 2 sites 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 12 inclusion and 13 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: withdrawn, as of the sponsor's last update on 11 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 2 sites 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
To characterize the quantitative and qualitative effects of BXCL701 on relevant immune effector cytokines and various immunological effector cells that are consistent with its known mechanism of action. (time frame: Up to 37 days post treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 11 Aug 2026.