A Pilot Study of BXCL701 in Patients With Pancreatic Cancer

NCT04123574 | Withdrawn Early Phase 1 FDA Drug

• 1 other identifier: BXCL701-001
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 2

Brief Summary

A study 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

Keywords

Pancreas; Cancer; Neoplasms; Pancreatic

Outcome Measures

Primary Outcomes (1)

• 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.

  Up to 37 days post treatment

Secondary Outcomes (3)

• Evaluate the tolerability of exposure to BXCL701: National Cancer Institute Common Terminology Criteria

  Up to 37 days post treatment

• Evaluate the effect of exposure to BCXL701 on cancer cell death

  Up to 37days post treatment

• Genomic analysis before and after treatment.

  Up to 37 days post treatment

Study Arms (1)

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.

Drug: Talabostat Mesylate

Interventions

Talabostat Mesylate DRUG

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.

Also known as: BXCL701

Single Arm

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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

Sponsors & Collaborators

• BioXcel Therapeutics Inc: lead
• IQVIA Biotech: collaborator

Study Sites (2)

BioXcel Clinical Research Site

Boston, Massachusetts, 02215, United States

Location

BioXcel Clinical Research Site

New York, New York, 10021, United States

Location

MeSH Terms

Conditions

Pancreatic Neoplasms; Neoplasms

Interventions

PT-100 dipeptide

Condition Hierarchy (Ancestors)

Digestive System Neoplasms; Neoplasms by Site; Endocrine Gland Neoplasms; Digestive System Diseases; Pancreatic Diseases; Endocrine System Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 30, 2019
• First Posted: October 11, 2019
• Study Start: October 15, 2019
• Primary Completion: December 6, 2021
• Study Completion: December 6, 2021
• Last Updated: February 24, 2022

Record last verified: 2022-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)