NXP800 for the Treatment of Patients With Advanced or Metastatic Cholangiocarcinoma

NCT06420349 | Terminated Phase 1 DMC FDA Drug

• 3 other identifiers: MC230406NCI-2024-0361323-012778
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 2

Brief Summary

This phase I trial tests the safety, best dose, and effectiveness of NXP800 in treating patients with cholangiocarcinoma that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) or that has spread from where it first started (primary site) to other places in the body (metastatic). NXP800 inhibits a pathway called the heat shock factor 1 (HSF1) pathway. The inhibition of this pathway inhibits proliferation, migration, survival, and metastasis in susceptible tumor cells. Overexpressed, amplified and/or overactivated in many cancer cells, HSF1 activates a set of genes that play a key role in tumor initiation, progression and metastasis. Inhibiting this pathway may in turn inhibit tumor initiation, progression, and/or metastasis. Giving NXP800 may be safe, tolerable and/or effective in treating patients with advanced or metastatic cholangiocarcinoma.

Conditions

Stage III Hilar Cholangiocarcinoma AJCC v8; Advanced Cholangiocarcinoma; Metastatic Cholangiocarcinoma; Refractory Cholangiocarcinoma; Stage III Intrahepatic Cholangiocarcinoma AJCC v8; Stage IV Hilar Cholangiocarcinoma AJCC v8; Stage IV Intrahepatic Cholangiocarcinoma AJCC v8

Outcome Measures

Primary Outcomes (2)

• Maximum tolerated dose (MTD)

  MTD is defined as the dose level below the lowest dose that induces dose-limiting toxicity in at least one-third of patients.

  Up to 12 months

• Recommended phase 2 dose

  Recommended phase 2 dose is based on MTD (Outcome 1).

  Up to 12 months

Secondary Outcomes (4)

• Incidence of adverse events

  Up to 30 days after the administration of the last dose of study drug

• Best response

  Up to 3 years

• Overall survival (OS)

  Up to 3 years

• Progression-free survival (PFS)

  Up to 3 years

Study Arms (1)

Treatment (NXP800)

EXPERIMENTAL

Patients receive NXP800 according to assigned treatment schedule. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo CT, MRI, and/or PET at baseline and on study. Patients may optionally undergo ultrasound-guided tumor biopsy and/or collection of blood samples on study and during follow up.

Procedure: Biospecimen Collection; Procedure: Computed Tomography; Biological: Heat Shock Factor 1 Pathway Inhibitor NXP800; Procedure: Magnetic Resonance Imaging; Procedure: Positron Emission Tomography; Procedure: Ultrasound-Guided Biopsy

Interventions

Heat Shock Factor 1 Pathway Inhibitor NXP800 BIOLOGICAL

Given PO

Also known as: CCT 361814, CCT-361814, CCT361814, HSF1 Pathway Inhibitor NXP800, NXP-800, NXP800, VK2019

Treatment (NXP800)

Magnetic Resonance Imaging PROCEDURE

Undergo MRI

Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI

Treatment (NXP800)

Biospecimen Collection PROCEDURE

Undergo collection of blood samples

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection

Treatment (NXP800)

Computed Tomography PROCEDURE

Undergo CT

Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, tomography

Treatment (NXP800)

Positron Emission Tomography PROCEDURE

Undergo PET

Also known as: Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron emission tomography (procedure), Positron Emission Tomography Scan, Positron-Emission Tomography, PT

Treatment (NXP800)

Ultrasound-Guided Biopsy PROCEDURE

Undergo ultrasound-guided tumor biopsy

Also known as: Ultrasound Biopsy, Ultrasound Guided Biopsy

Treatment (NXP800)

Eligibility Criteria

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

You may qualify if:

• Age ≥ 18 years
• Histologically/cytologically confirmed biliary tract cancer
• Advanced or metastatic disease that is refractory to gemcitabine or fluoropyrimidine based therapy, or if there is intolerance to these regimens
• Measurable disease by RECIST 1.1
• Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0 or 1
• Anticipated life expectancy of \> 12 weeks
• Hemoglobin ≥ 9.0 g/dL (obtained ≤ 14 days prior to registration)
• Absolute neutrophil count (ANC) ≥ 1500/mm\^3 (obtained ≤ 14 days prior to registration)
• Platelet count ≥ 100,000/mm\^3 (obtained ≤ 14 days prior to registration)
• Aspartate aminotransferase (AST) ≤ 5 x upper limit of normal (ULN) (obtained ≤ 14 days prior to registration)
• Alanine aminotransferase (ALT) ≤ 5 x ULN (obtained ≤ 14 days prior to registration)
• Total bilirubin ≤ 1.5 x ULN (obtained ≤ 14 days prior to registration)
• Serum creatinine ≤ 1.5 x ULN (obtained ≤ 14 days prior to registration)
• Provide written informed consent
• Negative pregnancy test done ≤ 7 days prior to registration, for persons of childbearing potential only

You may not qualify if:

• Any of the following because this study involves an investigational agent whose genotoxic, mutagenic, and teratogenic effects on the developing fetus and newborn are unknown:
• Pregnant persons.
• Nursing persons.
• Persons of childbearing potential who are unwilling to employ adequate contraception
• Systemic anti-neoplastic therapy or radiation therapy ≤ 14 days prior to registration
• Major surgical procedure ≤ 28 days prior to registration
• Ongoing therapy related events \> grade 2
• Presence of another primary malignancy not in remission
• New York Heart Classification 3 or greater heart failure
• QT/corrected QT (QTc) interval \> 470 ms using Fredericia's QT correction formula
• Uncontrolled brain metastatic disease
• Uncontrolled infection
• Any other comorbidities within the opinion of the investigator interfere with the investigation of the protocol
• Usage of drugs that strongly inhibit or induce CYP3A4 ≤ 7 days prior to registration and for the duration of NXP800 dosing. Drugs that are low, medium, or other inhibitors of CYP3A4 are not prohibited and should be used with caution. Drugs that inhibit BCRP are not prohibited but should be used with caution, since NXP800 was found to be a BCRP substrate
• Usage of seville oranges, grapefruit or grapefruit juice or products ≤ 7 days prior to registration and for the duration of NXP800 dosing

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (2)

Mayo Clinic in Arizona

Scottsdale, Arizona, 85259, United States

Location

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Publications (1)

• Kuipers H, Larson JJ, Conboy CB, Ahn DH, Bekaii-Saab T, Wu C, Sonbol MB, Ilyas SI, Gores GJ, Smoot RL, Borad MJ. A Phase 1 study of the novel GCN2 kinase activator NXP800 in patients with advanced cholangiocarcinoma. Future Oncol. 2025 Sep;21(21):2723-2730. doi: 10.1080/14796694.2025.2539611. Epub 2025 Jul 28.

  PMID: 40717483 DERIVED

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Cholangiocarcinoma; Klatskin Tumor

Interventions

Specimen Handling; Magnetic Resonance Spectroscopy

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Spectrum Analysis; Chemistry Techniques, Analytical

Study Officials

• Mitesh J. Borad, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: May 8, 2024
• First Posted: May 20, 2024
• Study Start: May 31, 2024
• Primary Completion: August 12, 2025
• Study Completion: September 9, 2025
• Last Updated: December 22, 2025

Record last verified: 2025-12

Locations

US (2)