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Advanced CholangiocarcinomaPhase 1NCT06420349

NXP800 for the Treatment of Patients With Advanced or Metastatic Cholangiocarcinoma

A Phase 1 study testing Biospecimen Collection, Computed Tomography and Heat Shock Factor 1 Pathway Inhibitor NXP800 in people with Advanced Cholangiocarcinoma and Metastatic Cholangiocarcinoma, run by Mayo Clinic at 2 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Biospecimen Collection, Computed Tomography and Heat Shock Factor 1 Pathway Inhibitor NXP800
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
7 participants
Where
2 sitesUnited States
Study dates
31 May 2024to 9 Sep 2025
Phase
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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
  • NOTE: If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
  • Willing to use a highly effective method of contraception from the first dose of study medication through 180 days after the last dose of study medication, for persons of childbearing potential or persons able to father a child only
  • Willing to return to enrolling institution for follow-up (during the active monitoring phase of the study)

Who cannot join

  • 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
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  • 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
  • Unwillingness to follow study related procedures
  • Inability to provide informed consent

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • Mayo Clinic in Rochester

    Rochester, Minnesota, 55905

  • Mayo Clinic in Arizona

    Scottsdale, Arizona, 85259

Other trials in United States

About this study

Official title: Phase 1b Study of the Novel GCN2 Kinase Activator NXP800 in Patients With Advanced Cholangiocarcionoma

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.

Read the detailed description

PRIMARY OBJECTIVE:

  1. To determine the maximum tolerated dose (MTD)/recommended phase 2 dose for heat shock factor 1 pathway inhibitor NXP800 (NXP800).

SECONDARY OBJECTIVES:

  1. To determine the toxicity profile of NXP800. II. To determine the best response for NXP800 using Response Evaluation Criteria in Solid Tumors (RECIST) version (v)1.1.

III. To estimate the overall survival (OS) for NXP800. IV. To estimate the progression-free survival (PFS) for NXP800.

EXPLORATORY OBJECTIVES:

  1. To evaluate transcriptomic features associated with sensitivity, resistance and pharmacodynamic effect of NXP800 using serial ribonucleic acid-sequencing (RNA-Seq).

II. To assess tumor evolution with NXP800 using serial whole genome-sequencing (Seq).

III. To assess tumor evolution with NXP800 using serial circulating tumor deoxyribonucleic acid (DNA) (ct-DNA).

IV. To estimate tumor marker response using serial CA19-9/carcinoembryonic antigen (CEA).

OUTLINE: This is a dose de-escalation study of NXP800 followed by a dose-expansion study.

Patients receive NXP800 orally (PO) according to assigned treatment schedule. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo computed tomography (CT), magnetic resonance imaging (MRI), and/or positron emission tomography (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.

After completion of study treatment, patients are followed up every 6 months until progressive disease or death for up to 3 years.

Being tested
Biospecimen Collection, Computed Tomography, Heat Shock Factor 1 Pathway Inhibitor NXP800, Magnetic Resonance Imaging, Positron Emission Tomography
Medical subject headings
Cholangiocarcinoma, Klatskin Tumor

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection

    Undergo collection of blood samples

  • Computed TomographyProcedure or surgery

    Also called CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography

    Undergo CT

  • Heat Shock Factor 1 Pathway Inhibitor NXP800Biological

    Also called CCT 361814, CCT-361814, CCT361814, HSF1 Pathway Inhibitor NXP800

    Given PO

  • Magnetic Resonance ImagingProcedure or surgery

    Also called Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan

    Undergo MRI

  • Positron Emission TomographyProcedure or surgery

    Also called Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron emission tomography (procedure)

    Undergo PET

  • Ultrasound-Guided BiopsyProcedure or surgery

    Also called Ultrasound Biopsy, Ultrasound Guided Biopsy

    Undergo ultrasound-guided tumor biopsy

Study groups in NCT06420349
Study groupRoleWhat they receive
Treatment (NXP800)ExperimentalPatients 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.
Receives: 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

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Up to 12 months

  2. Recommended phase 2 dose

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

    Measured: Up to 12 months

Secondary outcomes (4)

  1. Incidence of adverse events

    Overall toxicity incidence as well as toxicity profiles by dose level, patient and tumor site will be explored and summarized. Frequency distributions, graphical techniques and other descriptive measures will form the basis of these analyses. The grade 3+ adverse events will also be described and summarized in a similar fashion.

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

  2. Best response

    Best response is defined to be the best objective status recorded. The patient's best response assignment will depend on the achievement of both measurement and confirmation criteria. The Response Evaluation Criteria in Solid Tumors 1.1 criteria will be used for tumor evaluation and patients will be re-evaluated every 8 weeks. Responses will be summarized by simple descriptive summary statistics delineating complete and partial responses as well as stable and progressive disease in this patient population (overall and by dose level). The number of responses may indicate further evaluation for specific tumor types in a phase II setting.

    Measured: Up to 3 years

  3. Overall survival (OS)

    OS will be estimated using the Kaplan-Meier method. The median OS and corresponding 95% confidence interval (by Brookmeyer and Crowley) will be reported.

    Measured: Up to 3 years

  4. Progression-free survival (PFS)

    PFS will be estimated using the Kaplan-Meier method. The median PFS and corresponding 95% confidence interval (by Brookmeyer and Crowley) will be reported.

    Measured: Up to 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 May 2024
  2. Study start31 May 2024
  3. Primary completion12 Aug 2025
  4. Study completion9 Sep 2025

Why it stopped: study sponsor phasing out drug supply

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
7 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06420349
Sponsor's protocol ID
MC230406
Other IDs
NCI-2024-03613, MC230406, 23-012778
Status last verified
Dec 2025
First submitted
8 May 2024
Last updated
22 Dec 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • 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.

    PubMed 40717483

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Biospecimen Collection, Computed Tomography and Heat Shock Factor 1 Pathway Inhibitor NXP800 in people with Advanced Cholangiocarcinoma and Metastatic Cholangiocarcinoma, run by Mayo Clinic 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 18 inclusion and 17 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: stopped early, as of the sponsor's last update on 22 Dec 2025. 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?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 stopped early (reason given: study sponsor phasing out drug supply). Results are not available in the source record.

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?

Maximum tolerated dose (MTD) (time frame: Up to 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06420349, which also lists 1 linked publication.

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 22 Dec 2025.

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