Trial Title:
NXP800 for the Treatment of Patients with Advanced or Metastatic Cholangiocarcinoma
NCT ID:
NCT06420349
Condition:
Advanced Cholangiocarcinoma
Metastatic Cholangiocarcinoma
Refractory Cholangiocarcinoma
Stage III Hilar Cholangiocarcinoma AJCC V8
Stage III Intrahepatic Cholangiocarcinoma AJCC V8
Stage IV Hilar Cholangiocarcinoma AJCC V8
Stage IV Intrahepatic Cholangiocarcinoma AJCC V8
Conditions: Official terms:
Cholangiocarcinoma
Klatskin Tumor
Study type:
Interventional
Study phase:
Phase 1
Overall status:
Recruiting
Study design:
Allocation:
N/A
Intervention model:
Single Group Assignment
Primary purpose:
Treatment
Masking:
None (Open Label)
Intervention:
Intervention type:
Procedure
Intervention name:
Biospecimen Collection
Description:
Undergo collection of blood samples
Arm group label:
Treatment (NXP800)
Other name:
Biological Sample Collection
Other name:
Biospecimen Collected
Other name:
Specimen Collection
Intervention type:
Procedure
Intervention name:
Computed Tomography
Description:
Undergo CT
Arm group label:
Treatment (NXP800)
Other name:
CAT
Other name:
CAT Scan
Other name:
Computed Axial Tomography
Other name:
Computerized Axial Tomography
Other name:
Computerized axial tomography (procedure)
Other name:
Computerized Tomography
Other name:
Computerized Tomography (CT) scan
Other name:
CT
Other name:
CT Scan
Other name:
tomography
Intervention type:
Biological
Intervention name:
Heat Shock Factor 1 Pathway Inhibitor NXP800
Description:
Given PO
Arm group label:
Treatment (NXP800)
Other name:
CCT 361814
Other name:
CCT-361814
Other name:
CCT361814
Other name:
HSF1 Pathway Inhibitor NXP800
Other name:
NXP-800
Other name:
NXP800
Other name:
VK2019
Intervention type:
Procedure
Intervention name:
Magnetic Resonance Imaging
Description:
Undergo MRI
Arm group label:
Treatment (NXP800)
Other name:
Magnetic Resonance
Other name:
Magnetic Resonance Imaging (MRI)
Other name:
Magnetic resonance imaging (procedure)
Other name:
Magnetic Resonance Imaging Scan
Other name:
Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance
Other name:
MR
Other name:
MR Imaging
Other name:
MRI
Other name:
MRI Scan
Other name:
MRIs
Other name:
NMR Imaging
Other name:
NMRI
Other name:
Nuclear Magnetic Resonance Imaging
Other name:
sMRI
Other name:
Structural MRI
Intervention type:
Procedure
Intervention name:
Positron Emission Tomography
Description:
Undergo PET
Arm group label:
Treatment (NXP800)
Other name:
Medical Imaging, Positron Emission Tomography
Other name:
PET
Other name:
PET Scan
Other name:
Positron emission tomography (procedure)
Other name:
Positron Emission Tomography Scan
Other name:
Positron-Emission Tomography
Other name:
proton magnetic resonance spectroscopic imaging
Other name:
PT
Intervention type:
Procedure
Intervention name:
Ultrasound-Guided Biopsy
Description:
Undergo ultrasound-guided liver biopsy
Arm group label:
Treatment (NXP800)
Other name:
Ultrasound Biopsy
Other name:
Ultrasound Guided Biopsy
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.
Detailed description:
PRIMARY OBJECTIVE:
I. To determine the maximum tolerated dose (MTD)/recommended phase 2 dose for heat shock
factor 1 pathway inhibitor NXP800 (NXP800).
SECONDARY OBJECTIVES:
I. 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:
I. 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 liver 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.
Criteria for eligibility:
Criteria:
Inclusion Criteria:
- 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) ≤ 2.5 x upper limit of normal (ULN) (obtained ≤ 14
days prior to registration)
- Alanine aminotransferase (ALT) ≤ 2.5 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)
Exclusion Criteria:
- 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
- Unwillingness to follow study related procedures
- Inability to provide informed consent
Gender:
All
Minimum age:
18 Years
Maximum age:
N/A
Healthy volunteers:
No
Locations:
Facility:
Name:
Mayo Clinic in Arizona
Address:
City:
Scottsdale
Zip:
85259
Country:
United States
Status:
Recruiting
Contact:
Last name:
Clinical Trials Referral Office
Phone:
855-776-0015
Email:
mayocliniccancerstudies@mayo.edu
Contact backup:
Last name:
Mitesh J. Borad, M.D.
Start date:
May 31, 2024
Completion date:
May 2026
Lead sponsor:
Agency:
Mayo Clinic
Agency class:
Other
Source:
Mayo Clinic
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06420349
https://www.mayo.edu/research/clinical-trials
