NXP800 treatment for advanced cholangiocarcinoma
Phase 1b Study of the Novel GCN2 Kinase Activator NXP800 in Patients With Advanced Cholangiocarcionoma
This study is testing a new drug called NXP800 to see if it can safely help people with advanced bile duct cancer live longer and feel better.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Mayo Clinic Academic / other |
| Drugs / interventions | radiation |
| Locations | 2 sites (Scottsdale, Arizona and 1 other locations) |
| Trial ID | NCT06420349 on ClinicalTrials.gov |
What this trial studies
This phase I trial evaluates the safety, optimal dosage, and effectiveness of NXP800 in patients with advanced or metastatic cholangiocarcinoma. The study focuses on inhibiting the heat shock factor 1 (HSF1) pathway, which is implicated in tumor growth and spread. Participants will receive NXP800 orally, with treatment cycles repeating every 28 days, and will undergo various imaging and biomarker assessments to monitor response and safety. The trial aims to establish the maximum tolerated dose and assess overall and progression-free survival.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older with histologically confirmed advanced or metastatic biliary tract cancer that is refractory to standard therapies.
Not a fit: Patients with early-stage cholangiocarcinoma or those who have not received prior treatment may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with advanced cholangiocarcinoma who have limited treatment choices.
How similar studies have performed: While the approach of targeting the HSF1 pathway is innovative, similar studies have shown promise in other cancer types, indicating potential for success in this context.
Eligibility criteria
Show full inclusion / exclusion 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) ≤ 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) 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
Where this trial is running
Scottsdale, Arizona and 1 other locations
- Mayo Clinic in Arizona — Scottsdale, Arizona, United States (Recruiting)
- Mayo Clinic in Rochester — Rochester, Minnesota, United States (Recruiting)
Study contacts
- Principal investigator: Mitesh J. Borad, MD — Mayo Clinic
- Study coordinator: Clinical Trials Referral Office
- Email: mayocliniccancerstudies@mayo.edu
- Phone: 855-776-0015
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.