NXP800 for Bile Duct Cancer

Not currently recruiting at 1 trial location

Overseen By Clinical Trials Referral Office

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Mayo Clinic

Must not be taking: CYP3A4 inhibitors

Disqualifiers: Pregnancy, Heart failure, Brain metastasis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 28

Duration: Up to 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the safety and effectiveness of a new treatment, NXP800, for individuals with advanced or metastatic bile duct cancer. The goal is to determine if NXP800 can halt cancer growth and spread by targeting a specific pathway that many cancer cells use for survival and proliferation. Those whose bile duct cancer has not responded well to standard treatments like gemcitabine or fluoropyrimidine might be suitable candidates. Participants will receive NXP800 in cycles, with regular scans to monitor progress. As a Phase 1 trial, this research aims to understand how NXP800 works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial requires that you stop using drugs that strongly affect CYP3A4 (a liver enzyme) at least 7 days before starting and during the study. Other medications may be used with caution, but the protocol does not specify if you need to stop all current medications.

Is there any evidence suggesting that NXP800 is likely to be safe for humans?

Research has shown that NXP800, a treatment being tested for bile duct cancer, has shown promise in early studies. These studies found that NXP800 can slow the growth of cancer cells in the lab. This suggests it might be effective in treating cancer, but the main goal now is to assess its safety for people.

As an early-phase trial, the focus is on finding the best dose and identifying any side effects. Early-phase trials often have limited safety information, but they are crucial for determining if the treatment is safe enough for further testing.

In summary, NXP800 has potential, and this trial will help determine how well people tolerate it and the safest way to use it. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for bile duct cancer?

Researchers are excited about NXP800 for bile duct cancer because it offers a novel approach compared to traditional treatments like surgery, chemotherapy, and radiation. Unlike these standard options, NXP800 targets specific molecular pathways involved in cancer growth, potentially offering a more precise attack on the cancer cells. This targeted action may lead to fewer side effects and better outcomes, which is why there's a buzz in the medical community about its potential.

What evidence suggests that NXP800 might be an effective treatment for bile duct cancer?

Research has shown that NXP800, the investigational treatment in this trial, has potential in lab tests, where it slowed the growth of bile duct cancer cells and tumors. This treatment targets a specific pathway in the body that is often overly active in many cancers, including bile duct cancer. By blocking this pathway, NXP800 may help stop cancer cells from growing and spreading. Animal studies demonstrated its effectiveness, leading to its recognition as an "orphan drug" by the FDA, a special status for promising treatments of rare diseases. Although human trial data remains limited, these early results offer hope for treating advanced bile duct cancer. ^{1 2 4 5 6}

Who Is on the Research Team?

Mitesh J. Borad, MD

Principal Investigator

Mayo Clinic

Are You a Good Fit for This Trial?

This trial is for individuals with advanced or metastatic cholangiocarcinoma, which includes various types of bile duct cancer. Participants should have a form of the disease that has spread and are required to undergo specific imaging tests like PET scans, CT scans, MRIs, and biopsies.

Inclusion Criteria

Measurable disease by RECIST 1.1

Anticipated life expectancy of > 12 weeks

Hemoglobin ≥ 9.0 g/dL (obtained ≤ 14 days prior to registration)

Exclusion Criteria

Pregnant persons

Nursing persons

Persons of childbearing potential who are unwilling to employ adequate contraception

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive NXP800 orally according to assigned treatment schedule. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Up to 12 months

Every 28 days

Follow-up

Participants are monitored for safety and effectiveness after treatment. Follow-up includes CT, MRI, and/or PET scans, and optional liver biopsy and blood sample collection.

Every 6 months for up to 3 years

What Are the Treatments Tested in This Trial?

Interventions

• NXP800

Trial Overview The trial is evaluating NXP800's safety and optimal dosage. NXP800 targets the HSF1 pathway involved in tumor growth and spread. The study will assess if inhibiting this pathway can reduce tumor activity in patients with advanced or metastatic cholangiocarcinoma.

How Is the Trial Designed?

Group I: Treatment (NXP800) Experimental Treatment 6 Interventions

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.

NXP800 is already approved in United States for the following indications:

Approved in United States as NXP800 for:

• None approved; granted Fast Track and Orphan Drug Designation for ARID1a-deficient ovarian, fallopian tube, and peritoneal cancers, and Orphan Drug Designation for cholangiocarcinoma

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06420349

NCT06420349 | NXP800 for the Treatment of Patients With ... 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 ...

2. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/486995

NXP800 for the Treatment of Patients With Advanced ... This phase I trial tests the safety, best dose, and effectiveness of NXP800 in treating patients with cholangiocarcinoma that may have spread.

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40717483/

A Phase 1 study of the novel GCN2 kinase activator ... NXP800 has already been tested in the lab, where it slowed down the growth of bile duct cancer cells and human bile duct cancer tumors. It has ...

4. onclive.com onclive.com/view/fda-grants-orphan-drug-designation-to-nxp800-for-cholangiocarcinoma

FDA Grants Orphan Drug Designation to NXP800 for ... In September 2022, Nuvectis released positive preclinical data for NXP800. The study, which was performed in a xenograft mouse model of ARID1a- ...

5. pharmaceutical-technology.com pharmaceutical-technology.com/data-insights/nxp-800-nuvectis-pharma-bile-duct-cancer-cholangiocarcinoma-likelihood-of-approval/

NXP-800 by Nuvectis Pharma for Bile Duct Cancer ... According to GlobalData, Phase I drugs for Bile Duct Cancer (Cholangiocarcinoma) have an 87% phase transition success rate (PTSR) indication benchmark for ...

6. mayo.edu mayo.edu/research/clinical-trials/diseases-conditions/cholangiocarcinoma-(bile-duct-cancer)

Cholangiocarcinoma (Bile Duct Cancer) Clinical Trials The purpose of this study is assess the effectiveness of trifluridine/tipiracil plus irinotecan in treating advanced, refractory biliary tract cancers (BTCs).