NXP800 for Bile Duct Cancer

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

30 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

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.

Research Team

Mitesh J. Borad, MD

Principal Investigator

Mayo Clinic

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+