Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2025 Jul 28:1-8.
doi: 10.1080/14796694.2025.2539611. Online ahead of print.

A Phase 1 study of the novel GCN2 kinase activator NXP800 in patients with advanced cholangiocarcinoma

Hendrien Kuipers  1   2 , Joseph J Larson  3 , Caitlin B Conboy  4 , Daniel H Ahn  5 , Tanios Bekaii-Saab  5 , Christina Wu  5 , Mohamad Bassam Sonbol  5 , Sumera I Ilyas  6 , Gregory J Gores  6 , Rory L Smoot  1   2 , Mitesh J Borad  5
Affiliations

A Phase 1 study of the novel GCN2 kinase activator NXP800 in patients with advanced cholangiocarcinoma

Hendrien Kuipers et al. Future Oncol. .

Abstract

www.clinicaltrials.gov identifier is NCT06420349.

Keywords: Cholangiocarcinoma; GCN2; NXP800; clinical trials; integrated stress response.

Plain language summary

Bile duct cancer (also called cholangiocarcinoma) is a rare type of cancer that is often found at a late stage. Most patients do not respond well to standard treatments like chemotherapy, so new therapies are urgently needed. This paper describes a clinical trial that will test a new medicine called NXP800. This drug activates a natural process in cells, called the stress response, to cause cancer cells to stop growing or die. 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 also been tested in patients with other advanced cancers in another clinical trial, where the dose and side effects were studied. The new clinical trial described here will include patients with advanced bile duct cancer who have already tried chemotherapy. Patients will be given oral capsules of NXP800 once a day. The main goal of the study is to find the highest dose that can be safely given and the recommended dose for future studies. The trial will also assess side effects and whether the treatment helps slow or stop cancer growth.

PubMed Disclaimer

Similar articles

See all similar articles

Associated data

LinkOut - more resources