D07001 + Capecitabine for Biliary Tract Cancer

This trial investigates whether D07001-softgel capsules, combined with capecitabine, can improve survival in patients with advanced biliary tract cancer, which affects the bile ducts, gallbladder, or ampullary region. Participants will receive either one of two doses of D07001-softgel capsules or a placebo, all alongside capecitabine. The goal is to determine if the new treatment extends life compared to capecitabine with a placebo. The trial seeks individuals with advanced biliary tract cancer who have tried other treatments but need new options. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants access to a potentially life-extending treatment.

The trial protocol does not specify if you need to stop taking your current medications. However, you cannot participate if you are currently taking flucytosine or have taken brivudine, sorivudine, or similar drugs within 28 days before enrollment.

Research has shown that D07001-softgel capsules, when combined with capecitabine, are generally well-tolerated. In one study, patients with solid tumors safely took doses of D07001 up to 100 mg per day. However, some patients experienced serious liver damage at higher doses of 120 mg. This indicates that while most patients tolerated lower doses well, higher amounts led to liver issues for some.

Capecitabine, the other component of the combination, is a standard treatment for biliary tract cancer and is generally considered safe, though it can have side effects like many treatments.

Unlike the standard treatments for biliary tract cancer, which often involve chemotherapy drugs like gemcitabine and cisplatin, the investigational drug D07001 offers a novel approach. This treatment is combined with Capecitabine and is available in softgel capsules, making it potentially more convenient and easier to administer. Researchers are particularly excited about D07001 because it introduces a new active ingredient in the form of a softgel capsule, which may enhance absorption and effectiveness. Additionally, the unique dosing schedule of D07001 allows for targeted therapy that could improve patient outcomes.

This trial will examine the combination of D07001-softgel and capecitabine to determine if it can extend the lives of biliary tract cancer patients. Studies have shown that capecitabine, when combined with other treatments, can be effective for this cancer type. Capecitabine alone has also helped some patients live longer. Research suggests that D07001-softgel, a form of gemcitabine, might enhance effectiveness when paired with capecitabine. Previous studies demonstrated that D07001 is safe for patients with solid tumors, supporting its potential in this combination treatment.⁶⁷⁸⁹¹⁰