According to a story from globenewswire.com, the biopharmaceutical company Lexicon Pharmaceuticals has recently announced that it has completed its safety review of the initial cohort for its phase 2 clinical trial, which is testing the company’s drug telotristat ethyl (marketed as XERMELO) as a treatment for cholangiocarcinoma, a rare cancer that affects the bile ducts and biliary tract. The initial cohort includes six patients that were treated for at least 21 days with telotristat ethyl in combination with gemcitabine and cisplastin.
Cholangiocarcinoma is a form of cancer that affects the bile ducts. More specifically, the epithelial cells of the bile duct are the ones affected by this disease. The cause is not directly known; many patients who develop the disease also have no known risk factors, but there are some factors that are known to increase risk, such as primary sclerosing cholangitis, chronic liver diseases such as hepatitis, certain genetic disorders such as Caroli’s syndrome, infection with Helicobacter bacteria, and certain liver flukes, a type of parasitic animal that infiltrates the bile ducts and feeds on bile. Symptoms include fever, weight loss, jaundice, itching, and abdominal pain. Treatment may include surgery, chemotherapy, radiation, or liver transplant. Transplant is usually only useful if the disease is detected early; surgery is the best chance for curing cholangiocarcinoma. This disease is rapidly lethal if surgical removal is not possible. To learn more about cholangiocarcinoma, click here.
About The Trial
These patients were receiving the drug three times per day at a dose level of 500 mg. Two patients in this cohort have been undergoing treatment for six months and the findings support proceeding with enrollment for the phase 2 trial. The study is expected to include a total of 53 cholangiocarcinoma patients who may receive telotristat ethyl alongside standard chemotherapy (cisplatin and gemicitabine). The trial’s primary endpoint will be progression-free survival after six months. Secondary endpoints will include progression free survival after a year, overall survival after six months and after a year, and overall survival in general.
Telotristat ethyl is currently approved in the US and several other countries as a treatment for diarrhea caused by carcinoid syndrome.