Phase 3 trial initiated for treatment of rare bile duct cancer

A phase 3 clinical trial is under way to study the safety and efficacy of photodynamic therapy with porfimer sodium to treat a rare form of bile duct cancer that currently has no other therapeutic options, according to a press release.

Porfimer sodium (Photofrin, Pinnacle Biologics) is a photosensitizing injectable agent used to treat various cancers, including esophageal, non-small cell lung cancer and high grade dysplasia in Barrett’s esophagus. Pinnacle, a subsidiary of Concordia Healthcare, established an agreement with the FDA in January under a special protocol assessment to enroll patients in the trial.

“The fast pace at which we have advanced this trial … is an indication to us of the medical community’s support and need for new treatments like [photodynamic therapy] with Photofrin,” Mark Thompson, chief executive officer of Concordia, said in the release.

The trial includes 200 patients from the US, Switzerland, Germany, South Korea and Canada and will test the combination of photodynamic therapy with porfimer sodium to treat unresectable advanced perihilar cholangiocarcinoma Bismuth type III/IV. US patients are enrolled at Thomas Jefferson University Hospital, Philadelphia, the first site approved for enrollment, the release said.

“The approved treatment options for patients with bile duct cancer are inadequate and only prolong life by a few months,” David E. Loren, MD, associate director of endoscopy and co-director of the pancreaticobiliary section who will lead the trial at Jefferson, said in the release. “Previous studies have shown promise for photodynamic therapy with Photofrin in prolonging life more than a year for those affected by this terrible cancer. We are hopeful that this trial will provide the results needed to approve the use of this treatment for use in cholangiocarcinoma.”

Porfimer sodium was granted orphan drug designation (ODD) by the FDA in 2004 to treat cholangiocarcinoma and in December 2011, the FDA granted a second ODD to the drug as adjuvant therapy to surgery for the treatment of malignant pleural mesothelioma, according to the release.