The newly initiated Focus trial will examine the safety, efficacy and pharmocokinetic profile of its Melphalan and HDS system when compared with the standard of care, the New York City-based company said.
“We believe the Focus trial puts us on the fastest path to a regulatory submission in the U.S. and initiation of this trial is a landmark event for Delcath. We are delighted to be working with the Moffitt Cancer Center and look forward to activating a number of other premier cancer centers as clinical sites in the coming months. Our goal is to have an interim analysis, which we expect to occur in the 2nd half of 2017. We look forward to bringing this potentially life-saving therapy to patients suffering with hepatic ocular melanoma,” CEO Dr. Jennifer Simpson said in a press release.
The trial is slated to enroll 240 patients, with a primary endpoint comparing overall survival between 2 arms of the trial and secondary endpoints including progression-free survival, overall response rate and quality of life measures.
The company is conducting the trial under a Special Protocol Assessment it received from the FDA earlier this week, and will be the only Phase III trial required for the company to submit a New Drug Application, Delcath said.
“I am particularly pleased to serve as principal investigator in this very promising study as I have treated patients with Melphalan/HDS through both formal clinical research and compassionate use since 2007. Our experience at Moffitt with Melphalan/HDS in patients with hepatic dominant ocular melanoma has shown significant potential. We are pleased to be taking a leadership role in the Focus trial, and look forward to verifying the potential for Melphalan/HDS in this life-threatening cancer with no effective treatment options,” trial principal investigator Dr. Jonathan Zager of Tampa, Fla.’s Moffitt Cancer Center said in a prepared statement.
For the treatment phase of the trial, patients will be split into arms and monitored on either the standard-of-care or Melphalan and HDS treatment over intervals of 6 to 8 weeks for up to 12 months. Tumor response will be assessed in both study arms every 12 weeks to gather evidence, the company said. In the follow-up phase of the trial, patients will be monitored eveyr 12 weeks for up to 12 years.
“The Focus trial will utilize an improved Melphalan/HDS product/procedure that addresses safety issues raised in our previous Phase 3 study. Based on our commercial experience in Europe, and the bolus of clinical data recently presented and published, we are optimistic that the Focus Trial will demonstrate a compelling benefit/risk profile and that the study’s objectives will be met,” Dr. Simpson said in prepared remarks.