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Lexicon early on established an expanded access trial for those who screen-failed that would collect long-term safety data.
Fresh from moderating a Fireside Chat regarding issues around compassionate use with Kenneth Moch at the Partnerships in Clinical Trials conference in Boston, the former CEO of Chimerix, Pablo Lapuerta, Executive Vice President, Clinical Development and Chief Medical Office of Lexicon Pharmaceuticals, delivered his presentation on how his company achieved a patient-centric study design for its Phase III trial of Carcinoid Syndrome therapy. Carcinoid syndrome are rare tumors that occur mostly in the gastrointestinal tract or lungs.
In establishing the Phase III trial, Lapuerto explained the goal was to focus on the patient. That meant keep the protocol very friendly by including home visits, limiting the number of procedures and keeping real-world care in mind. This cancer is so rare that it took two years to enroll 23 patients.
Lexicon early on established an expanded access trial for those who screen-failed that would collect long-term safety data. The sites become more engaged with this second offering, as Lapuerto explained, because they wouldn’t be excluding those patients who could benefit from the investigational drug.
Lexicon’s executive team was very involved with the sites and would visit them often to learn about the barriers to the trial. This showed the sites that the sponsor was very committed to its goals, according to Lapeurto. In addition, the company asked investigators and its CRO to attend the scientific meetings in this specialty to help education.
The company is hoping to roll out top-line Phase III results later this summer. And Lapuerto believes that the second arm for expanded access was a better option to address patient needs than a compassionate use program would have been.