Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis

This is a Phase 3, multi-site, prospective, randomized, double-blind, placebo-controlled trial of eRapa administered to patients with FAP who are at high risk of disease progression. 168 patients with FAP will be enrolled in the trial and randomized 2:1 to receive 0.5 mg eRapa or matching placebo orally, once a day (QD) every other week. There is no minimum treatment duration as this is an event-driven trial; however, the intervention period will continue until disease progression, participant withdrawal from treatment, or until the overall trial endpoint is reached. Participant eligibility is restricted to patients under active surveillance for genetic or clinically diagnosed FAP and who have an intact colon; who are postcolectomy/subtotal colectomy and have documented residual polyps in the rectum/sigmoid or who are post-proctocolectomy with ileal-pouch anal anastomosis and documented polyps in the pouch. Eligible participants will undergo a baseline endoscopy and subsequent endoscopic procedures performed every 6 months to monitor for disease progression. Randomized patients will be stratified based on the following disease characteristics: * Intact colon versus post-surgical resection with retained rectum/sigmoid or pouch, and * Duodenal polyposis (current Spigelman stage score ≤2 versus Spigelman stage score ≥3) For the purposes of this trial, high-risk for disease progression is defined as meeting one of the following: * Patients who have intact colons and have \>100 polyps but ≤500 polyps * Patients who have retained rectum/sigmoid or ileal-pouch-anal anastomosis and have ≥10 polyps that are ≥3 mm in diameter, or * Patients who have a history of duodenal polyposis Spigelman stage score of 3 or 4 with at least 1 duodenal polyp that has been removed within 18 months of screening. Trial assessments should be conducted as per the Schedule of Activities with a visit occurring about once every 3 months. Assessment of Spigelman stage will not require a biopsy unless the lesion has an abnormal appearance and/or is ≥10 mm.