Phase 3 Study of eRapa to Slow Disease Progression in Patients With Familial Adenomatous Polyposis Randomized, Double-Blind, Placebo-Controlled Trial Across Multiple Sites

What this Trial Is About

Researchers are evaluating if the drug eRapa can slow the progression of Familial Adenomatous Polyposis FAP, a condition characterized by the development of numerous polyps in the gastrointestinal tract. This Phase 3 trial compares eRapa to a placebo to understand its impact on disease progression, safety, number of polyps, and patients quality of life. The study involves patients at high risk of disease progression under active surveillance with specific colon or post-surgical conditions. Participants will be randomly assigned to receive either 0.5 mg of eRapa or a matching placebo orally once daily every other week. The treatment continues until disease progression, withdrawal, or trial endpoint. Patients will have baseline and follow-up endoscopies every 6 months to monitor disease status, with visits every 3 months for check-ups and tests. Stratification will consider colon status and duodenal polyposis severity. During the study, participants will attend clinic visits approximately every 3 months for assessments, including endoscopies to evaluate disease progression. Researchers will measure progression-free survival over three years, safety and tolerability of eRapa, effects on GI polyposis and Spigelman stage score, and quality of life using standardized questionnaires. The trial is designed to monitor participants closely for changes and treatment effects over time.

Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis