A Phase 3, Multi-Site, Prospective, Randomized, Double-Blind, Placebo-Controlled Trial of eRapa to Improve Clinical Outcomes in Patients With Familial Adenomatous Polyposis
Rapamycin Holdings Inc.
Summary
The main goal of this clinical trial is to learn if the drug eRapa works to slow down the progression of disease in patients diagnosed with Familial Adenomatous Polyposis (FAP). Researchers will compare eRapa to Placebo. The questions to be answered by this trial are: * Does taking eRapa help to slow down the progression of the disease in patients with FAP? * Is eRapa a safe treatment for patients diagnosed with FAP? * What is the effect of eRapa on the number of polyps found in GI tract of patients diagnosed with FAP? * How does treatment with eRapa affect a patient's quality of life? Participants will: * Take eRapa or placebo once per day every other week until disease progresses (gets worse), stops taking part in the trial or dies. * Visit the clinic once every 3 months for check ups and tests. * Have an endoscopy at the start of the trial and then every 6 months to check on whether the disease is getting better or worse.
Description
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…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Participant must be ≥18 years of age inclusive. 2. Participant must have documented FAP, confirmed by adenomatous polyposis coli genotype mutation testing. 3. Participant must have at least 1 of the following high-risk features: \>100 polyps but ≤500 polyps in the colon, or ≥10 polyps in the retained rectum/sigmoid or ileal pouch (≥3 mm in size), or Spigelman stage 3 or 4 with at least 1 polyp ≥10 mm to be removed at baseline or on endoscopy performed within 18 months of screening. 4. Contraceptive use by participants or participant partners until at at least 12 weeks a…
Interventions
- DrugeRapa (encapsulated rapamycin)
0.5 mg capsules for oral use; white opaque capsule filled with off-white powder; Trial intervention will be provided in 28-count round high-density polyethylene bottles with a polypropylene child-resistant screw cap and foil induction seal.
- DrugPlacebo
Capsules in 28-count round high-density polyethylene bottles with a polypropylene child-resistant screw cap and foil induction seal.
Locations (26)
- City of HopeArcadia, California
- Yale Cancer CenterNew Haven, Connecticut
- Georgetown UniversityWashington D.C., District of Columbia
- Digestive & Liver Center of FloridaOrlando, Florida
- Cleveland Clinic FloridaWeston, Florida
- University of ChicagoChicago, Illinois