Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis (NCT06950385) | Clinical Trial Compass
RecruitingPhase 3
Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis
United States, Denmark, Germany168 participantsStarted 2025-07-18
Plain-language 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Participant must be ≥18 years of age inclusive.
. Participant must have documented FAP, confirmed by adenomatous polyposis coli genotype mutation testing.
. 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.
. Contraceptive use by participants or participant partners until at at least 12 weeks after stopping study treatment.
. Agree not to donate gametes for the purpose of reproduction until at at least 12 weeks after stopping study treatment.
. Willing to undergo endoscopic evaluation.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Progression-free survival (PFS) in high-risk patients with FAP treated with eRapa versus placebo.
. Participant has unresected or incompletely resected high-grade dysplasia or cancer within the duodenum, colon, rectum, or ileal pouch at screening endoscopy.
. Participant has any polyps ≥8 mm in the duodenum, colon, rectum, or ileal pouch remaining after screening endoscopy (polyps ≥8 mm are to be resected during screening endoscopy).
. Participant has had surgery within 6 weeks of the trial.
. Participant has active malignancy or history of malignancy diagnosed within 24 months of first dose of trial intervention.
. Participant has a history of, or currently has, an acquired or primary (congenital) immunodeficiency.
. Participant has active and clinically significant tuberculosis (positive Quantiferon Gold test), bacterial, fungal, or viral infection, including human immunodeficiency virus (HIV).
. Participant has any medical or social condition that, in the opinion of the Investigator, might increase participant risk if enrolled, prevent participant compliance to trial procedures, or present an unacceptable confound to safety or clinical trial data.