Phase 3 Trial of eRapa for Familial Adenomatous Polyposis
This clinical trial is studying whether a drug called eRapa (encapsulated rapamycin) can slow down the progression of Familial Adenomatous Polyposis (FAP), a condition that causes many polyps (growths) in the colon and rectum. Researchers want to see if eRapa is safe and effective compared to a placebo (an inactive substance). You might be able to join if you are at least 18 years old, have FAP confirmed by genetic testing, and have certain high-risk features like a large number of polyps. The main goal is to see if eRapa can improve progression-free survival (how long you live without your disease getting worse) over three years. The current recruitment status is unclear.
- Study design
- This is a randomized, double-blind, placebo-controlled study involving 168 participants. Participants will be randomly assigned to receive either eRapa or a placebo.
- What's involved
- You will take eRapa or placebo once a day every other week until your disease progresses, you stop participating, or the trial ends. You will also have endoscopy procedures every 6 months to monitor your disease.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary endpoint for measuring progression-free survival is at 3 years.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis
At a glance
Conditions
NCT06950385
Where you'd take part
This study runs at 29 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Amsterdam UMC
Amsterdam, Netherlandsstudy coordinator listed
Recruiting
Benaroya Research Institute at Virginia Mason
Seattle, Washingtonstudy coordinator listed
Recruiting
City of Hope
Arcadia, Californiastudy coordinator listed
Recruiting
Cleveland Clinic
Cleveland, Ohiostudy coordinator listed
Recruiting
Cleveland Clinic Florida
Weston, Floridastudy coordinator listed
Recruiting
Copenhagen University Hospital
Copenhagen, Denmarkstudy coordinator listed
Recruiting
Dana Farber Cancer Institute
Boston, Massachusettsstudy coordinator listed
Recruiting
Department of Surgery, Section of Colon Rectal and Surgery
St Louis, Missouristudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Gary Shangold, MD · STUDY_DIRECTOR · Biodexa
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Progression-free survival (PFS) in high-risk patients with FAP treated with eRapa versus placebo.3 years
* Death from any cause * Cancer/high-grade dysplasia * Major FAP-related surgery (e.g., colectomy, proctectomy, total proctocolectomy with ileal pouch anal anastomosis \[IPAA\], pouch resection, ileostomy, duodenectomy, or surgical ampullectomy) * Advancement of Spigelman stage (not related solely to increase in polyp number) * Meets criteria for surgery (consistent with United States \[US\] and European Union \[EU\] practice guidelines) (Yang, Gurudu et al. 2020, Zaffaroni, Mannucci et al.2024) * Retained rectum/sigmoid or pouch (≥10 polyps ≥3 mm in size at baseline) * Duodenum (Stage 3/4 and at least 1 polyp ≥10 mm removed in last 18 months)