Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis

Condition(s) studied

Investigational drug(s) / intervention(s)

eRapa (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.

Placebo: Capsules in 28-count round high-density polyethylene bottles with a polypropylene child-resistant screw cap and foil induction seal.

Study 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.

Eligibility

Primary outcome measure(s)

• 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)

Trial sites (29)

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