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Familial Adenomatous Polyposis (FAP)Rare diseaseDrugPhase 3

What this study is

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.

What's being tested

  • eRapa (encapsulated rapamycin)

    Intervention · DRUG

    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

    Intervention · DRUG

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

Study details

Official title
A Phase 3, Multi-Site, Prospective, Randomized, Double-Blind, Placebo-Controlled Trial of eRapa to Improve Clinical Outcomes in Patients With Familial Adenomatous Polyposis
Sponsor
Rapamycin Holdings Inc.
Conditions
Familial Adenomatous Polyposis (FAP)
Enrolling
168 people
Starts
2025-07-18
Est. completion
2031-01
Design
Quadruple-blind, randomized, parallel groups, Phase 3
Record
View on ClinicalTrials.gov ↗