Phase 3 eRapa treatment for people with Familial Adenomatous Polyposis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a randomized, double-blind, placebo-controlled Phase 3 trial enrolling 168 adults with genetically confirmed FAP who are considered high risk for progression. Participants are randomized 2:1 to oral 0.5 mg eRapa or matching placebo given once daily on an every-other-week dosing schedule, with treatment continued until disease progression or trial endpoint. Clinic visits occur every three months and endoscopic evaluations are performed at baseline and every six months to count and remove polyps and monitor progression. Patients are stratified by colon status (intact colon versus retained rectum/sigmoid or pouch) and by duodenal Spigelman stage.

Why it matters

Who can join: the full rules

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 after stopping study treatment.
5. Agree not to donate gametes for the purpose of reproduction until at at least 12 weeks after stopping study treatment.
6. Willing to undergo endoscopic evaluation.

Exclusion Criteria:

1. Participant has unresected or incompletely resected high-grade dysplasia or cancer within the duodenum, colon, rectum, or ileal pouch at screening endoscopy.
2. 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).
3. Participant has had surgery within 6 weeks of the trial.
4. Participant has active malignancy or history of malignancy diagnosed within 24 months of first dose of trial intervention.
5. Participant has a history of, or currently has, an acquired or primary (congenital) immunodeficiency.
6. Participant has active and clinically significant tuberculosis (positive Quantiferon Gold test), bacterial, fungal, or viral infection, including human immunodeficiency virus (HIV).
7. 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.

Where it's running

• City of Hope
  Arcadia, California, United States Recruiting
• Yale Cancer Center
  New Haven, Connecticut, United States Recruiting
• Georgetown University
  Washington D.C., District of Columbia, United States Recruiting
• Digestive & Liver Center of Florida
  Orlando, Florida, United States Recruiting
• Cleveland Clinic Florida
  Weston, Florida, United States Recruiting
• University of Chicago
  Chicago, Illinois, United States Recruiting

Study contacts

• Study coordinator: Vice President Clinical Operations
• Email: jim.kostka@biodexapharma.com
• Phone: XXX-XXX-XXXX

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.