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Phase 3 eRapa treatment for people with Familial Adenomatous Polyposis

A Phase 3, Multi-Site, Prospective, Randomized, Double-Blind, Placebo-Controlled Trial of eRapa to Improve Clinical Outcomes in Patients With Familial Adenomatous Polyposis

Phase 3 Interventional Rapamycin Holdings Inc. · NCT06950385

This will see if taking eRapa instead of placebo can slow disease progression and reduce polyps in adults with Familial Adenomatous Polyposis (FAP).

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment168 (estimated)
Ages18 Years and up
SexAll
SponsorRapamycin Holdings Inc. Industry-sponsored
Locations26 sites (Arcadia, California and 25 other locations)
Trial IDNCT06950385 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Adults (≥18) with genetically confirmed FAP who meet high-risk criteria such as >100 but ≤500 colonic polyps, or ≥10 polyps in a retained rectum/sigmoid or ileal pouch, or Spigelman stage 3–4 with at least one ≥10 mm polyp are the intended participants.

Not a fit: Patients with unresected or incompletely resected high-grade dysplasia or active cancer at screening, people without high-risk FAP features, or those unable to undergo regular endoscopy are unlikely to benefit from this trial.

Why it matters

Potential benefit: If successful, eRapa could slow FAP progression, reduce polyp burden, and delay or reduce the need for additional surgeries, improving patient outcomes and quality of life.

How similar studies have performed: Preclinical data and small early-phase reports suggest mTOR inhibitors like rapamycin can reduce polyp growth, but large randomized phase 3 evidence in FAP is limited, so this approach is relatively novel at scale.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 this trial is running

Arcadia, California and 25 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Familial Adenomatous PolyposisFAPPolyposisPolypsileal pouch
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.