Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis

Launched by RAPAMYCIN HOLDINGS INC. · Apr 24, 2025

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ClinConnect Summary

This clinical trial is studying a new drug called eRapa to see if it can help slow down the progression of Familial Adenomatous Polyposis (FAP), a condition that causes many polyps (growths) to form in the intestines. Researchers want to find out if eRapa is effective, safe, and how it affects the quality of life for patients with FAP compared to a placebo, which is a dummy treatment that has no active ingredients. Participants in the trial will take eRapa or the placebo once a day every other week and will have regular check-ups at the clinic every three months to monitor their health.

To be eligible for this trial, participants must be at least 18 years old and have been diagnosed with FAP, confirmed by specific genetic testing. They should also have certain high-risk features, such as having a specific number of polyps in their colon. Participants will undergo an endoscopy, which is a procedure to look inside the intestines, at the beginning of the trial and then every six months to assess their condition. It’s important to note that those with certain serious health issues or who have had recent surgeries may not qualify for the study. Overall, this trial is a step towards finding new ways to manage FAP and improve the lives of those affected by it.

Gender

ALL

Eligibility 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.