A Phase 3 interventional study of eRapa (encapsulated rapamycin) and Placebo in Familial Adenomatous Polyposis (FAP), sponsored by Rapamycin Holdings Inc.. Recruiting at 29 sites in 7 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-06-02.
Sponsored by Rapamycin Holdings Inc. · Phase 3, Interventional, and Treatment
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:
Participants will:
This is a Phase 3, multi-site, prospective, randomized, double-blind, placebo-controlled trial of eRapa administered to patients with FAP who are at high risk of disease progression. 168 patients with FAP will be enrolled in the trial and randomized 2:1 to receive 0.5 mg eRapa or matching placebo orally, once a day (QD) every other week. There is no minimum treatment duration as this is an event-driven trial; however, the intervention period will continue until disease progression, participant withdrawal from treatment, or until the overall trial endpoint is reached. Participant eligibility is restricted to patients under active surveillance for genetic or clinically diagnosed FAP and who have an intact colon; who are postcolectomy/subtotal colectomy and have documented residual polyps in the rectum/sigmoid or who are post-proctocolectomy with ileal-pouch anal anastomosis and documented polyps in the pouch. Eligible participants will undergo a baseline endoscopy and subsequent endoscopic procedures performed every 6 months to monitor for disease progression.
Randomized patients will be stratified based on the following disease characteristics:
Trial assessments should be conducted as per the Schedule of Activities with a visit occurring about once every 3 months.
Assessment of Spigelman stage will not require a biopsy unless the lesion has an abnormal appearance and/or is ≥10 mm.
92 studies on the registry are indexed under Adenomatous Polyposis Coli; 29 are open to participants now.
This study's planned enrollment of 168 is above the median of 40 across 61 interventional studies indexed under Adenomatous Polyposis Coli.
Browse Adenomatous Polyposis Coli studies →Rapamycin Holdings Inc. is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
0.5 mg eRapa once a day (QD) every other week
Drug: eRapa (encapsulated rapamycin)
Placebo once a day (QD) every other week
Drug: Placebo
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.
Capsules in 28-count round high-density polyethylene bottles with a polypropylene child-resistant screw cap and foil induction seal.
Progression-free survival (PFS) in high-risk patients with FAP treated with eRapa versus placebo.
* 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)
Time frame: 3 years
The safety and tolerability of eRapa in patients with FAP
-Frequency of all grades (as per the Common Terminology Criteria for Adverse Events \[CTCAE\] version \[v\] 5.0) treatment-emergent adverse events (TEAEs) in participants receiving eRapa; * Frequency of Grade 3 and higher (as per the CTCAE v5.0) TEAEs in participants receiving eRapa * Percentage of participants discontinuing eRapa treatment due to adverse drug reactions (ADRs)
Time frame: 3 years
The effect of eRapa treatment on GI polyposis in patients with FAP
* Percent change from baseline in the PB (i.e., the sum of the diameters of all polyps \>3 mm) at 6, 12, 18, 24, 30, and 36 months as observed by surveillance endoscopy Note: The total PB will be based on polyps observed in the upper GI tract (duodenum) and the lower GI tract (colon, retained rectum/sigmoid or pouch) * Percent change from baseline in the PB in the upper GI tract (duodenum) at 6, 12, 18, 24, 30, and 36 months as observed by upper endoscopy * Percent change from baseline in the PB in the lower GI tract (colon, retained rectum/ sigmoid or pouch) at 6, 12, 18, 24, 30, and 36 months as observed by lower endoscopy
Time frame: 3 years
The effect of eRapa treatment on Spigelman stage score in patients with FAP
Change from baseline in Spigelman stage score at 6, 12, 18, 24, 30, and 36 months
Time frame: 3 years
The effect of eRapa treatment on quality-of-life measures, assessed by the 5 level EuroQoL-5 Dimension (EQ-5D-5L)
-Change from baseline in EQ-5D-5L score at 6, 12, 18, 24, 30, and 36 months;
Time frame: 3 years
Determine the immunomodulating effect of eRapa treatment in patients with FAP
• Change from baseline in T cell phenotypes and function at 12, 24, and 36 months
Time frame: 3 years
The effect of eRapa treatment on quality-of-life measures, assessed by EORTC-30
-Change from baseline in EORTC-30 score at 6, 12, 18, 24, 30, and 36 months;
Time frame: 3 years
Plan to share: Undecided
No publications or documents are linked to this record.
Started Jul 2025 (actual). Primary completion is estimated for Jul 2030, 60 months after the start.
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Studies of the drug tested here: Encapsulated Rapamycin
Rapamycin Holdings Inc.