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Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis

NCT ID: NCT06950385

Last Updated: 2025-12-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

168 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-07-18

Study Completion Date

2031-01-31

Brief Summary

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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.

Detailed Description

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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:

* Intact colon versus post-surgical resection with retained rectum/sigmoid or pouch, and
* Duodenal polyposis (current Spigelman stage score ≤2 versus Spigelman stage score ≥3) For the purposes of this trial, high-risk for disease progression is defined as meeting one of the following:
* Patients who have intact colons and have \>100 polyps but ≤500 polyps
* Patients who have retained rectum/sigmoid or ileal-pouch-anal anastomosis and have ≥10 polyps that are ≥3 mm in diameter, or
* Patients who have a history of duodenal polyposis Spigelman stage score of 3 or 4 with at least 1 duodenal polyp that has been removed within 18 months of screening.

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.

Conditions

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Familial Adenomatous Polyposis (FAP)

Keywords

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FAP Polyposis Polyps ileal pouch

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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eRapa

0.5 mg eRapa once a day (QD) every other week

Group Type EXPERIMENTAL

eRapa (encapsulated rapamycin)

Intervention Type 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

Placebo once a day (QD) every other week

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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

Interventions

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eRapa (encapsulated rapamycin)

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.

Intervention Type DRUG

Placebo

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

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Biodexa Pharmaceuticals

INDUSTRY

Sponsor Role collaborator

Rapamycin Holdings Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Vance Sohn, MD

Role: STUDY_DIRECTOR

LumaBridge

Locations

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City of Hope

Arcadia, California, United States

Site Status NOT_YET_RECRUITING

Yale Cancer Center

New Haven, Connecticut, United States

Site Status RECRUITING

Georgetown University

Washington D.C., District of Columbia, United States

Site Status NOT_YET_RECRUITING

Digestive & Liver Center of Florida

Orlando, Florida, United States

Site Status RECRUITING

Cleveland Clinic Florida

Weston, Florida, United States

Site Status RECRUITING

University of Chicago

Chicago, Illinois, United States

Site Status NOT_YET_RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status RECRUITING

Johns Hopkins University

Baltimore, Maryland, United States

Site Status RECRUITING

Dana Farber

Boston, Massachusetts, United States

Site Status NOT_YET_RECRUITING

University of Michigan

Ann Arbor, Michigan, United States

Site Status RECRUITING

Department of Surgery, Section of Colon Rectal and Surgery

St Louis, Missouri, United States

Site Status RECRUITING

Cleveland Clinic

Cleveland, Ohio, United States

Site Status RECRUITING

Ohio State University

Columbus, Ohio, United States

Site Status RECRUITING

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Site Status NOT_YET_RECRUITING

Benaroya Research Institute at Virginia Mason

Seattle, Washington, United States

Site Status NOT_YET_RECRUITING

University of Washington - Fred Hutchinson

Seattle, Washington, United States

Site Status RECRUITING

Copenhagen University Hospital

Copenhagen, , Denmark

Site Status NOT_YET_RECRUITING

Universitätsklinikum Bonn

Bonn, , Germany

Site Status RECRUITING

Amsterdam UMC

Amsterdam, , Netherlands

Site Status NOT_YET_RECRUITING

Radboud University Medical Center

Nijmegen, , Netherlands

Site Status NOT_YET_RECRUITING

Hospital Oncologico - Puerto Rico Medical Center

Rio Piedras, , Puerto Rico

Site Status RECRUITING

Hospital Clínic de Barcelona

Barcelona, , Spain

Site Status NOT_YET_RECRUITING

Hospital Comarcal de Inca

Inca, , Spain

Site Status NOT_YET_RECRUITING

Hospital La Fe de Valencia

Valencia, , Spain

Site Status NOT_YET_RECRUITING

Countries

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United States Denmark Germany Netherlands Puerto Rico Spain

Central Contacts

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Amy Ellenberger

Role: CONTACT

Phone: 210-346-8330

Email: [email protected]

Facility Contacts

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Gregory Idos

Role: primary

Edgar Benitez

Role: primary

Priyanth Kanth

Role: primary

Alisa Del Vecchio

Role: primary

Michael Nicolas

Role: primary

Sonia Kupfer

Role: primary

Llana Abella

Role: primary

Lisa Datta

Role: primary

Samantha Kuney

Role: primary

Erika Koeppe

Role: primary

Asima Badic

Role: primary

Lindsey Reardon

Role: primary

Kebire Gofar

Role: primary

Sara Booz

Role: primary

Alicia Reyes

Role: primary

Brian Forester

Role: primary

John Karstensen

Role: primary

Robert Hüneburg

Role: primary

Evelien Dekker

Role: primary

Tanya Bisseling

Role: primary

Ramon Vega

Role: primary

Frencesc Balaguer Prunes

Role: primary

Jose Reyes Moreno

Role: primary

Marco Bustamante

Role: primary

Other Identifiers

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MTX230-301

Identifier Type: -

Identifier Source: org_study_id