What this study is
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.
What's being tested
- eRapa (encapsulated rapamycin)
Intervention · 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
Intervention · DRUG
Capsules in 28-count round high-density polyethylene bottles with a polypropylene child-resistant screw cap and foil induction seal.
Study details
- Official title
- A Phase 3, Multi-Site, Prospective, Randomized, Double-Blind, Placebo-Controlled Trial of eRapa to Improve Clinical Outcomes in Patients With Familial Adenomatous Polyposis
- Sponsor
- Rapamycin Holdings Inc.
- Conditions
- Familial Adenomatous Polyposis (FAP)
- Enrolling
- 168 people
- Starts
- 2025-07-18
- Est. completion
- 2031-01
- Design
- Quadruple-blind, randomized, parallel groups, Phase 3
- Record
- View on ClinicalTrials.gov ↗
Who can join
Age 18 Years and overSex ALLHealthy volunteers Not acceptedYou may qualify if
- Participant must be ≥18 years of age inclusive.
- Participant must have documented FAP, confirmed by adenomatous polyposis coli genotype mutation testing.
- 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.
- Contraceptive use by participants or participant partners until at at least 12 weeks after stopping study treatment.
- Agree not to donate gametes for the purpose of reproduction until at at least 12 weeks after stopping study treatment.
- Willing to undergo endoscopic evaluation.
You can't join if
- Participant has unresected or incompletely resected high-grade dysplasia or cancer within the duodenum, colon, rectum, or ileal pouch at screening endoscopy.
- 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).
- Participant has had surgery within 6 weeks of the trial.
- Participant has active malignancy or history of malignancy diagnosed within 24 months of first dose of trial intervention.
- Participant has a history of, or currently has, an acquired or primary (congenital) immunodeficiency.
- Participant has active and clinically significant tuberculosis (positive Quantiferon Gold test), bacterial, fungal, or viral infection, including human immunodeficiency virus (HIV).
- 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.
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
Loading treatment details…
Study locations (29)
29 openCity of Hope
Arcadia, California, United States
- Gregory Idos · Contact626-218-2570gidos@coh.org
- Andrew Fernandez · Contactandfernandez@coh.org
RecruitingYale Cancer Center
New Haven, Connecticut, United States
- Edgar Benitez · Contact2033936591edgar.benitez@yale.edu
RecruitingGeorgetown University
Washington D.C., District of Columbia, United States
- Priyanth Kanth · Contact202-444-1431priyanka.kanth@medstar.net
RecruitingDigestive & Liver Center of Florida
Orlando, Florida, United States
- Alisa Del Vecchio · Contact407-490-4526alisa.delvecchio@dlcfl.com
RecruitingCleveland Clinic Florida
Weston, Florida, United States
- Michael Nicolas · Contact954-659-6213nicolam4@ccf.org
RecruitingUniversity of Chicago
Chicago, Illinois, United States
- Sonia Kupfer · Contact773-702-6140cancerclinicaltrials@bsd.uchicago.edu
- Kristi Kearney · Contactkristi.kearney@uchicagomedicine.org
RecruitingUniversity of Kansas Medical Center
Kansas City, Kansas, United States
- Llana Abella · Contact913-584-0533labella@kumc.edu
RecruitingJohns Hopkins University
Baltimore, Maryland, United States
- Lisa Datta · Contact410-614-1982erapastudy@live.johnshopkins.edu
RecruitingDana Farber Cancer Institute
Boston, Massachusetts, United States
- Samantha Kuney · Contact617-582-9095ColonScreening@dfci.harvard.edu
RecruitingUniversity of Michigan
Ann Arbor, Michigan, United States
- Erika Koeppe · Contact734-998-1274eskoeppe@med.umich.edu
RecruitingDepartment of Surgery, Section of Colon Rectal and Surgery
St Louis, Missouri, United States
- Asima Badic · Contact314- 362-2646asimabadic@wustl.edu
RecruitingCleveland Clinic
Cleveland, Ohio, United States
- Lindsey Reardon · Contact(216) 444-7493Reardol3@ccf.org
RecruitingOhio State University
Columbus, Ohio, United States
- Kebire Gofar · Contact614-600-2113kebire.gofar@osumc.edu
RecruitingGeisinger Medical Center
Danville, Pennsylvania, United States
- Kay Reiner · Contact570-214-5421kmreiner1@geisinger.edu
RecruitingUniversity of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
- Sara Booz · Contact412-864-7516boozs@upmc.edu
RecruitingThe University of Texas MD Anderson Cancer Center
Houston, Texas, United States
- Annie Lincoln · Contact919-923-4430aglincoln@mdanderson.org
RecruitingBenaroya Research Institute at Virginia Mason
Seattle, Washington, United States
- Alicia Reyes · Contact(206)-341-1450mariaalicia.reyes@commonspirit.org
RecruitingUniversity of Washington - Fred Hutchinson
Seattle, Washington, United States
- Brian Forester · Contact(206) 685-1179foerstb@uw.edu
Recruiting
11 sites outside the US
Copenhagen University Hospital
Copenhagen, Denmark
- John Karstensen · Contactjohn.gasdal.karstensen@regionh.dk
Recruiting- Recruiting
IRCCS Ospedale San Raffaele
Milan, Italy
- Giulia Martina Cavestro, M.D · ContactXXX-XXX-XXXXcavestro.giuliamartina@hsr.it
RecruitingIstituto Nazionale dei Tumori
Milan, Italy
- Marco Vitellaro, M.D · Contact+ 39 02 23902540marco.vitellaro@istitutotumori.mi.it
Recruiting- Recruiting
Radboud University Medical Center
Nijmegen, Netherlands
- Tanya Bisseling · Contacttanya.bisseling@radboudumc.nl
RecruitingHospital Oncologico - Puerto Rico Medical Center
Rio Piedras, Puerto Rico
- Ramon Vega · Contact787-407-3333ramon.vega@panoncologytrials.com
Recruiting- Recruiting
- Recruiting
Hospital General Universitario Gregorio Marañón
Madrid, Spain
- Luis Miguel Jiménez-Gomez, M.D · Contact+34 915868522luismiguel.jimenez@salud.madrid.org
Recruiting- Recruiting
Study team
Sponsor
Rapamycin Holdings Inc.
Study ID MTX230-301
Study director
Gary Shangold, MD
Biodexa
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.