Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis
Recruiting · NCT06950385 · Interventional (participants receive a specific treatment) · Lead sponsor: Rapamycin Holdings Inc.
View the official record on ClinicalTrials.gov →Interventions studied
What this trial is about
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.
Who can take part
Inclusion criteria
- 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.
Exclusion criteria
- 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.
Where it is running
29 locations listed across 14 US states.
- City of Hope - Arcadia, California, United States
- Yale Cancer Center - New Haven, Connecticut, United States
- Georgetown University - Washington D.C., District of Columbia, United States
- Digestive & Liver Center of Florida - Orlando, Florida, United States
- Cleveland Clinic Florida - Weston, Florida, United States
- University of Chicago - Chicago, Illinois, United States
- University of Kansas Medical Center - Kansas City, Kansas, United States
- Johns Hopkins University - Baltimore, Maryland, United States
- Dana Farber Cancer Institute - Boston, Massachusetts, United States
- University of Michigan - Ann Arbor, Michigan, United States
- Department of Surgery, Section of Colon Rectal and Surgery - St Louis, Missouri, United States
- Cleveland Clinic - Cleveland, Ohio, United States
- Ohio State University - Columbus, Ohio, United States
- Geisinger Medical Center - Danville, Pennsylvania, United States
- University of Pittsburgh Medical Center - Pittsburgh, Pennsylvania, United States
- The University of Texas MD Anderson Cancer Center - Houston, Texas, United States
- Benaroya Research Institute at Virginia Mason - Seattle, Washington, United States
- University of Washington - Fred Hutchinson - Seattle, Washington, United States
- Amsterdam UMC - Amsterdam, Netherlands
- Hospital Clínic de Barcelona - Barcelona, Spain
- Universitätsklinikum Bonn - Bonn, Germany
- Copenhagen University Hospital - Copenhagen, Denmark
- Hospital Comarcal de Inca - Inca, Spain
- Hospital General Universitario Gregorio Marañón - Madrid, Spain
- IRCCS Ospedale San Raffaele - Milan, Italy
- Istituto Nazionale dei Tumori - Milan, Italy
- Radboud University Medical Center - Nijmegen, Netherlands
- Hospital Oncologico - Puerto Rico Medical Center - Rio Piedras, Puerto Rico
- Hospital La Fe de Valencia - Valencia, Spain